NeoplasmsPhase 3NCT04607421
A Study of Encorafenib Plus Cetuximab With or Without Chemotherapy in People With Previously Untreated Metastatic Colorectal Cancer
A Phase 3 study testing Encorafenib, Cetuximab and Oxaliplatin in people with Neoplasms, run by Pfizer at 274 sites in 29 countries.
Trial summary
The purpose of this study is to evaluate two study medicines (encorafenib plus cetuximab) taken alone or together with standard chemotherapy for the potential treatment of colorectal cancer that: has spread to other parts of the body (metastatic); has a certain type of abnormal gene called "BRAF"; and has not received… Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Neoplasms
- What is being tested?
- Encorafenib, Cetuximab and Oxaliplatin
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 841 participants
- Where
- 274 sitesin 29 countries
- Study dates
- 21 Dec 2020to 29 Jul 2027
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Safety Lead-In = Male/female ≥ 18 years old
- Phase 3 and Cohort 3: Male/female ≥ 16 years old (where permitted locally)
- Histologically or cytologically confirmed Stage IV CRC that contains BRAF V600E mutation
- Prior systemic treatment in metastatic setting: 0-1 regimens for Safety Lead In; none for Phase 3 and Cohort 3. (Note: Prior adjuvant or neoadjuvant therapy considered metastatic treatment if relapse/metastasis < 6 month from end of adj/neoadjuvant treatment )
- Measurable disease (Phase 3 and Cohort 3)/ Measurable or evaluable disease (Safety Lead-in)
- ECOG PS 0-1
- Adequate organ function
Who cannot join
- Tumors that are locally confirmed or unknown MSI-H or dMMR unless participant is ineligible to receive immune checkpoint inhibitors due to a pre-existing medical condition
- Active bacterial or viral infections in 2 weeks prior to starting dosing
- Symptomatic brain metastases
Trial locations and contacts
274 sites in 29 countries.
274 sites in 29 countries
United States63 sites
Mount Sinai Comprehensive Cancer Center, Aventura
Aventura, Florida, 33180
Memorial Sloan Kettering Cancer Center - Basking Ridge
Basking Ridge, New Jersey, 07920
Summit Medical Group
Berkeley Heights, New Jersey, 07922
UChicago Medicine - River East
Chicago, Illinois, 60611
University of Chicago Medical Center
Chicago, Illinois, 60637
Siteman Cancer Center - St Peters
City of Saint Peters, Missouri, 63376
Cleveland Clinic Taussig Cancer Center Investigational Pharmacy
Cleveland, Ohio, 44195
Cleveland Clinic
Cleveland, Ohio, 44195
The Ohio State University James Cancer Hospital and Solove Research Institute
Columbus, Ohio, 43210
The Ohio State University Wexner Medical Center Investigational Drug Services
Columbus, Ohio, 43210
Stefanie Spielman Comprehensive Breast Cancer
Columbus, Ohio, 43212
Martha Morehouse Medical Plaza
Columbus, Ohio, 43221
The James Outpatient Care West Campus
Columbus, Ohio, 43221
Memorial Sloan Kettering Cancer Center Commack
Commack, New York, 11725
Siteman Cancer Center - West County
Creve Coeur, Missouri, 63141
Summit Medical Group
Florham Park, New Jersey, 07932
Siteman Cancer Center - North County
Florissant, Missouri, 63031
UChicago Medicine at Ingalls - Flossmoor
Flossmoor, Illinois, 60422
The West Clinic. PLLC. dba West Cancer Center
Germantown, Tennessee, 38138
Memorial Sloan Kettering Cancer Center - Westchester
Harrison, New York, 10604
UChicago Medicine Ingalls Memorial
Harvey, Illinois, 60426
The University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
Keck Hospital of USC
Los Angeles, California, 90033
LAC & USC Medical Center
Los Angeles, California, 90033
USC / Norris Comprehensive Cancer Center
Los Angeles, California, 90033
USC/Norris Comprehensive Cancer Center/Investigational Drug Services
Los Angeles, California, 90033
USC/Norris Comprehensive Cancer Center
Los Angeles, California, 90033
University of Wisconsin Clinical Science Center
Madison, Wisconsin, 53792
Mount Sinai Comprehensive Cancer Center
Miami Beach, Florida, 33140
Mount Sinai Medical Center
Miami Beach, Florida, 33140
Memorial Sloan Kettering Cancer Center- Monmouth
Middletown, New Jersey, 07748
Memorial Sloan Kettering Cancer Center- Bergen
Montvale, New Jersey, 07645
Henry-Joyce Cancer Clinic
Nashville, Tennessee, 37232
Vanderbilt-Ingram Cancer Center
Nashville, Tennessee, 37232
University of Chicago Comprehensive Cancer Center at Silver Cross Hospital
New Lenox, Illinois, 60451
Ochsner Clinic Foundation
New Orleans, Louisiana, 70121
Memorial Sloan Kettering Cancer Center
New York, New York, 10022
Memorial Sloan Kettering Cancer Center - Main Campus
New York, New York, 10065
University of Oklahoma Health Sciences Center, OU Health Stephenson Cancer Center
Oklahoma City, Oklahoma, 73104
Oncology Hematology West PC dba Nebraska Cancer Specialists
Omaha, Nebraska, 68114
Oncology Hematology West PC dba Nebraska Cancer Specialists
Omaha, Nebraska, 68124
Oncology Hematology West PC dba Nebraska Cancer Specialists
Omaha, Nebraska, 68130
The University of Chicago Medicine Center for Advanced Care Orland Park
Orland Park, Illinois, 60462
Oncology Hematology West PC dba Nebraska Cancer Specialists
Papillion, Nebraska, 68046
Keck Hospital of USC Pasadena
Pasadena, California, 91105
Mayo Clinic Hospital
Phoenix, Arizona, 85054
UPMC Hillman Cancer Center
Pittsburgh, Pennsylvania, 15232
BRCR Global
Plantation, Florida, 33322
BRCR Medical Center Inc.
Plantation, Florida, 33322
Providence Cancer Institute Franz Clinic
Portland, Oregon, 97213
Providence Portland Medical Center
Portland, Oregon, 97213
Providence Onc and Heme Care Clinic - Westside
Portland, Oregon, 97225
Providence St Vincent Medical Center
Portland, Oregon, 97225
Virginia Commonwealth University
Richmond, Virginia, 23219
Mayo Clinic Rochester
Rochester, Minnesota, 55905
Mayo Clinic in Arizona - Scottsdale
Scottsdale, Arizona, 85259
Seattle Cancer Care Alliance
Seattle, Washington, 98109
University of Washington Medical Center
Seattle, Washington, 98195
Barnes- Jewish Hospital
St Louis, Missouri, 63110
Washington University School of Medicine
St Louis, Missouri, 63110
Siteman Cancer Center - South County
St Louis, Missouri, 63129
UChicago Medicine at Ingalls - Tinley Park
Tinley Park, Illinois, 60477
Memorial Sloan Kettering Cancer Center- Nassau
Uniondale, New York, 11553
China21 sites
Cancer Hospital Chinese Academy of Medical Science
Beijing, Beijing Municipality, 100021
Peking University First Hospital
Beijing, Beijing Municipality, 100034
Beijing Cancer hospital
Beijing, Beijing Municipality, 100142
Beijing Hospital
Beijing, Beijing Municipality, 100730
The Second Xiangya Hospital of Central South University
Changsha, Hunan, 410011
The Third Xiangya Hospital of Central South University
Changsha, Hunan, 410013
Sichuan Province Cancer Hospital
Chengdu, Sichuan, 610041
Chongqing University Cancer Hospital
Chongqing, Chongqing Municipality, 400030
Fujian Medical University Union Hospital
Fuzhou, Fujian, 350001
The Sixth Affiliated Hospital of Sun Yat-sen University
Guangzhou, Guangdong, 510655
The second Affiliated Hospital of College of Medicine, Zhejiang University
Hangzhou, Zhejiang, 310000
Shandong province cancer hospital
Jinan, Shandong, 250117
Yunnan Cancer Hospital(The Third Affiliated Hospital of Kunming Medical University)
Kunming, Yunnan, 650118
Nanjing Drum Tower Hospital The Affiliated Hospital of Nanjing University Medical School
Nanjing, Jiangsu, 210008
Affiliated Tumor Hospital of Guangxi Medical University
Nanning, Guangxi, 530200
Shanghai General Hospital
Shanghai, Shanghai Municipality, 200080
Ruijin Hospital Shanghai Jiaotong University School of Medicine
Shanghai, Shanghai Municipality, 201800
Fudan University Shanghai Cancer Center
Shanghai, 201321
Shengjing Hospital Of China Medical University
Shenyang, Liaoning, 110022
Tianjin Union Medical Center
Tianjin, 300000
Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology
Wuhan, Hubei, 430030
Japan19 sites
Chiba cancer center
Chiba, Chiba, 260-8717
National Cancer Center Hospital
Chuo-ku, Tokyo, 104-0045
National Hospital Organization Kyushu Cancer Center
Fukuoka, 811-1395
Saitama Medical University International Medical Center
Hidaka, Saitama, 350-1298
Saitama Prefectural Cancer Center
Ina-machi, Saitama, 362-0806
Kanazawa University Hospital
Kanazawa, Ishikawa-ken, 920-8641
National Cancer Center Hospital East
Kashiwa, Chiba, 277-8577
St. Marianna University Hospital
Kawasaki, Kanagawa, 216-8511
The Cancer Institute Hospital of JFCR
Koto-ku, Tokyo, 135-8550
Aichi Cancer Center Hospital
Nagoya, Nagoya, Aichi, 464-8681
Shizuoka Cancer Center
Nakatogari, Shizuoka, 411-8777
Osaka Prefectural Hospital Organization Osaka International Cancer Institute
Osaka, Osaka, 5418567
National Hospital Organization - Osaka National Hospital - Institute For Clinical Research
Osaka, 540-0006
Hokkaido University Hospital
Sapporo, Hokkaido, 060-8648
Kindai University Hospital
Sayama, Osaka, 589-8511
Keio university hospital
Shinjuku-ku, Tokyo, 160-8582
Osaka University Hospital
Suita, Osaka, 565-0871
Osaka Medical and Pharmaceutical University Hospital
Takatsuki, Osaka, 569-8686
Kanagawa cancer center
Yokohama, Kanagawa, 2418515
Germany13 sites
Onkologische Schwerpunktpraxis Kurfuerstendamm
Berlin, 10707
HELIOS Klinikum Berlin Buch GmbH
Berlin, 13125
Helios Klinikum Berlin-Buch
Berlin, 13125
Radiologie Berlin
Charlottenburg, 10719
Technische Universität Dresden, Medizinische Fakultät Carl Gustav Carus
Dresden, 01307
Universitätsklinikum Carl Gustav Carus Dresden
Dresden, 01307
Institut für Klinisch Onkologische Forschung
Frankfurt am Main, Hesse, 60488
Facharztzentrum Eppendorf
Hamburg, 20249
Israelitisches Krankenhaus IK
Hamburg, 22297
Radiologie im Israelitischen Krankenhaus
Hamburg, 22297
Medizinische Hochschule Hannover
Hanover, Lower Saxony, 30625
Universitätsklinikum Leipzig
Leipzig, Saxony, 04103
Muenchen Klinik Neuperlach, Klinik fuer Haematologie und Onkologie
Munich, Bavaria, 81737
Australia12 sites
Central Adelaide Local Health Network Incorporated
Adelaide, South Australia, 5000
The Queen Elizabeth Hospital
Adelaide, South Australia, 5011
Chris O'Brien Lifehouse
Camperdown, New South Wales, 2050
Monash Health
Clayton, Victoria, 3168
Austin Health
Heidelberg, Victoria, 3084
Royal Brisbane and Women's Hospital
Herston, Queensland, 4029
Liverpool Hospital
Liverpool, New South Wales, 2170
Peter MacCallum Cancer Centre
Melbourne, Victoria, 3000
Alfred Health
Melbourne, Victoria, 3004
GenesisCare - North Shore
St Leonards, New South Wales, 2065
GenesisCare North Shore
St Leonards, New South Wales, 2065
Princess Alexandra Hospital
Woolloongabba, Queensland, 4102
Russia12 sites
GBUZ
Chelyabinsk, 454087
Kaluga Regional Clinical Oncology Center
Kaluga, 248007
FSAEI HE I.M Sechenov First MSMU MoH Russia (Sechenovskiy University),
Moscow, 119991
BHI of Omsk Region "Clinical Oncology Dispensary"
Omsk, 644013
BHI of Omsk Region "Clinical Oncology Dispensary"
Omsk, 644046
Private Medical Institution "Euromedservice"
Pushkin, Sankt-Peterburg, 196603
LLC "Medicina Severnoy Stolitsy"
Saint Petersburg, 191025
LLC "Severo-Zapadny Medical Center"
Saint Petersburg, 192007
Private Healthcare Institution "Clinical Hospital "RZD-Medicine" of St. Petersburg
Saint Petersburg, 195271
LLC "EuroCityClinic"
Saint Petersburg, 197022
FSBI "Russian Scientific Center For Radiology and Surgical Technologies n.a. Academician A.M. Granov
Saint Petersburg, 197758
SHI YR Regional Clinical Oncology Hospital
Yaroslavl, 150054
Spain12 sites
Hospital Universitario Vall d'Hebron
Barcelona, 08035
Hospital Clinic Barcelona
Barcelona, 08036
Hospital General Universitario de Elche
Elche, Alicante, 03203
ICO L'Hospitalet (Hospital Duran i Reynals)
L'Hospitalet de Llobregat, Barcelona, 08908
Hospital General Universitario Gregorio Marañon
Madrid, 28007
Hospital Universitario Ramon Y Cajal
Madrid, 28034
Hospital Universitario 12 de Octubre
Madrid, 28041
Complejo Hospitalario Universitario Santiago de Compostela
Santiago de Compostela, A Coruña, 15706
Hospital Universitario Virgen Del Rocio
Seville, 41013
Hospital Clinico Universitario de Valencia
Valencia, 46010
Hospital General Universitario de Valencia
Valencia, 46014
Hospital Universitario Miguel Servet
Zaragoza, 50009
Italy10 sites
Fondazione Poliambulanza Istituto Ospedaliero
Brescia, 25124
Fondazione del Piemonte per l'Oncologia - Istituto di Candiolo IRCCS
Candiolo, Torino, 10060
ASST Grande Ospedale Metropolitano Niguarda
Milan, Milano, 20162
Istituto Europeo di Oncologia IRCCS
Milan, 20141
Azienda Ospedaliera Universitaria di Cagliari - Presidio Policlinico Universitario "D.Casula"
Monserrato (CA), Cagliari, 09042
Azienda Ospedaliera Universitaria dell'Università "Luigi Vanvitelli" di Napoli
Naples, 80131
Azienda Ospedaliero Universitaria San Luigi Gonzaga
Orbassano, Torino, 10043
IRCCS Istituto Oncologico Veneto (IOV)
Padova, 35128
Azienda USL - IRCCS di Reggio Emilia - Arcispedale Santa Maria Nuova
Reggio Emilia, 42123
IRCCS Casa Sollievo della Sofferenza
San Giovanni Rotondo, Foggia, 71013
Brazil9 sites
Fundação Pio XII - Hospital de Câncer de Barretos
Barretos, São Paulo, 14.780-070
Fundação Pio XII - Hospital de Câncer de Barretos
Barretos, São Paulo, 14784400
Reichow - Centro de Ensino e Pesquisa
Blumenau, Santa Catarina, 89010-340
Clínica de Neoplasias Litoral
Itajaí, Santa Catarina, 88301-220
Hospital de Clinicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, 90035-903
Instituto Nacional de Câncer José Alencar Gomes da Silva - INCA
Rio de Janeiro, Rio de Janeiro, 20230-130
CEPHO - Centro de Estudos e Pesquisas de Hematologia e Oncologia - Faculdade de Medicina do ABC
Santo André, São Paulo, 09060-650
FUNDAÇÃO DO ABC - Faculdade de Medicina do ABC - Centro de Estudos e Pesquisas de Hematologia e Onco
Santo André, São Paulo, 09060-870
Fundação Faculdade Regional de Medicina de São José do Rio Preto
São José do Rio Preto, São Paulo, 15090000
South Korea9 sites
Dong-A University Hospital
Busan, 49201
Kyungpook National University Chilgok Hospital
Daegu, 41404
National Cancer Center
Goyang-si, Gyeonggi-do, 10408
Gachon University Gil Medical Center
Incheon, 21565
Korea University Anam Hospital
Seoul, 02841
Seoul National University Hospital
Seoul, 03080
Severance Hospital, Yonsei University Health System
Seoul, 03722
Asan Medical Center
Seoul, 05505
Samsung Medical Center
Seoul, 06351
Belgium8 sites
ZNA Middelheim
Antwerp, 2020
Université Libre de Bruxelles - Hôpital Erasme
Brussels, Bruxelles-capitale, Région de, 1070
Cliniques universitaires Saint-Luc
Brussels, Bruxelles-capitale, Région de, 1200
Grand Hôpital de Charleroi
Charleroi, Hainaut, 6060
AZ Groeninge Campus Kennedylaan
Kortrijk, West-vlaanderen, 8500
UZ Leuven
Leuven, 3000
Centre Hospitalier Universitaire de Liège - Domaine Universitaire du Sart Tilman
Liège, 4000
ZAS Augustinus
Wilrijk, 2610
Finland8 sites
Docrates Syöpäsairaala
Helsinki, Uusimaa, 00180
Helsinki University Central Hospital
Helsinki, Uusimaa, 00290
Oulu University Hospital
Oulu, 90220
Satakunnan Keskussairaala
Pori, 28500
Tampereen yliopistollinen sairaala
Tampere, 33520
Tampereen yliopistollinen sairaala
Tampere, 33521
Turku University Hospital
Turku, Southwest Finland, 20520
Turku University Hospital
Turku, 20520
Taiwan8 sites
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, 807
China Medical University Hospital
Taichung, 404
National Cheng-Kung University Hospital
Tainan, 704
Chi Mei Hospital, Liouying
Tainan, 73657
National Taiwan University Hospital
Taipei, 100
Taipei Medical University Hospital
Taipei, 11031
Taipei Veterans General Hospital
Taipei, 112
Chang Gung Medical Foundation-Linkou Branch
Taoyuan, 333
Bulgaria7 sites
MHAT "Dr. Tota Venkova" AD
Gabrovo, 5300
MHAT Uni Hospital OOD
Panagyurishte, Pazardzhik, 4500
MHAT Central Onco Hospital OOD
Plovdiv, 4000
Complex Oncology Center - Plovdiv EOOD
Plovdiv, 4004
Medical Center Nadezhda Clinical EOOD
Sofia, 1303
Acibadem City Clinic MHAT Tokuda
Sofia, 1407
University Multiprofile Hospital for Active Treatment Sofiamed
Sofia, 1750
Canada7 sites
Alberta Health Services - Cancer Care, Tom Baker Cancer Centre
Calgary, Alberta, T2N 4N2
Arthur J.E. Child Comprehensive Cancer Centre
Calgary, Alberta, T3N 4N1
Cross Cancer Institute
Edmonton, Alberta, T6G 1Z2
Alberta Health Services and The Governors of The University of Alberta
Edmonton, Alberta, T6G 2C8
London Regional Cancer Program, London Health Sciences Centre
London, Ontario, N6A 5W9
Jewish General Hospital
Montreal, Quebec, H3T 1E2
Sunnybrook Health Sciences Centre
Toronto, Ontario, M4N 3M5
Denmark6 sites
Aalborg Universitetshospital, Syd
Aalborg, North Denmark, 9000
Rigshospitalet
Copenhagen, 2100
Herlev and Gentofte Hospital
Herlev, 2730
Odense University Hospital
Odense C, 5000
Vejle Hospital-Sygehus Lillebaelt
Vejle, Region Syddanmark, 7100
Vejle Sygehus
Vejle, Region Syddanmark, 7100
India6 sites
Sawai Man Singh Medical College Hospital (SMS Hospital)
Jaipur, Rajasthan, 302004
Tata Memorial Hospital
Mumbai, Maharashtra, 400012
Rajiv Gandhi Cancer Institute And Research Centre
New Delhi, National Capital Territory of Delhi, 110085
Deenanath Mangeshkar Hospital & Research Centre
Pune, Maharashtra, 411 004
Sahyadri Speciality Hospital
Pune, Maharashtra, 411 004
Bhakti Vedanta Hospital and Research Institute
Thane, Maharashtra, 401107
United Kingdom6 sites
Heartlands Hospital
Birmingham, B9 5SS
Hammersmith Hospital, Imperial College Healthcare NHS Trust
London, W12 0HS
Hammersmith Hospital
London, W6 8RF
Freeman Hospital
Newcastle upon Tyne, HIGH Heaton, NE7 7DN
Churchill Hospital - Oncology
Oxford, OX3 7LE
Royal Marsden NHS Foundation Trust
Sutton, Surrey, SM2 5PT
Czechia5 sites
Fakultní nemocnice Brno Bohunice
Brno, Brno-město, 625 00
Fakultni nemocnice Hradec Kralove
Hradec Králové, Hradec Králové, 500 05
Fakultni nemocnice Olomouc
Olomouc, 779 00
Fakultni Thomayerova nemocnice
Prague, Praha 4, 14059
Fakultni nemocnice Bulovka
Prague, 180 81
Poland5 sites
Szpital Specjalistyczny W Brzozowie, Podkarpacki Osrodek Onkologiczny Im.Ks.B.Markiewicza
Brzozów, 36-200
Wojewodzki Szpital Specjalistyczny Nr 4 w Bytomiu Oddzial Onkologii
Bytom, 41-902
COPERNICUS PL sp. z. o. o. Wojewodzkie Centrum Onkologii w Gdansku Ambulatoryjna
Gdansk, 80-219
COPERNICUS Podmiot Leczniczy Sp. z o.o. Wojewodzkie Centrum Onkologii
Gdansk, 80-219
Przychodnia Lekarska KOMED
Konin, Greater Poland Voivodeship, 62-500
Sweden5 sites
Sahlgrenska Universitetssjukhuset
Gothenburg, Västra Götalands LÄN [se14], 413 45
Karolinska Universitetssjukhuset Solna
Solna, Stockholms LÄN [se-01], 171 64
Department of Pelvic cancer, colorecta section, Karolinska University Hospital
Stockholm, 171 76
Norrlands universitetssjukhus
Umeå, Västerbottens LÄN [se-24], 90185
Akademiska sjukhuset
Uppsala, Uppsala LÄN [se-03], 751 85
Argentina4 sites
Instituto Médico Especializado Alexander Fleming
Buenos Aires, 1426
Clinica Universitaria Reina Fabiola
Córdoba, X5004FHP
Hospital Privado Centro Médico de Córdoba
Córdoba, X5016KEH
Centro Medico San Roque
San Miguel de Tucumán, Tucumán Province, 4000
Norway4 sites
Sørlandet Sykehus Kristiansand
Kristiansand, Vest-agder, N-4615
Oslo universitetssykehus, Radiumhospitalet
Oslo, 0379
Oslo Universitetssykehus Ullevål
Oslo, 0450
St. Olavs hospital
Trondheim, Sør-trøndelag, 7030
Ukraine4 sites
Municipal Non-profit Enterprise "City Clinical Hospital #4" of Dnipro City Council
Dnipro, 49102
Ivano-Frankivsk National Medical University
Ivano-Frankivsk, 76018
MNPE "Prykarpatski Clinical Oncological Center" of Ivano-Frankivsk Regional Council"
Ivano-Frankivsk, 76018
Communal enterprise "Kryvyi Rih Oncology Dispensary" of Dnipropetrovsk Regional Council
Kryvyi Rih, 50048
Netherlands3 sites
Nederlands Kanker Instituut - Antoni van Leeuwenhoek (NKI-AVL)
Amsterdam, North Holland, 1066 CX
Catharina Ziekenhuis
Eindhoven, North Brabant, 5623 EJ
Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX
South Africa3 sites
Wits Health Consortium (Pty) Ltd
Johannesburg, 2193
Cancercare Langenhoven Drive Oncology Centre
Port Elizabeth, Eastern Cape, 6045
Cancercare Rondebosch Oncology
Rondebosch, CAPE TOWN, 7700
Mexico2 sites
Accelerium, S. de R.L. de C.V.
Monterrey, Nuevo León, 64000
Centro de Investigacion Clinica de Oaxaca
Oaxaca City, 68020
Slovakia2 sites
Narodny Onkologicky Ustav
Bratislava, 833 10
Vychodoslovensky onkologicky ustav, a.s.
Košice, 04191
About this study
Official title: AN OPEN-LABEL, MULTICENTER, RANDOMIZED PHASE 3 STUDY OF FIRST-LINE ENCORAFENIB PLUS CETUXIMAB WITH OR WITHOUT CHEMOTHERAPY VERSUS STANDARD OF CARE THERAPY WITH A SAFETY LEAD-IN OF ENCORAFENIB AND CETUXIMAB PLUS CHEMOTHERAPY IN PARTICIPANTS WITH METASTATIC BRAF V600E-MUTANT COLORECTAL CANCER
The purpose of this study is to evaluate two study medicines (encorafenib plus cetuximab) taken alone or together with standard chemotherapy for the potential treatment of colorectal cancer that:
- has spread to other parts of the body (metastatic);
- has a certain type of abnormal gene called "BRAF"; and
- has not received prior treatment.
Read more
Participants in this study will receive one of the following study treatments:
- Encorafenib plus cetuximab: These participants will receive encorafenib by mouth at home every day and cetuximab once every two weeks by intravenous (IV) infusion (an injection into the vein) at the study clinic.
- Encorafenib plus cetuximab with chemotherapy: These participants will receive encorafenib and cetuximab in the way described in the bullet above. Additionally, they will receive standard chemotherapy by IV infusion and oral treatment at home.
- Chemotherapy alone: These participants will receive chemotherapy, the standard treatment for this condition, by IV infusion at the study clinics and oral treatment at home.
This study is currently enrolling participants who will receive either encorafenib plus cetuximab with chemotherapy or chemotherapy alone.
The study team will monitor how each participant responds to the study treatment for up to about 3 years.
Read the detailed description
The purpose of the study is to evaluate whether encorafenib plus cetuximab (EC), alone or in combination with chemotherapy, can improve clinical outcomes relative to current standard of-care chemotherapy in participants with previously untreated BRAF V600E-mutant mCRC. Since encorafenib has not previously been combined with chemotherapy, the tolerability and PK of EC in combination with mFOLFOX6 and in combination with FOLFIRI will be evaluated in separate cohorts in the safety lead-in portion of the trial in order to identify which chemotherapy combination is to be used in the Phase 3 portion of the study.
- Conditions
- Neoplasms
- Being tested
- Encorafenib, Cetuximab, Oxaliplatin, Irinotecan, Leucovorin
- Keywords
- Colorectal cancer, Colon cancer, Rectal cancer, Colorectal Neoplasms, Intestinal cancer, Intestinal Neoplasms, Gastrointestinal cancer, Gastrointestinal Neoplasms, Digestive system cancer, Digestive System Neoplasms, Digestive System Diseases, Gastrointestinal Diseases, Colonic Diseases, Intestinal Diseases, Rectal Diseases, BREAKWATER Study
- Medical subject headings
- Neoplasms, Colorectal Neoplasms, Colonic Neoplasms, Rectal Neoplasms, Intestinal Neoplasms, Gastrointestinal Neoplasms, Digestive System Neoplasms, Digestive System Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EncorafenibDrug
Also called Braftovi, PF-07263896, LGX818, ONO-7702
75 mg capsules
CetuximabDrug
Also called Erbitux
Injection for intravenous use 100 mg/vial, 200 mg/vial, or 500 mg/vial
OxaliplatinDrug
Also called Eloxatin
Powder for solution for intravenous use 50 mg/vial, 100 mg/vial, or 200 mg/vial
IrinotecanDrug
Also called Campostar
Solution for intravenous infusion 40 mg/vial, 100 mg/vial, or 300 mg/vial
LeucovorinDrug
Also called Wellcovorin, Fusilev, Khapzory
Injection 50 mg/vial, 100 mg/vial, 200 mg/vial, or 350 mg/vial
5-FUDrug
Also called Fluorouracil
Injection for intravenous use 250 mg/vial, 500 mg/vial, or 1000 mg/vial
CapecitabineDrug
Also called Xeloda
150 mg or 500 mg Tablet
BevacizumabDrug
Also called Zirabev
Optional Injection for intravenous use 100 mg/vial or 400 mg/vial
| Study group | Role | What they receive |
|---|---|---|
| Safety Lead-in Cohort 1 | Experimental | Encorafenib 300 mg orally once daily Cetuximab 500 mg/m2 (120-minute IV infusion) every two weeks Irinotecan 180 mg/m2 (90-minute IV infusion) every two weeks Leucovorin 400 mg/m2 (120-minute IV infusion) every two weeks 5-FU 400 mg/m2 IV bolus, then 5-FU 2400 mg/m2 continuous IV infusion over 46-48 hours every two weeks Receives: Drug: Encorafenib; Drug: Cetuximab; Drug: Irinotecan; Drug: Leucovorin; Drug: 5-FU |
| Safety Lead-in Cohort 2 | Experimental | Encorafenib 300 mg orally once daily Cetuximab 500 mg/m2 (120 minute IV infusion) every two weeks Oxaliplatin 85 mg/m2 (120-minute IV infusion) every two weeks Leucovorin 400 mg/m2 (120-minute IV infusion) every two weeks 5-FU 400 mg/m2 IV bolus, then 5-FU 2400 mg/m2 continuous IV infusion over 46-48 hours every two weeks Receives: Drug: Encorafenib; Drug: Cetuximab; Drug: Oxaliplatin; Drug: Leucovorin; Drug: 5-FU |
| Phase 3 Arm A | Experimental | Encorafenib 300 mg orally once daily Cetuximab 500 mg/m2 (120-minute IV infusion) every two weeks Receives: Drug: Encorafenib; Drug: Cetuximab |
| Phase 3 Arm B | Experimental | Encorafenib 300 mg orally once daily Cetuximab 500 mg/m2 (120 minute IV infusion) every two weeks Oxaliplatin 85 mg/m2 (120-minute IV infusion) every two weeks Leucovorin 400 mg/m2 (120-minute IV infusion) every two weeks 5-FU 400 mg/m2 IV bolus, then 5-FU 2400 mg/m2 continuous IV infusion over 46-48 hours every two weeks Receives: Drug: Encorafenib; Drug: Cetuximab; Drug: Oxaliplatin; Drug: Leucovorin; Drug: 5-FU |
| Phase 3 Arm C | Active comparator | Every two weeks: Oxaliplatin 85 mg/m2 (120-minute IV infusion) Leucovorin 400 mg/m2 (120-minute IV infusion) 5-FU 400 mg/m2 IV bolus, then 5-FU 2400 mg/m2 continuous IV infusion over 46-48 hours Bevacizumab (optional; given per prescribing instructions) -OR- Every two weeks: Irinotecan 165 mg/m2 (90-minute IV infusion) Oxaliplatin 85 mg/m2 (120-minute IV infusion) Leucovorin 400 mg/m2 (120-minute IV infusion) 5-FU 2400 or 3200 mg/m2 continuous IV infusion over 46 48 hours Bevacizumab (optional; given per prescribing instructions) -OR- Oxaliplatin 130 mg/m2 (120-minute IV infusion) every 3 weeks Capecitabine 1000 mg/m2 oral tablet twice daily on Days 1-14 Bevacizumab (optional; given per prescribing instructions) Receives: Drug: Oxaliplatin; Drug: Irinotecan; Drug: Leucovorin; Drug: 5-FU; Drug: Capecitabine; Drug: Bevacizumab |
| Cohort 3 Arm D | Experimental | Encorafenib 300 mg orally once daily Cetuximab 500 mg/m2 (120-minute IV infusion) every two weeks Irinotecan 180 mg/m2 (90-minute IV infusion) every two weeks Leucovorin 400 mg/m2 (120-minute IV infusion) every two weeks 5-FU 400 mg/m2 IV bolus, then 5-FU 2400 mg/m2 continuous IV infusion over 46-48 hours every two weeks Receives: Drug: Encorafenib; Drug: Cetuximab; Drug: Irinotecan; Drug: Leucovorin; Drug: 5-FU |
| Cohort 3 Arm E | Active comparator | Irinotecan 180 mg/m2 (90-minute IV infusion) every 2 weeks, Leucovorin 400 mg/m2 (120-minute IV infusion) every 2 weeks, 5-FU 400 mg/m2 IV bolus, then 5-FU 2400 mg/m2 continuous IV infusion over 46-48 hours every two weeks, Bevacizumab (optional; given per prescribing instructions) Receives: Drug: Irinotecan; Drug: Leucovorin; Drug: 5-FU; Drug: Bevacizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
SLI: Number of Participants With Dose Limiting Toxicity (DLTs)
DLT: Any AE/lab value during first 28 days (D) of treatment that met at least 1 criteria: AE/lab value unrelated to underlying disease,PD,intercurrent illness,concomitant medications resulting in inability to tolerate atleast 75% of planned dose intensity of study drug,fatigue grade (G)3>14 consecutive D, interstitial lung disease G>=2,rash,hand foot skin reaction G3>14 consecutive D or G4,diarrhea G3 >=48 hours or G4,nausea/vomiting G3>=48 hours or G4,mucositis G>=3,total bilirubin G>=3, aspartate aminotransferase/alanine aminotransferase G>=3 in conjunction with total bilirubin G>=2 or G3 >7 consecutive D or G4,Serum creatinine G>=3,absolute neutrophil count G4 >7 consecutive D, >=G3 febrile neutropenia,G3 platelet count decreased with signs of bleeding,platelet count G4,ECG QTcF prolonged >=G3,G>=3 uveitis >21 consecutive D,G4 confirmed by ophthalmic examination,paresthesia/dysesthesias G>=3,other G>=3 hematologic/nonhematologic toxicity except lymphocyte count decreased G>=3.
Measured: Cycle 1 (28 days)
Phase 3: Progression Free Survival (PFS) as Assessed by Blinded Independent Central Review (BICR) for Arm B vs Arm C - FAS
PFS was defined as the time from date of randomization to earliest documented disease progression (PD) per Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 or death due to any cause as assessed by BICR. PD: 20% increase in sum of diameters of target measurable lesions above smallest sum observed, with minimum absolute increase of 5 millimeter (mm) for target disease or unequivocal progression of pre-existing lesions for non-target disease or any new lesion. Censoring details: no adequate baseline assessment, including no disease at baseline: participants were censored to randomization date; no PD, lost to follow-up, withdrawal of consent, PD or death >12 (or 16) weeks after the last adequate tumor assessment, and start of new anticancer treatment: participants were censored to last adequate tumor assessment documenting no PD prior to new anticancer therapy, or missed assessments. Analysis was performed using Kaplan Meier method.
Measured: From date of randomization to earliest documentation of PD by BICR or death or censoring date, whichever occurred first (maximum up to 37.25 months)
Phase 3: Objective Response Rate (ORR) as Assessed by BICR for Arm B vs Arm C - FAS ORR Subset
ORR was defined as the percentage of participants who achieved best overall response (BOR) of confirmed complete response (CR) or partial response (PR) according to RECIST v 1.1 as assessed by BICR. CR: complete disappearance of all target lesions (with the exception of nodal disease) and non-target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm. PR: >=30% decrease under baseline of the sum of diameters of all target measurable lesions. PD: 20% increase in sum of diameters of target measurable lesions above smallest sum observed, with minimum absolute increase of 5 mm for target disease or unequivocal progression of pre-existing lesions for non-target disease or any new lesion.
Measured: From date of randomization to until documented PD by BICR, or start of subsequent anticancer therapy or death, whichever occurred first (maximum up to 24.71 months)
Cohort 3: ORR as Assessed by BICR for Arm D vs Arm E - FAS
ORR was defined as the percentage of participants who achieved BOR of confirmed CR or PR according to RECIST v 1.1 as assessed by BICR. CR: complete disappearance of all target lesions (with the exception of nodal disease) and non-target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm. PR: >=30% decrease under baseline of the sum of diameters of all target measurable lesions. PD: 20% increase in sum of diameters of target measurable lesions above smallest sum observed, with minimum absolute increase of 5 mm for target disease or unequivocal progression of pre-existing lesions for non-target disease or any new lesion.
Measured: From date of randomization until documented PD by BICR, or start of subsequent anticancer therapy or death, whichever occurred first (maximum up to 14.1 months)
Secondary outcomes (82)
SLI: Number of Participants With Adverse Events (AEs)
An AE was any untoward medical occurrence in clinical study participant temporally associated with use of study intervention, whether/ not considered related to study intervention. An Serious adverse event (SAE) was defined as any untoward medical occurrence that, at any dose, met one or more of the following criteria - resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, or other important medical event. AEs included both SAEs and all non-SAEs.
Measured: Through end of the study
SLI: Number of Participants With Grade 3 or 4 AEs and Grade 5 AEs
An AE was any untoward medical occurrence in clinical study participant temporally associated with use of study intervention, whether or not considered related to study intervention. AEs were graded according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version (v) 4.03 where grade 1= mild, grade 2= moderate, grade 3= severe, grade 4= life-threatening and grade 5= death related to AE. In this outcome measure, number of participants with grade 3 or 4 AEs and grade 5 AEs were reported.
Measured: Through end of the study
SLI: Number of Participants With Shift From Grade <=2 at Baseline to Grade >=3 Post-Baseline in Hematology and Coagulation Parameters
The following hematology and coagulation parameters were assessed: activated partial thromboplastin time prolonged, anemia, hemoglobin increased, international normalized ratio (INR) increased, leukocytosis, neutrophil count decreased, platelet count decreased, and white blood cell decreased. Laboratory abnormalities were graded according to NCI CTCAE version 4.03 where grade 1= mild, grade 2= moderate, grade 3= severe, grade 4= life-threatening event due to AE, and grade 5= death. Only those hematology and coagulation parameters in which at least 1 participant in any of the reporting arm had any shift from grade <=2 at baseline to grade >=3 post-baseline are reported in this outcome measure.
Measured: Through end of the study
SLI: Number of Participants With Shift From Grade <=2 at Baseline to Grade >=3 Post-Baseline in Chemistry Parameters
The following chemistry parameters were assessed: alanine aminotransferase increased, alkaline phosphatase increased, aspartate aminotransferase increased, blood bilirubin increased, creatinine increased, hypercalcemia, hyperglycemia, hyperkalemia, hypermagnesemia, hypernatremia, hypoalbuminemia, hypocalcemia, hypoglycemia, hypokalemia, hypomagnesemia, hyponatremia, and lipase increased. Laboratory abnormalities were graded according to NCI CTCAE v4.03 where grade 1= mild, grade 2= moderate, grade 3= severe, grade 4= life-threatening, and grade 5= death related to AE. Only those chemistry parameters in which at least 1 participant in any of the reporting arm had any shift from grade <=2 at baseline to grade >=3 post-baseline are reported in this outcome measure.
Measured: Through end of the study
SLI: Number of Participants According to Categorization of Vital Signs Data
The criteria for vital signs included: Systolic blood pressure (millimeters of mercury [mmHg]): value <= 90 mmHg and decrease from baseline >= 20 mmHg, and value >= 160 mmHg and increase from baseline >= 20 mmHg. Diastolic blood pressure (mmHg): value <= 50 mmHg and decrease from baseline >= 15 mmHg, and value >= 100 mmHg and increase from baseline >= 15 mmHg. Pulse rate (beats per minute [bpm]): value <= 50 bpm and decrease from baseline >= 15 bpm, and value >= 120 bpm and increase from baseline >= 15 bpm. Weight (kilograms [kg]): change >= 20 % decrease from baseline, and change >=10% increase from baseline. Temperature (degree Celsius): value <=36 degree Celsius, and value >=37.5 degree Celsius. Only those criteria in which at least 1 participant in any of the reporting arm had any vital signs data are reported in this outcome measure.
Measured: Through end of the study
SLI: Number of Participants According to Categorization of Electrocardiogram (ECGs) Findings
ECG criteria included: ECG mean heart rate (beats per minute [bpm]): increase from baseline >25% and to a value >100 bpm; decrease from baseline >25% and to a value <50 bpm, PR interval not otherwise specified (milliseconds [msec]): new >280 msec, QRS interval not otherwise specified (msec): new >120 msec, QT Interval Corrected Using Fridericia's Formula (QTcF) not otherwise specified: new >450 msec; new >480 msec; new >500 msec; increase from baseline >30 msec and increase from baseline >60 msec. Only those criteria in which at least 1 participant in any of the reporting arm had any ECG findings are reported in this outcome measure.
Measured: Through end of the study
SLI: Number of Participants With Dose Modification of Any Study Intervention Due to AEs
An AE was any untoward medical occurrence in clinical study participant temporally associated with use of study intervention, whether or not considered related to study intervention. Dose interruption: for encorafenib = 0 mg dose administered for >=1 days; for cetuximab, oxaliplatin, leucovorin, fluorouracil, irinotecan: >20 days between successive start dates with non-zero actual doses. Dose reduction: decrease in dose of at least 10%, from the protocol-planned dose and a decrease from the previous non-zero dose; for encorafenib to qualify as a dose reduction, it should have lasted for >=2 days. Dose modifications included both dose interruptions and reduction.
Measured: Through end of the study
SLI: Number of Participants With Dose Discontinuation of Any Study Intervention Due to AEs
An AE was any untoward medical occurrence in clinical study participant temporally associated with use of study intervention, whether or not considered related to study intervention. Number of participants with dose discontinuation due to AEs were reported in this outcome measure.
Measured: Through end of the study
SLI: ORR as Assessed by Investigator According to Line of Therapy - FAS
ORR: percentage of participants who achieved BOR of confirmed CR/PR per RECIST v1.1 as assessed by response reported by investigator on eCRF. CR: complete disappearance of all target lesions (with exception of nodal disease) and non-target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis <10 mm. PR: >=30% decrease under baseline of sum of diameters of all target measurable lesions. PD: 20% increase in sum of diameters of target measurable lesions above smallest sum observed, with minimum absolute increase of 5 mm for target disease or unequivocal progression of pre-existing lesions for non-target disease or any new lesion. Percentage of participants with ORR for first line (no prior treatment) and second line (participant received prior treatment viz. advanced/metastatic or locoregional disease or maintenance or if neoadjuvant/adjuvant with a disease recurrence occurred during or within 6 months of last therapy dose) is presented.
Measured: From date of randomization until documented PD, or start of subsequent anticancer therapy or death, whichever occurred first (maximum up to 33.9 months)
SLI: Duration of Response (DOR) as Assessed by Investigator According to Line of Therapy - FAS
DOR: time from date of first radiographic evidence of response (CR/PR) to earliest documented PD per RECIST v1.1 as assessed by response reported by investigator on eCRF, or death by any cause. CR: disappearance of all target lesions and non-target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis <10 mm. PR: >=30% decrease under baseline of sum of diameters of all target measurable lesions. PD: 20% increase in sum of diameters of target measurable lesions above smallest sum observed, with minimum absolute increase of 5 mm for target disease or unequivocal progression of pre-existing lesions for non-target disease or any new lesion. DOR for first line and second line is presented. Analysis performed by Kaplan Meier method.
Measured: From date of first radiographic evidence of response (CR or PR) to earliest documented PD or death due to any cause whichever occurred first (maximum up to 33.9 months)
SLI: PFS as Assessed by Investigator According to Line of Therapy - FAS
PFS: time from date of randomization to earliest documented PD per RECIST version 1.1 or death due to any cause as assessed by response reported by investigator on eCRF. PD: 20% increase in sum of diameters of target measurable lesions above smallest sum observed, with minimum absolute increase of 5 mm for target disease or unequivocal progression of pre-existing lesions for non-target disease or any new lesion. Censoring details: no adequate baseline assessment, including no disease at baseline: participants were censored to randomization date; no PD, lost to follow-up, withdrawal of consent, PD or death >12 (or 16) weeks after the last adequate tumor assessment, and start of new anticancer treatment: participants were censored to last adequate tumor assessment documenting no PD prior to new anticancer therapy, or missed assessments. Participants with PFS for first line and second line were presented. Analysis performed using Kaplan Meier method.
Measured: From date of randomization to earliest documentation of PD or death or censoring date, whichever occurred first (maximum up to 33.9 months)
SLI: Time to Response (TTR) as Assessed by Investigator According to Line of Therapy - FAS
TTR was defined as the time from the date of first dose to first radiographic evidence of response (CR or PR) per RECIST v1.1 as assessed by response reported by investigator on eCRF. CR: disappearance of all target lesions and non-target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm. PR: >= 30% decrease under baseline of the sum of diameters of all target measurable lesions. In this outcome measure, TTR for first line (no prior treatment) and second line (participant received a prior treatment defined as advanced/metastatic or locoregional disease or maintenance or if neoadjuvant/adjuvant with a disease recurrence occurred during or within 6 months of the last dose of therapy) were reported.
Measured: From date of first dose to first radiographic evidence of response (CR or PR) (maximum up to 33.9 months [147.3 weeks])
SLI: Overall Survival (OS) According to Line of Therapy - FAS
OS was defined as the time from the date of first dose to death due to any cause. If a participant was not known to have died at the time of the cutoff for analysis, then OS was censored at the date of last contact. In this outcome measure, OS for first line (no prior treatment) and second line (participant received a prior treatment defined as advanced/metastatic or locoregional disease or maintenance or if neoadjuvant/adjuvant with a disease recurrence occurred during or within 6 months of the last dose of therapy) was presented. Analysis was performed using Kaplan Meier method.
Measured: From date of first dose to death due to any cause or censoring date, whichever occurred first (maximum up to 33.9 months)
SLI: Maximum Plasma Concentration (Cmax) of Encorafenib and Its Metabolite LHY746
Cmax was observed directly from data. LHY746 is a metabolite of Encorafenib.
Measured: Cohort 1: Pre-dose (0 hour [hr]), 0.75, 1.5, 2.5, 3.5, 5.5, 7.5, and 48 hrs post-dose on Day 15 of Cycle 1; Cohort 2: Pre-dose (0 hr), 1, 2, 3, 4, 6, 8, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI: Area Under the Concentration Time Profile From Time Zero to 6 Hours (AUC6) for Encorafenib and Its Metabolite LHY746
The AUC was estimated from time 0 to 6 hours post dose. AUC6 was computed using the Linear/Log trapezoidal method. LHY746 is a metabolite of Encorafenib.
Measured: Cohort 1: Pre-dose (0 hr), 0.75, 1.5, 2.5, 3.5, 5.5, and 6 hrs post-dose on Day 15 of Cycle 1; Cohort 2: Predose (0 hr), 1, 2, 3, 4, and 6 hrs post-dose on Days 1 and 15 of Cycle 1
SLI: Area Under the Concentration-time Profile From Time Zero to the Time Tau (AUCtau) of Encorafenib and Its Metabolite LHY746
AUCtau was area under the concentration-time profile from time zero to time tau, the dosing interval. tau = 24 hrs for QD dosing of encorafenib. LHY746 is a metabolite of encorafenib. AUCtau can be calculated directly from the data using 24 hrs (tau) sample or can be approximately calculated by kel, where kel is the terminal phase rate constant calculated by a linear regression of the log-linear concentration-time curve.
Measured: Cohort 1: Pre-dose (0 hr), 0.75, 1.5, 2.5, 3.5, 5.5, 7.5, and 48 hrs post-dose on Day 15 of Cycle 1; Cohort 2: Pre-dose (0 hr), 1, 2, 3, 4, 6, 8, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI: Time to Maximum Plasma Concentration (Tmax) of Encorafenib and Its Metabolite LHY746
Tmax: time (hours) to Cmax. LHY746 is a metabolite of Encorafenib.
Measured: Cohort 1: Pre-dose (0 hr), 0.75, 1.5, 2.5, 3.5, 5.5, 7.5, and 48 hrs post-dose on Day 15 of Cycle 1; Cohort 2: Pre-dose (0 hr), 1, 2, 3, 4, 6, 8, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI: Apparent Total Clearance (CL/F) of Encorafenib
Clearance of a drug is a measure of the rate at which a drug is metabolized or eliminated by normal biological processes. CL/F was calculated as Dose/AUCinf where dose is the dose administered divided by area under the concentration curve from time 0 extrapolated to infinite time (AUCinf). AUCinf was calculated as AUClast + (Clast*/kel), where Clast is last plasma concentration from the log-linear regression analysis, and kel is the terminal phase rate constant calculated by a linear regression of the log-linear concentration-time curve.
Measured: Cohort 1: Pre-dose (0 hr), 0.75, 1.5, 2.5, 3.5, 5.5, 7.5, and 48 hrs post-dose on Day 15 of Cycle 1; Cohort 2: Pre-dose (0 hr), 1, 2, 3, 4, 6, 8, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI, Cohort 1: Cmax of Irinotecan and Its Metabolite SN-38
Cmax was observed directly from data. SN-38 is a metabolite of Irinotecan.
Measured: Cohort 1: Pre-dose (0 hr), 0.75, 1.5, 2.5, 3.5, 5.5, 7.5, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI, Cohort 1: AUClast of Irinotecan and Its Metabolite SN-38
AUClast was defined as area under the plasma concentration time curve from time zero to the last measurable concentration. AUClast was calculated using linear/log trapezoidal method. SN-38 is a metabolite of Irinotecan.
Measured: Cohort 1: Pre-dose (0 hr), 0.75, 1.5, 2.5, 3.5, 5.5, 7.5, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI, Cohort 1: Apparent Terminal Elimination Half-Life (t1/2) of Irinotecan and Its Metabolite SN-38
t1/2 was the time measured for the drug concentration to decrease by one half. t1/2 was determined by Loge (2)/kel, where kel is the terminal phase rate constant calculated by a linear regression of the log-linear concentration-time curve. SN-38 is a metabolite of Irinotecan.
Measured: Cohort 1: Pre-dose (0 hr), 0.75, 1.5, 2.5, 3.5, 5.5, 7.5, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI, Cohort 1: CL/F of Irinotecan
Clearance of a drug is a measure of the rate at which a drug is metabolized or eliminated by normal biological processes. CL/F was calculated as dose administered divided by area under the concentration curve from time 0 extrapolated to infinite time (AUCinf). AUCinf was calculated as AUClast + (Clast*/kel), where Clast is the predicted plasma concentration at the last quantifiable time point from the log-linear regression analysis, and kel is the terminal phase rate constant calculated by a linear regression of the loglinear concentration-time curve.
Measured: Cohort 1: Pre-dose (0 hr), 0.75, 1.5, 2.5, 3.5, 5.5, 7.5, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI, Cohort 2: Cmax of Oxaliplatin
Cmax was observed directly from data. Oxaliplatin was evaluated as total platinum in plasma and platinum in plasma ultrafiltrate.
Measured: Cohort 2: Pre-dose (0 hr), 1, 2, 3, 4, 6, 8, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI, Cohort 2: AUClast of Oxaliplatin
AUClast was defined as area under the plasma concentration time curve from time zero to the last measurable concentration. AUClast was calculated using linear/log trapezoidal method. Oxaliplatin was evaluated as total platinum in plasma and platinum in plasma ultrafiltrate.
Measured: Cohort 2: Pre-dose (0 hr), 1, 2, 3, 4, 6, 8, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI, Cohort 2: CL/F of Oxaliplatin
Clearance of a drug is a measure of the rate at which a drug is metabolized or eliminated by normal biological processes. CL/F was calculated as dose administered divided by area under the concentration curve from time 0 extrapolated to infinite time (AUCinf). AUCinf was calculated as AUClast + (Clast*/kel), where Clast is the predicted plasma concentration at the last quantifiable time point from the log-linear regression analysis, and kel is the terminal phase rate constant calculated by a linear regression of the loglinear concentration-time curve. Oxaliplatin consisted of platinum of plasma and platinum in plasma-ultrafiltrate. The clearance of platinum-ultrafiltrate has been presented in this outcome measure.
Measured: Cohort 2: Pre-dose (0 hr), 1, 2, 3, 4, 6, 8, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI, Cohort 1: Ratio of AUCinf on Cycle 1 Day 15 as Compared to Cycle 1 Day 1 for Irinotecan and Its Metabolite SN-38
AUCinf was calculated as AUClast + (Clast*/kel), where Clast was the predicted plasma concentration at the last quantifiable time point from the log-linear regression analysis, and kel was the terminal phase rate constant calculated by a linear regression of the loglinear concentration-time curve. The ratio between geometric least square (LS) mean (within Cohort 1) for AUCinf on Cycle 1 Day 15 and geometric LS mean (within Cohort 1) for AUCinf on Cycle 1 Day 1 for irinotecan and its metabolite SN-38 has been presented in this outcome measure, which explains the data reported for this outcome measure using measure type as "Number".
Measured: Cohort 1: Pre-dose (0 hr), 0.75, 1.5, 2.5, 3.5, 5.5, 7.5, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI, Cohort 1: Ratio of AUClast on Cycle 1 Day 15 as Compared to Cycle 1 Day 1 for Irinotecan and Its Metabolite SN-38
AUClast was defined as area under the plasma concentration time curve from time zero to the last measurable concentration. AUClast was calculated using linear/log trapezoidal method. The ratio between geometric LS mean (within Cohort 1) for AUClast on Cycle 1 Day 15 and geometric LS mean (within Cohort 1) for AUClast on Cycle 1 Day 1 for irinotecan and its metabolite SN-38 has been presented in this outcome measure, which explains the data reported for this outcome measure using measure type as "Number".
Measured: Cohort 1: Pre-dose (0 hr), 0.75, 1.5, 2.5, 3.5, 5.5, 7.5, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI, Cohort 1: Ratio of Cmax on Cycle 1 Day 15 as Compared to Cycle 1 Day 1 for Irinotecan and Its Metabolite SN-38
Cmax was observed directly from data. The ratio between geometric LS mean (within Cohort 1) for Cmax on Cycle 1 Day 15 and geometric LS mean (within Cohort 1) for Cmax on Cycle 1 Day 1 for irinotecan and its metabolite SN-38 has been presented in this outcome measure, which explains the data reported for this outcome measure using measure type as "Number".
Measured: Cohort 1: Pre-dose (0 hr), 0.75, 1.5, 2.5, 3.5, 5.5, 7.5, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI, Cohort 2: Ratio of AUClast on Cycle 1 Day 15 as Compared to Cycle 1 Day 1 for Oxaliplatin
AUClast was defined as area under the plasma concentration time curve from time zero to the last measurable concentration. AUClast was calculated using linear/log trapezoidal method. Oxaliplatin was evaluated as total platinum in plasma and platinum in plasma ultrafiltrate. The ratio between geometric LS mean (within Cohort 2) for AUClast on Cycle 1 Day 15 and geometric LS mean (within Cohort 2) for AUClast on Cycle 1 Day 1 for Oxaliplatin has been presented in this outcome measure, which explains the data reported for this outcome measure using measure type as "Number".
Measured: Cohort 2: Pre-dose (0 hr), 1, 2, 3, 4, 6, 8, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI, Cohort 2: Ratio of Cmax on Cycle 1 Day 15 as Compared to Cycle 1 Day 1 for Oxaliplatin
Cmax was observed directly from data. Oxaliplatin was evaluated as total platinum in plasma and platinum in plasma ultrafiltrate. The ratio between geometric LS mean (within Cohort 2) for Cmax on Cycle 1 Day 15 and geometric LS mean (within Cohort 2) for Cmax on Cycle 1 Day 1 for Oxaliplatin has been presented in this outcome measure, which explains the data reported for this outcome measure using measure type as "Number".
Measured: Cohort 2: Pre-dose (0 hr), 1, 2, 3, 4, 6, 8, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
Phase 3: OS for Arm B vs Arm C - FAS
OS was defined as the time from the date of first dose to death due to any cause. If a participant was not known to have died at the time of the cutoff for analysis, then OS was censored at the date of last contact. Analysis was performed using Kaplan Meier method.
Measured: From date of first dose to death due to any cause or censoring date, whichever occurred first (maximum up to 37.25 months)
Phase 3: ORR by Derived Investigator Assessment for Arm B vs Arm C - FAS ORR Subset
ORR was defined as the percentage of participants who achieved BOR of confirmed CR or PR according to RECIST v 1.1 by derived investigator assessment. CR: complete disappearance of all target lesions (with the exception of nodal disease) and non-target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm. PR: >=30% decrease under baseline of the sum of diameters of all target measurable lesions. PD: 20% increase in sum of diameters of target measurable lesions above smallest sum observed, with minimum absolute increase of 5 mm for target disease or unequivocal progression of pre-existing lesions for non-target disease or any new lesion.
Measured: From date of randomization until documented PD, or start of subsequent anticancer therapy, or death, whichever occurred first (maximum up to 24.71 months)
Phase 3: ORR as Assessed by BICR - FAS
ORR was defined as the percentage of participants who achieved BOR of confirmed CR or PR according to RECIST v 1.1 as assessed by BICR. CR: complete disappearance of all target lesions (with the exception of nodal disease) and non-target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm. PR: >=30% decrease under baseline of the sum of diameters of all target measurable lesions. PD: 20% increase in sum of diameters of target measurable lesions above smallest sum observed, with minimum absolute increase of 5 mm for target disease or unequivocal progression of pre-existing lesions for non-target disease or any new lesion.
Measured: From date of randomization until documented PD by BICR, or start of subsequent anticancer therapy, or death, whichever occurred first (maximum up to 37.25 months)
Phase 3: DOR as Assessed by BICR for Arm B Versus Arm C - FAS ORR Subset
DOR was defined as the time from the date of first radiographic evidence of response (CR or PR) to the earliest documented PD per RECIST v1.1 as assessed by BICR, or death due to any cause. CR: disappearance of all target lesions and non-target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm. PR: >= 30% decrease under baseline of the sum of diameters of all target measurable lesions. PD: 20% increase in sum of diameters of target measurable lesions above smallest sum observed, with minimum absolute increase of 5 mm for target disease or unequivocal progression of pre-existing lesions for non-target disease or any new lesion. Analysis was performed using Kaplan Meier method.
Measured: From date of first radiographic evidence of response (CR or PR) to earliest documented PD or death due to any cause, whichever occurred first (maximum up to 24.71 months)
Phase 3: DOR by Derived Investigator Assessment for Arm B Versus Arm C - FAS ORR Subset
DOR was defined as the time from the date of first radiographic evidence of response (CR or PR) to the earliest documented PD per RECIST v1.1 by derived investigator assessment, or death due to any cause. CR: disappearance of all target lesions and non-target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm. PR: >= 30% decrease under baseline of the sum of diameters of all target measurable lesions. PD: 20% increase in sum of diameters of target measurable lesions above smallest sum observed, with minimum absolute increase of 5 mm for target disease or unequivocal progression of pre-existing lesions for non-target disease or any new lesion. Analysis was performed using Kaplan Meier method.
Measured: From date of first radiographic evidence of response (CR or PR) to earliest documented PD or death due to any cause, whichever occurred first (maximum up to 24.71 months)
Phase 3: PFS as Assessed by BICR - FAS
PFS was defined as the time from date of randomization to earliest documented PD per RECIST version 1.1 or death due to any cause as assessed by BICR. PD: 20% increase in sum of diameters of target measurable lesions above smallest sum observed, with minimum absolute increase of 5 mm for target disease or unequivocal progression of pre-existing lesions for non-target disease or any new lesion. Censoring details: no adequate baseline assessment, including no disease at baseline: participants were censored to randomization date; no PD, lost to follow-up, withdrawal of consent, PD or death >12 (or 16) weeks after the last adequate tumor assessment, and start of new anticancer treatment: participants were censored to last adequate tumor assessment documenting no PD prior to new anticancer therapy, or missed assessments. Analysis was performed using Kaplan Meier method.
Measured: From date of randomization to earliest documentation of PD or death or censoring date, whichever occurred first (maximum up to 37.25 months)
Phase 3: OS - FAS
OS was defined as the time from the date of first dose to death due to any cause. If a participant was not known to have died at the time of the cutoff for analysis, then OS was censored at the date of last contact. Analysis was performed using Kaplan Meier method.
Measured: From date of first dose to death due to any cause or censoring date, whichever occurred first (maximum up to 37.25 months)
Phase 3: PFS by Derived Investigator Assessment - FAS
PFS was defined as the time from date of randomization to earliest documented PD per RECIST version 1.1 or death due to any cause by derived investigator assessment. PD: 20% increase in sum of diameters of target measurable lesions above smallest sum observed, with minimum absolute increase of 5 mm for target disease or unequivocal progression of pre-existing lesions for non-target disease or any new lesion. Censoring details: no adequate baseline assessment, including no disease at baseline: participants were censored to randomization date; no PD, lost to follow-up, withdrawal of consent, PD or death >12 (or 16) weeks after the last adequate tumor assessment, and start of new anticancer treatment: participants were censored to last adequate tumor assessment documenting no PD prior to new anticancer therapy, or missed assessments. Analysis was performed using Kaplan Meier method.
Measured: From date of first dose to death due to any cause or censoring date, whichever occurred first (maximum up to 37.25 months)
Phase 3: TTR as Assessed by BICR for Arm B vs Arm C - FAS ORR Subset
TTR was defined as the time from the date of first dose to first radiographic evidence of response (CR or PR) per RECIST v1.1 as assessed by BICR. CR: disappearance of all target lesions and non-target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm. PR: >= 30% decrease under baseline of the sum of diameters of all target measurable lesions.
Measured: From date of first dose to CR or PR (maximum up to 24.71 months [107.37 weeks])
Phase 3: TTR by Derived Investigator Assessment for Arm B vs Arm C - FAS ORR Subset
TTR was defined as the time from the date of first dose to first radiographic evidence of response (CR or PR) per RECIST v1.1 by derived investigator assessment. CR: disappearance of all target lesions and non-target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm. PR: >= 30% decrease under baseline of the sum of diameters of all target measurable lesions.
Measured: From date of first dose to CR or PR (maximum up to 24.71 months [107.37 weeks])
Phase 3: Progression After Next Line of Treatment (PFS2) - FAS
PFS2 was defined as the time from the date of randomization to the date of discontinuation of next-line treatment after first objective PD by investigator assessment, to second objective disease progression (PD2), or death from any cause, whichever occurred first. PD2: was progressive disease after the start of subsequent anticancer therapy based on investigator assessment. PFS2 was censored at start date of next-line anticancer treatment (NTX) if PD date > NTX start date and there was no death, at last contact date if withdrawal of consent date >= date of randomization or end of study or if participant lost to follow-up or if no prior conditions are met or PD and no NTX and there was no death.
Measured: From date of randomization to date of discontinuation of next-line treatment after PD or PD2 or death or censoring date, whichever occurred first (maximum up to 37.25 months)
Phase 3: Number of Participants With AEs
An AE was any untoward medical occurrence in clinical study participant temporally associated with use of study intervention, whether/ not considered related to study intervention. SAE was defined as any untoward medical occurrence that, at any dose, met one or more of the following criteria - resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, or other important medical event. AEs included both SAEs and all non-SAEs.
Measured: Through end of the study
Phase 3: Number of Participants With Grade 3 or 4 AEs and Grade 5 AEs
An AE was any untoward medical occurrence in clinical study participant temporally associated with use of study intervention, whether/ not considered related to study intervention. AEs were graded according to NCI-CTCAE v4.03 where grade 1= mild, grade 2= moderate, grade 3= severe, grade 4= life-threatening and grade 5= death related to AE. In this outcome measure, number of participants with grade 3 or 4 AEs and grade 5 AEs were reported.
Measured: Through end of the study
Phase 3: Number of Participants With Shift From Grade <=2 at Baseline to Grade >=3 Post-Baseline in Hematology and Coagulation Parameters
The following hematology and coagulation parameters were assessed: activated partial thromboplastin time prolonged, anemia, hemoglobin increased, INR increased, leukocytosis, neutrophil count decreased, platelet count decreased, and white blood cell decreased. Laboratory abnormalities were graded according to NCI CTCAE v4.03 where grade 1= mild, grade 2= moderate, grade 3= severe, grade 4= life-threatening event due to AE, and grade 5= death. Only those hematology and coagulation parameters in which at least 1 participant in any of the reporting arm had any shift from grade <=2 at baseline to grade >=3 post-baseline are reported in this outcome measure.
Measured: Through end of the study
Phase 3: Number of Participants With Shift From Grade <=2 at Baseline to Grade >=3 Post-Baseline in Chemistry Parameters
The following chemistry parameters were assessed: alanine aminotransferase increased, alkaline phosphatase increased, aspartate aminotransferase increased, blood bilirubin increased, creatinine increased, hypercalcemia, hyperglycemia, hyperkalemia, hypermagnesemia, hypernatremia, hypoalbuminemia, hypocalcemia, hypoglycemia, hypokalemia, hypomagnesemia, hyponatremia, and lipase increased. Laboratory abnormalities were graded according to NCI CTCAE v4.03 where grade 1= mild, grade 2= moderate, grade 3= severe, grade 4= life-threatening and grade 5= death related to AE. Only those chemistry parameters in which at least 1 participant in any of the reporting arm had any shift from grade <=2 at baseline to grade >=3 post-baseline are reported in this outcome measure.
Measured: Through end of the study
Phase 3: Number of Participants According to Categorization of Vital Signs Data
The criteria for vital signs included: Systolic blood pressure (mmHg): value <= 90 mmHg and decrease from baseline >= 20 mmHg, and value >= 160 mmHg and increase from baseline >= 20 mmHg. Diastolic blood pressure (mmHg): value <= 50 mmHg and decrease from baseline >= 15 mmHg, and value >= 100 mmHg and increase from baseline >= 15 mmHg. Pulse rate (bpm): value <= 50 bpm and decrease from baseline >= 15 bpm, and value >= 120 bpm and increase from baseline >= 15 bpm. Weight (kg): change >= 20 % decrease from baseline, and change >=10% increase from baseline. Temperature (degree Celsius): value <=36 degree Celsius, and value >=37.5 degree Celsius. Only those criteria in which at least 1 participant in any of the reporting arm had any vital signs data are reported in this outcome measure.
Measured: Through end of the study
Phase 3: Number of Participants According to Categorization of ECGs Findings
ECG criteria included: ECG mean heart rate (bpm): increase from baseline >25% and to a value >100 bpm; decrease from baseline >25% and to a value <50 bpm, PR interval not otherwise specified (msec): new >280 msec, QRS interval not otherwise specified (msec): new >120 msec, QTcF not otherwise specified: new >450 msec; new >480 msec; new >500 msec; increase from baseline >30 msec and increase from baseline >60 msec. Only those criteria in which at least 1 participant in any of the reporting arm had any ECG findings are reported in this outcome measure.
Measured: Through end of the study
Phase 3: European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Cancer Patients - 30 Item Core Questionnaire (EORTC QLQC30) Global Health Status/Quality of Life Scores (QoL) at Baseline and Week 72
EORTC QLQ-C30 contains 30 items and is composed of both multi-item scales and single-item measures. These included five functional scales (physical, role, emotional, cognitive and social functioning), three symptom scales (fatigue, nausea/vomiting, and pain), six single items (dyspnea, insomnia, appetite loss, constipation, diarrhea and financial impact) and a global health status/QoL scale. Global health status/QoL scale ranged from 0 to 100; the higher score represents better level of functioning. In this outcome measure, global health status/QoL scores are presented.
Measured: Baseline and Week 72
Phase 3: EuroQol Quality of Life 5-Dimension 5-Level Scale (EQ-5D-5L) Visual Analogue Score (VAS) at Baseline and Week 72
EQ-5D-5L is a standardized measure of health status. The EQ-5D-5L consisted of EQ-5D-5L descriptive system and the EQ VAS. For EQ VAS participant rated their overall health status from 0 (worst imaginable) to 100 (best imaginable), higher scores indicating a better health state.
Measured: Baseline and Week 72
Phase 3: Number of Participants According to Response to Patient Global Impression of Severity (PGIS) Assessment at Baseline and at Week 30
PGIS is a single-item scale where participants rated the severity of colorectal cancer as follows: none, mild, moderate, severe and very severe.
Measured: Baseline and Week 30
Phase 3: Number of Participants According to Response to Patient Global Impression of Change (PGIC) Assessment at Week 30
The PGIC is a single-item scale where participants rated the overall change in colorectal cancer since participant started taking the study medication as follows: much better, a little better, no change, a little worse, and much worse.
Measured: Week 30
Phase 3: Trough Plasma Concentration of Encorafenib and Its Metabolite LHY746
Trough plasma concentration of encorafenib and its metabolite LHY746 was measured in this outcome measure.
Measured: Predose on Cycle 1 Day 1, Cycle 2 Day 1, Cycle 3 Day 1, Cycle 4 Day 1, Cycle 5 Day 1 and Cycle 6 Day 1
Phase 3: Cmax of Encorafenib and Its Metabolite LHY746 in Mainland China Participants
Cmax was observed directly from data. LHY746 is a metabolite of Encorafenib.
Measured: Pre-dose (0 hr), 0.5, 1, 2, 3, 4, 5, 6, 8 and 24 hrs post-dose on Days 1 and 15 of Cycle 1
Phase 3: AUC6 of Encorafenib and Its Metabolite LHY746 in Mainland China Participants
The AUC was estimated from time 0 to 6 hours post dose. AUC6 was computed using the Linear/Log trapezoidal method. LHY746 is a metabolite of Encorafenib.
Measured: Pre-dose (0 hr), 0.5, 1, 2, 3, 4, 5 and 6 hrs post-dose on Days 1 and 15 of Cycle 1
Phase 3: AUCtau of Encorafenib and Its Metabolite LHY746 in Mainland China Participants
AUCtau was area under the concentration-time profile from time zero to time tau, the dosing interval. tau = 24 hrs for QD dosing of encorafenib. LHY746 is a metabolite of encorafenib.
Measured: Pre-dose (0 hr), 0.5, 1, 2, 3, 4, 5, 6, 8 and 24 hrs post-dose on Days 1 and 15 of Cycle 1
Phase 3: Tmax of Encorafenib and Its Metabolite LHY746 in Mainland China Participants
Tmax: time (hours) to Cmax. LHY746 is a metabolite of Encorafenib.
Measured: Pre-dose (0 hr), 0.5, 1, 2, 3, 4, 5, 6, 8 and 24 hrs post-dose on Days 1 and 15 of Cycle 1
Phase 3: CL/F of Encorafenib in Mainland China Participants
Clearance of a drug is a measure of the rate at which a drug is metabolized or eliminated by normal biological processes. CL/F was calculated as dose administered divided by area under the concentration curve from time 0 extrapolated to infinite time (AUCinf). AUCinf was calculated as AUClast + (Clast*/kel), where Clast is the predicted plasma concentration at the last quantifiable time point from the log-linear regression analysis, and kel is the terminal phase rate constant calculated by a linear regression of the loglinear concentration-time curve.
Measured: Pre-dose (0 hr), 0.5, 1, 2, 3, 4, 5, 6, 8 and 24 hrs post-dose on Days 1 and 15 of Cycle 1
Phase 3: Number of Participants Classified According to Microsatellite Instability (MSI) Status as Determined by Retrospective Central Testing of Baseline Tumor Tissue
MSI status was classified as follows; microsatellite instability-high (MSI-H): included participants with no negative test results and at least one positive test result, microsatellite stable (MSS): included participants with at least one negative test result and MSI-unknown: included participants with intermediate test results or not analyzed for MSI. The number of participants as per their MSI status (MSI-H, MSS, and MSI-unknown) have been reported in this outcome measure.
Measured: Baseline
Phase 3: Number of Participants According to Circulating Tumor Deoxyribonucleic Acid (ctDNA) Status
ctDNA status was classified as follows; detected: overall variant allele frequency (VAF) was greater than zero, not detected: overall VAF equalled zero, and not evaluable: overall VAF could not be calculated. The VAF was defined as percentage of deoxyribonucleic acid (DNA) reads at a specific position that show a genetic variant instead of the normal (reference) sequence. The number of participants as per their ctDNA status (detected, not detected and not evaluable) have been reported in this outcome measure.
Measured: Cycle 1 Day 1, Cycle 1 Day 15, Cycle 2 Day 15, Cycle 7 Day 1 and End of Treatment (anytime till maximum of 37.25 months)
Phase 3: Number of Participants According to B-Raf Serine/Threonine-Protein Kinase (BRAF) Valine 600 (V600) Status From ctDNA
BRAF V600 status from ctDNA was classified as follows; measurable: participants with detectable ctDNA had measurable BRAF V600 tumor biomarker alterations and not measurable: participants with detectable ctDNA did not have measurable BRAF V600 biomarker alterations or overall ctDNA was not detectable. The number of participants as per their BRAF V600 status from ctDNA (measurable and not measurable) have been reported in this outcome measure. In this outcome measure, data is presented for participants outside China and Mainland China participants.
Measured: Cycle 1 Day 1, Cycle 1 Day 15, Cycle 2 Day 15, Cycle 7 Day 1 and End of Treatment (anytime till maximum of 37.25 months)
Cohort 3: PFS as Assessed by BICR - FAS
PFS was defined as the time from date of randomization to earliest documented PD per RECIST version 1.1 or death due to any cause as assessed by BICR. PD: 20% increase in sum of diameters of target measurable lesions above smallest sum observed, with minimum absolute increase of 5 mm for target disease or unequivocal progression of pre-existing lesions for non-target disease or any new lesion. Censoring details: no adequate baseline assessment, including no disease at baseline: participants were censored to randomization date; no PD, lost to follow-up, withdrawal of consent, PD or death >12 (or 16) weeks after the last adequate tumor assessment, and start of new anticancer treatment: participants were censored to last adequate tumor assessment documenting no PD prior to new anticancer therapy, or missed assessments. Analysis was performed using Kaplan Meier method.
Measured: From date of randomization to earliest documentation of PD by BICR or death or censoring date, whichever occurred first
Cohort 3: ORR by Derived Investigator Assessment - FAS
ORR was defined as the percentage of participants who achieved BOR of confirmed CR or PR according to RECIST v 1.1 by derived investigator assessment. CR: complete disappearance of all target lesions (with the exception of nodal disease) and non-target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm. PR: >=30% decrease under baseline of the sum of diameters of all target measurable lesions. PD: 20% increase in sum of diameters of target measurable lesions above smallest sum observed, with minimum absolute increase of 5 mm for target disease or unequivocal progression of pre-existing lesions for non-target disease or any new lesion.
Measured: From date of randomization until documented PD, or start of subsequent anticancer therapy, or death, whichever occurred first (maximum up to 14.1 months)
Cohort 3: DOR as Assessed by BICR - FAS
DOR was defined as the time from the date of first radiographic evidence of response (CR or PR) to the earliest documented PD per RECIST v1.1 as assessed by BICR, or death due to any cause. CR: disappearance of all target lesions and non-target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm. PR: >= 30% decrease under baseline of the sum of diameters of all target measurable lesions. PD: 20% increase in sum of diameters of target measurable lesions above smallest sum observed, with minimum absolute increase of 5 mm for target disease or unequivocal progression of pre-existing lesions for non-target disease or any new lesion. Analysis was performed using Kaplan Meier method.
Measured: From date of first radiographic evidence of response (CR or PR) to earliest documented PD or death due to any cause, whichever occurred first (maximum up to 14.1 months)
Cohort 3: DOR by Derived Investigator Assessment - FAS
DOR was defined as the time from the date of first radiographic evidence of response (CR or PR) to the earliest documented PD per RECIST v1.1 by derived investigator assessment, or death due to any cause. CR: disappearance of all target lesions and non-target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm. PR: >= 30% decrease under baseline of the sum of diameters of all target measurable lesions. PD: 20% increase in sum of diameters of target measurable lesions above smallest sum observed, with minimum absolute increase of 5 mm for target disease or unequivocal progression of pre-existing lesions for non-target disease or any new lesion. Analysis was performed using Kaplan Meier method.
Measured: From date of first radiographic evidence of response (CR or PR) to earliest documented PD or death due to any cause, whichever occurred first (maximum up to 14.1 months)
Cohort 3: PFS by Derived Investigator Assessment - FAS
PFS was defined as the time from date of randomization to earliest documented PD per RECIST version 1.1 or death due to any cause by derived investigator assessment. PD: 20% increase in sum of diameters of target measurable lesions above smallest sum observed, with minimum absolute increase of 5 mm for target disease or unequivocal progression of pre-existing lesions for non-target disease or any new lesion. Censoring details: no adequate baseline assessment, including no disease at baseline: participants were censored to randomization date; no PD, lost to follow-up, withdrawal of consent, PD or death >12 (or 16) weeks after the last adequate tumor assessment, and start of new anticancer treatment: participants were censored to last adequate tumor assessment documenting no PD prior to new anticancer therapy, or missed assessments. Analysis was performed using Kaplan Meier method.
Measured: From date of randomization to earliest documentation of PD or death or censoring date, whichever occurred first
Cohort 3: OS - FAS
OS was defined as the time from the date of first dose to death due to any cause. If a participant was not known to have died at the time of the cutoff for analysis, then OS was censored at the date of last contact. Analysis was performed using Kaplan Meier method.
Measured: From date of first dose to death due to any cause or censoring date, whichever occurred first
Cohort 3: TTR as Assessed by BICR - FAS
TTR was defined as the time from the date of first dose to first radiographic evidence of response (CR or PR) per RECIST v1.1 as assessed by BICR. CR: disappearance of all target lesions and non-target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm. PR: >= 30% decrease under baseline of the sum of diameters of all target measurable lesions.
Measured: From date of first dose to CR or PR (maximum up to 14.1 months [61.29 weeks])
Cohort 3: TTR by Derived Investigator Assessment - FAS
TTR was defined as the time from the date of first dose to first radiographic evidence of response (CR or PR) per RECIST v1.1 by derived investigator assessment. CR: disappearance of all target lesions and non-target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm. PR: >= 30% decrease under baseline of the sum of diameters of all target measurable lesions.
Measured: From date of first dose to CR or PR (maximum up to 14.1 months [61.29 weeks])
Cohort 3: Number of Participants With AEs
An AE was any untoward medical occurrence in clinical study participant temporally associated with use of study intervention, whether/ not considered related to study intervention. SAE was defined as any untoward medical occurrence that, at any dose, met one or more of the following criteria - resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, or other important medical event. AEs included both SAEs and all non-SAEs.
Measured: Through end of the study
Cohort 3: Number of Participants With Grade 3 or 4 AEs and Grade 5 AEs
An AE was any untoward medical occurrence in clinical study participant temporally associated with use of study intervention, whether/ not considered related to study intervention. AEs were graded according to NCI-CTCAE v4.03 where grade 1= mild, grade 2= moderate, grade 3= severe, grade 4= life-threatening and grade 5= death related to AE. In this outcome measure, number of participants with grade 3 or 4 AEs and grade 5 AEs were reported.
Measured: Through end of the study
Cohort 3: Number of Participants With Shift From Grade <=2 at Baseline to Grade >=3 Post-Baseline in Hematology and Coagulation Parameters
The following hematology and coagulation parameters were assessed: activated partial thromboplastin time prolonged, anemia, hemoglobin increased, INR increased, leukocytosis, neutrophil count decreased, platelet count decreased, and white blood cell decreased. Laboratory abnormalities were graded according to NCI CTCAE v4.03 where grade 1= mild, grade 2= moderate, grade 3= severe, grade 4= life-threatening event due to AE, and grade 5= death. Only those hematology and coagulation parameters in which at least 1 participant in any of the reporting arm had any shift from grade <=2 at baseline to grade >=3 post-baseline are reported in this outcome measure.
Measured: Through end of the study
Cohort 3: Number of Participants With Shift From Grade <=2 at Baseline to Grade >=3 Post-Baseline in Chemistry Parameters
The following chemistry parameters were assessed: alanine aminotransferase increased, alkaline phosphatase increased, aspartate aminotransferase increased, blood bilirubin increased, creatinine increased, hypercalcemia, hyperglycemia, hyperkalemia, hypermagnesemia, hypernatremia, hypoalbuminemia, hypocalcemia, hypoglycemia, hypokalemia, hypomagnesemia, hyponatremia, and lipase increased. Laboratory abnormalities were graded according to NCI CTCAE v4.03 where grade 1= mild, grade 2= moderate, grade 3= severe, grade 4= life-threatening and grade 5= death related to AE. Only those chemistry parameters in which at least 1 participant in any of the reporting arm had any shift from grade <=2 at baseline to grade >=3 post-baseline are reported in this outcome measure.
Measured: Through end of the study
Cohort 3: Number of Participants According to Categorization of Vital Signs Data
The criteria for vital signs included: Systolic blood pressure (mmHg): value <= 90 mmHg and decrease from baseline >= 20 mmHg, and value >= 160 mmHg and increase from baseline >= 20 mmHg. Diastolic blood pressure (mmHg): value <= 50 mmHg and decrease from baseline >= 15 mmHg, and value >= 100 mmHg and increase from baseline >= 15 mmHg. Pulse rate (bpm): value <= 50 bpm and decrease from baseline >= 15 bpm, and value >= 120 bpm and increase from baseline >= 15 bpm. Weight (kg): change >= 20 % decrease from baseline, and change >=10% increase from baseline. Temperature (degree Celsius): value <=36 degree Celsius, and value >=37.5 degree Celsius. Only those criteria in which at least 1 participant in any of the reporting arm had any vital signs data are reported in this outcome measure.
Measured: Through end of the study
Cohort 3: Number of Participants According to Categorization of ECGs Findings
ECG criteria included: ECG mean heart rate (bpm): increase from baseline >25% and to a value >100 bpm; decrease from baseline >25% and to a value <50 bpm, PR interval not otherwise specified (msec): new >280 msec, QRS interval not otherwise specified (msec): new >120 msec, QTcF not otherwise specified: new >450 msec; new >480 msec; new >500 msec; increase from baseline >30 msec and increase from baseline >60 msec. Only those criteria in which at least 1 participant in any of the reporting arm had any ECG findings are reported in this outcome measure.
Measured: Through end of the study
Cohort 3: EORTC QLQC30 Global Health Status/QoL
EORTC QLQ-C30 contains 30 items and is composed of both multi-item scales and single-item measures. These included five functional scales (physical, role, emotional, cognitive and social functioning), three symptom scales (fatigue, nausea/vomiting, and pain), six single items (dyspnea, insomnia, appetite loss, constipation, diarrhea and financial impact) and a global health status/QoL scale. Global health status/QoL scale ranged from 0 to 100; the higher score represents better level of functioning.
Measured: Through end of the study
Cohort 3: EQ-5D-5L VAS
EQ-5D-5L is a standardized measure of health status. The EQ-5D-5L consisted of EQ-5D-5L descriptive system and the EQ VAS. For EQ VAS participant rated their overall health status from 0 (worst imaginable) to 100 (best imaginable), higher scores indicating a better health state.
Measured: Through end of the study
Cohort 3: Number of Participants According to Response to PGIS Assessment
PGIS is a single-item scale where participants rated the severity of colorectal cancer as follows: none, mild, moderate, severe and very severe.
Measured: Through end of the study
Cohort 3: Number of Participants According to Response to PGIC Assessment
The PGIC is a single-item scale where participants rated the overall change in colorectal cancer since participant started taking the study medication as follows: much better, a little better, no change, a little worse, and much worse.
Measured: Through end of the study
Cohort 3: Trough Plasma Concentration of Encorafenib and Its Metabolite LHY746
Trough plasma concentration of encorafenib and its metabolite LHY746 was measured in this outcome measure.
Measured: Predose on Cycle 1 Day 1, Cycle 2 Day 1, Cycle 3 Day 1, Cycle 4 Day 1, Cycle 5 Day 1 and Cycle 6 Day 1
Cohort 3: Number of Participants Classified According to MSI Status as Determined by Retrospective Central Testing
MSI status was classified as follows; MSI-H: included participants with no negative test results and at least one positive test result, MSS: included participants with at least one negative test result and MSI-unknown: included participants with intermediate test results or not analyzed for MSI. The number of participants as per their MSI status (MSI-H, MSS, and MSI-unknown) have been reported in this outcome measure.
Measured: Through end of the study
Cohort 3: Number of Participants According to ctDNA Status
ctDNA status was classified as follows; detected: overall variant allele frequency (VAF) was greater than zero, not detected: overall VAF equalled zero, and not evaluable: overall VAF could not be calculated. The VAF was defined as percentage of deoxyribonucleic acid (DNA) reads at a specific position that show a genetic variant instead of the normal (reference) sequence. The number of participants as per their ctDNA status (detected, not detected and not evaluable) have been reported in this outcome measure.
Measured: Through end of the study
Cohort 3: Number of Participants According to BRAF V600 Status From ctDNA
BRAF V600 status from ctDNA was classified as follows; measurable: participants with detectable ctDNA had measurable BRAF V600 tumor biomarker alterations and not measurable: participants with detectable ctDNA did not have measurable BRAF V600 biomarker alterations or overall ctDNA was not detectable. The number of participants as per their BRAF V600 status from ctDNA (measurable and not measurable) have been reported in this outcome measure.
Measured: Through end of the study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Oct 2020
- Study start21 Dec 2020
- Primary completion1 Mar 2025
- Study completion29 Jul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for NeoplasmsStudies of the same condition, open and under way.
- More trials run by PfizerThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 841 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Ono Pharmaceutical Co., Ltd., Merck KGaA, Darmstadt, Germany, Eli Lilly and Company
- Responsible party
- Sponsor
- Investigators
- Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04607421
- Sponsor's protocol ID
- C4221015
- Other IDs
- 2023-509405-77-00, BREAKWATER, 2023
- Status last verified
- Oct 2026
- First submitted
- 5 Oct 2020
- Last updated
- 5 Nov 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.
Publications
Kopetz S, Tabernero J, Elez E. BREAKWATER Phase III: results for encorafenib and cetuximab plus mFOLFOX6 in first-line BRAF V600E-mutant metastatic colorectal cancer. Future Oncol. 2025 Dec;21(28):3585-3588. doi: 10.1080/14796694.2025.2579882. Epub 2025 Nov 13.
PubMed 41230651Elez E, Yoshino T, Shen L, Lonardi S, Van Cutsem E, Eng C, Kim TW, Wasan HS, Desai J, Ciardiello F, Yaeger R, Maughan TS, Morris VK, Wu C, Usari T, Laliberte R, Dychter SS, Zhang X, Tabernero J, Kopetz S; BREAKWATER Trial Investigators. Encorafenib, Cetuximab, and mFOLFOX6 in BRAF-Mutated Colorectal Cancer. N Engl J Med. 2025 Jun 26;392(24):2425-2437. doi: 10.1056/NEJMoa2501912. Epub 2025 May 30.
PubMed 40444708Kopetz S, Yoshino T, Van Cutsem E, Eng C, Kim TW, Wasan HS, Desai J, Ciardiello F, Yaeger R, Maughan TS, Beyzarov E, Zhang X, Ferrier G, Zhang X, Tabernero J. Encorafenib, cetuximab and chemotherapy in BRAF-mutant colorectal cancer: a randomized phase 3 trial. Nat Med. 2025 Mar;31(3):901-908. doi: 10.1038/s41591-024-03443-3. Epub 2025 Jan 25.
PubMed 39863775Van Cutsem E, Taieb J, Yaeger R, Yoshino T, Grothey A, Maiello E, Elez E, Dekervel J, Ross P, Ruiz-Casado A, Graham J, Kato T, Ruffinelli JC, Andre T, Carriere Roussel E, Klauck I, Groc M, Vedovato JC, Tabernero J. ANCHOR CRC: Results From a Single-Arm, Phase II Study of Encorafenib Plus Binimetinib and Cetuximab in Previously Untreated BRAFV600E-Mutant Metastatic Colorectal Cancer. J Clin Oncol. 2023 May 10;41(14):2628-2637. doi: 10.1200/JCO.22.01693. Epub 2023 Feb 10.
PubMed 36763936
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- AN OPEN-LABEL, MULTICENTER, RANDOMIZED PHASE 3 STUDY OF FIRST-LINE ENCORAFENIB PLUS CETUXIMAB WITH OR WITHOUT CHEMOTHERAPY VERSUS STANDARD OF CARE THERAPY WITH A SAFETY LEAD-IN OF ENCORAFENIB AND CETUXIMAB PLUS CHEMOTHERAPY IN PARTICIPANTS WITH METASTATIC BRAF V600E-MUTANT COLORECTAL CANCER
- ClinicalTrials.gov ID
- NCT04607421
- Acronym / sponsor ID
- C4221015
- Conditions
- Neoplasms
- Keywords
- Colorectal cancer, Colon cancer, Rectal cancer, Colorectal Neoplasms, Intestinal cancer, Intestinal Neoplasms, Gastrointestinal cancer, Gastrointestinal Neoplasms
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 841 participants (actual)
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- Collaborators
- Ono Pharmaceutical Co., Ltd., Merck KGaA, Darmstadt, Germany, Eli Lilly and Company
- Study start
- 21 Dec 2020 (actual)
- Primary completion
- 1 Mar 2025 (actual)
- Study completion
- 29 Jul 2027 (estimated)
- First posted
- 29 Oct 2020 (actual)
- Results
- Posted 11 Jun 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate two study medicines (encorafenib plus cetuximab) taken alone or together with standard chemotherapy for the potential treatment of colorectal cancer that:
- has spread to other parts of the body (metastatic);
- has a certain type of abnormal gene called "BRAF"; and
- has not received prior treatment.
Participants in this study will receive one of the following study treatments:
- Encorafenib plus cetuximab: These participants will receive encorafenib by mouth at home every day and cetuximab once every two weeks by intravenous (IV) infusion (an injection into the vein) at the study clinic.
- Encorafenib plus cetuximab with chemotherapy: These participants will receive encorafenib and cetuximab in the way described in the bullet above. Additionally, they will receive standard chemotherapy by IV infusion and oral treatment at home.
- Chemotherapy alone: These participants will receive chemotherapy, the standard treatment for this condition, by IV infusion at the study clinics and oral treatment at home.
This study is currently enrolling participants who will receive either encorafenib plus cetuximab with chemotherapy or chemotherapy alone.
The study team will monitor how each participant responds to the study treatment for up to about 3 years.
Detailed description
The purpose of the study is to evaluate whether encorafenib plus cetuximab (EC), alone or in combination with chemotherapy, can improve clinical outcomes relative to current standard of-care chemotherapy in participants with previously untreated BRAF V600E-mutant mCRC. Since encorafenib has not previously been combined with chemotherapy, the tolerability and PK of EC in combination with mFOLFOX6 and in combination with FOLFIRI will be evaluated in separate cohorts in the safety lead-in portion of the trial in order to identify which chemotherapy combination is to be used in the Phase 3 portion of the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 7
| Arm | Type | Receives |
|---|---|---|
| Safety Lead-in Cohort 1 | Experimental | Drug: Encorafenib, Drug: Cetuximab, Drug: Irinotecan, Drug: Leucovorin, Drug: 5-FUEncorafenib 300 mg orally once daily Cetuximab 500 mg/m2 (120-minute IV infusion) every two weeks Irinotecan 180 mg/m2 (90-minute IV infusion) every two weeks Leucovorin 400 mg/m2 (120-minute IV infusion) every two weeks 5-FU 400 mg/m2 IV bolus, then 5-FU 2400 mg/m2 continuous IV infusion over 46-48 hours every two weeks |
| Safety Lead-in Cohort 2 | Experimental | Drug: Encorafenib, Drug: Cetuximab, Drug: Oxaliplatin, Drug: Leucovorin, Drug: 5-FUEncorafenib 300 mg orally once daily Cetuximab 500 mg/m2 (120 minute IV infusion) every two weeks Oxaliplatin 85 mg/m2 (120-minute IV infusion) every two weeks Leucovorin 400 mg/m2 (120-minute IV infusion) every two weeks 5-FU 400 mg/m2 IV bolus, then 5-FU 2400 mg/m2 continuous IV infusion over 46-48 hours every two weeks |
| Phase 3 Arm A | Experimental | Drug: Encorafenib, Drug: CetuximabEncorafenib 300 mg orally once daily Cetuximab 500 mg/m2 (120-minute IV infusion) every two weeks |
| Phase 3 Arm B | Experimental | Drug: Encorafenib, Drug: Cetuximab, Drug: Oxaliplatin, Drug: Leucovorin, Drug: 5-FUEncorafenib 300 mg orally once daily Cetuximab 500 mg/m2 (120 minute IV infusion) every two weeks Oxaliplatin 85 mg/m2 (120-minute IV infusion) every two weeks Leucovorin 400 mg/m2 (120-minute IV infusion) every two weeks 5-FU 400 mg/m2 IV bolus, then 5-FU 2400 mg/m2 continuous IV infusion over 46-48 hours every two weeks |
| Phase 3 Arm C | Active comparator | Drug: Oxaliplatin, Drug: Irinotecan, Drug: Leucovorin, Drug: 5-FU, Drug: Capecitabine, Drug: BevacizumabEvery two weeks: Oxaliplatin 85 mg/m2 (120-minute IV infusion) Leucovorin 400 mg/m2 (120-minute IV infusion) 5-FU 400 mg/m2 IV bolus, then 5-FU 2400 mg/m2 continuous IV infusion over 46-48 hours Bevacizumab (optional; given per prescribing instructions) -OR- Every two weeks: Irinotecan 165 mg/m2 (90-minute IV infusion) Oxaliplatin 85 mg/m2 (120-minute IV infusion) Leucovorin 400 mg/m2 (120-minute IV infusion) 5-FU 2400 or 3200 mg/m2 continuous IV infusion over 46 48 hours Bevacizumab (optional; given per prescribing instructions) -OR- Oxaliplatin 130 mg/m2 (120-minute IV infusion) every 3 weeks Capecitabine 1000 mg/m2 oral tablet twice daily on Days 1-14 Bevacizumab (optional; given per prescribing instructions) |
| Cohort 3 Arm D | Experimental | Drug: Encorafenib, Drug: Cetuximab, Drug: Irinotecan, Drug: Leucovorin, Drug: 5-FUEncorafenib 300 mg orally once daily Cetuximab 500 mg/m2 (120-minute IV infusion) every two weeks Irinotecan 180 mg/m2 (90-minute IV infusion) every two weeks Leucovorin 400 mg/m2 (120-minute IV infusion) every two weeks 5-FU 400 mg/m2 IV bolus, then 5-FU 2400 mg/m2 continuous IV infusion over 46-48 hours every two weeks |
| Cohort 3 Arm E | Active comparator | Drug: Irinotecan, Drug: Leucovorin, Drug: 5-FU, Drug: BevacizumabIrinotecan 180 mg/m2 (90-minute IV infusion) every 2 weeks, Leucovorin 400 mg/m2 (120-minute IV infusion) every 2 weeks, 5-FU 400 mg/m2 IV bolus, then 5-FU 2400 mg/m2 continuous IV infusion over 46-48 hours every two weeks, Bevacizumab (optional; given per prescribing instructions) |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Safety Lead-In = Male/female ≥ 18 years old
- Phase 3 and Cohort 3: Male/female ≥ 16 years old (where permitted locally)
- Histologically or cytologically confirmed Stage IV CRC that contains BRAF V600E mutation
- Prior systemic treatment in metastatic setting: 0-1 regimens for Safety Lead In; none for Phase 3 and Cohort 3. (Note: Prior adjuvant or neoadjuvant therapy considered metastatic treatment if relapse/metastasis < 6 month from end of adj/neoadjuvant treatment )
- Measurable disease (Phase 3 and Cohort 3)/ Measurable or evaluable disease (Safety Lead-in)
Key exclusion criteria (3 listed)
- Tumors that are locally confirmed or unknown MSI-H or dMMR unless participant is ineligible to receive immune checkpoint inhibitors due to a pre-existing medical condition
- Active bacterial or viral infections in 2 weeks prior to starting dosing
- Symptomatic brain metastases
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EncorafenibAlso: Braftovi, PF-07263896, LGX818, ONO-770275 mg capsules | Drug | Cohort 3 Arm D, Phase 3 Arm A, Phase 3 Arm B, Safety Lead-in Cohort 1, Safety Lead-in Cohort 2 |
| CetuximabAlso: ErbituxInjection for intravenous use 100 mg/vial, 200 mg/vial, or 500 mg/vial | Drug | Cohort 3 Arm D, Phase 3 Arm A, Phase 3 Arm B, Safety Lead-in Cohort 1, Safety Lead-in Cohort 2 |
| OxaliplatinAlso: EloxatinPowder for solution for intravenous use 50 mg/vial, 100 mg/vial, or 200 mg/vial | Drug | Phase 3 Arm B, Phase 3 Arm C, Safety Lead-in Cohort 2 |
| IrinotecanAlso: CampostarSolution for intravenous infusion 40 mg/vial, 100 mg/vial, or 300 mg/vial | Drug | Cohort 3 Arm D, Cohort 3 Arm E, Phase 3 Arm C, Safety Lead-in Cohort 1 |
| LeucovorinAlso: Wellcovorin, Fusilev, KhapzoryInjection 50 mg/vial, 100 mg/vial, 200 mg/vial, or 350 mg/vial | Drug | Cohort 3 Arm D, Cohort 3 Arm E, Phase 3 Arm B, Phase 3 Arm C, Safety Lead-in Cohort 1, Safety Lead-in Cohort 2 |
| 5-FUAlso: FluorouracilInjection for intravenous use 250 mg/vial, 500 mg/vial, or 1000 mg/vial | Drug | Cohort 3 Arm D, Cohort 3 Arm E, Phase 3 Arm B, Phase 3 Arm C, Safety Lead-in Cohort 1, Safety Lead-in Cohort 2 |
| CapecitabineAlso: Xeloda150 mg or 500 mg Tablet | Drug | Phase 3 Arm C |
| BevacizumabAlso: ZirabevOptional Injection for intravenous use 100 mg/vial or 400 mg/vial | Drug | Cohort 3 Arm E, Phase 3 Arm C |
Primary outcome
SLI: Number of Participants With Dose Limiting Toxicity (DLTs)
Time frame: Cycle 1 (28 days)
DLT: Any AE/lab value during first 28 days (D) of treatment that met at least 1 criteria: AE/lab value unrelated to underlying disease,PD,intercurrent illness,concomitant medications resulting in inability to tolerate atleast 75% of planned dose intensity of study drug,fatigue grade (G)3>14 consecutive D, interstitial lung disease G>=2,rash,hand foot skin reaction G3>14 consecutive D or G4,diarrhea G3 >=48 hours or G4,nausea/vomiting G3>=48 hours or G4,mucositis G>=3,total bilirubin G>=3, aspartate aminotransferase/alanine aminotransferase G>=3 in conjunction with total bilirubin G>=2 or G3 >7 consecutive D or G4,Serum creatinine G>=3,absolute neutrophil count G4 >7 consecutive D, >=G3 febrile neutropenia,G3 platelet count decreased with signs of bleeding,platelet count G4,ECG QTcF prolonged >=G3,G>=3 uveitis >21 consecutive D,G4 confirmed by ophthalmic examination,paresthesia/dysesthesias G>=3,other G>=3 hematologic/nonhematologic toxicity except lymphocyte count decreased G>=3.
Phase 3: Progression Free Survival (PFS) as Assessed by Blinded Independent Central Review (BICR) for Arm B vs Arm C - FAS
Time frame: From date of randomization to earliest documentation of PD by BICR or death or censoring date, whichever occurred first (maximum up to 37.25 months)
PFS was defined as the time from date of randomization to earliest documented disease progression (PD) per Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 or death due to any cause as assessed by BICR. PD: 20% increase in sum of diameters of target measurable lesions above smallest sum observed, with minimum absolute increase of 5 millimeter (mm) for target disease or unequivocal progression of pre-existing lesions for non-target disease or any new lesion. Censoring details: no adequate baseline assessment, including no disease at baseline: participants were censored to randomization date; no PD, lost to follow-up, withdrawal of consent, PD or death >12 (or 16) weeks after the last adequate tumor assessment, and start of new anticancer treatment: participants were censored to last adequate tumor assessment documenting no PD prior to new anticancer therapy, or missed assessments. Analysis was performed using Kaplan Meier method.
Phase 3: Objective Response Rate (ORR) as Assessed by BICR for Arm B vs Arm C - FAS ORR Subset
Time frame: From date of randomization to until documented PD by BICR, or start of subsequent anticancer therapy or death, whichever occurred first (maximum up to 24.71 months)
ORR was defined as the percentage of participants who achieved best overall response (BOR) of confirmed complete response (CR) or partial response (PR) according to RECIST v 1.1 as assessed by BICR. CR: complete disappearance of all target lesions (with the exception of nodal disease) and non-target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm. PR: >=30% decrease under baseline of the sum of diameters of all target measurable lesions. PD: 20% increase in sum of diameters of target measurable lesions above smallest sum observed, with minimum absolute increase of 5 mm for target disease or unequivocal progression of pre-existing lesions for non-target disease or any new lesion.
Cohort 3: ORR as Assessed by BICR for Arm D vs Arm E - FAS
Time frame: From date of randomization until documented PD by BICR, or start of subsequent anticancer therapy or death, whichever occurred first (maximum up to 14.1 months)
ORR was defined as the percentage of participants who achieved BOR of confirmed CR or PR according to RECIST v 1.1 as assessed by BICR. CR: complete disappearance of all target lesions (with the exception of nodal disease) and non-target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm. PR: >=30% decrease under baseline of the sum of diameters of all target measurable lesions. PD: 20% increase in sum of diameters of target measurable lesions above smallest sum observed, with minimum absolute increase of 5 mm for target disease or unequivocal progression of pre-existing lesions for non-target disease or any new lesion.
82 secondary outcomes
SLI: Number of Participants With Adverse Events (AEs)
Time frame: Through end of the study
SLI: Number of Participants With Grade 3 or 4 AEs and Grade 5 AEs
Time frame: Through end of the study
SLI: Number of Participants With Shift From Grade <=2 at Baseline to Grade >=3 Post-Baseline in Hematology and Coagulation Parameters
Time frame: Through end of the study
SLI: Number of Participants With Shift From Grade <=2 at Baseline to Grade >=3 Post-Baseline in Chemistry Parameters
Time frame: Through end of the study
SLI: Number of Participants According to Categorization of Vital Signs Data
Time frame: Through end of the study
SLI: Number of Participants According to Categorization of Electrocardiogram (ECGs) Findings
Time frame: Through end of the study
SLI: Number of Participants With Dose Modification of Any Study Intervention Due to AEs
Time frame: Through end of the study
SLI: Number of Participants With Dose Discontinuation of Any Study Intervention Due to AEs
Time frame: Through end of the study
SLI: ORR as Assessed by Investigator According to Line of Therapy - FAS
Time frame: From date of randomization until documented PD, or start of subsequent anticancer therapy or death, whichever occurred first (maximum up to 33.9 months)
SLI: Duration of Response (DOR) as Assessed by Investigator According to Line of Therapy - FAS
Time frame: From date of first radiographic evidence of response (CR or PR) to earliest documented PD or death due to any cause whichever occurred first (maximum up to 33.9 months)
SLI: PFS as Assessed by Investigator According to Line of Therapy - FAS
Time frame: From date of randomization to earliest documentation of PD or death or censoring date, whichever occurred first (maximum up to 33.9 months)
SLI: Time to Response (TTR) as Assessed by Investigator According to Line of Therapy - FAS
Time frame: From date of first dose to first radiographic evidence of response (CR or PR) (maximum up to 33.9 months [147.3 weeks])
SLI: Overall Survival (OS) According to Line of Therapy - FAS
Time frame: From date of first dose to death due to any cause or censoring date, whichever occurred first (maximum up to 33.9 months)
SLI: Maximum Plasma Concentration (Cmax) of Encorafenib and Its Metabolite LHY746
Time frame: Cohort 1: Pre-dose (0 hour [hr]), 0.75, 1.5, 2.5, 3.5, 5.5, 7.5, and 48 hrs post-dose on Day 15 of Cycle 1; Cohort 2: Pre-dose (0 hr), 1, 2, 3, 4, 6, 8, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI: Area Under the Concentration Time Profile From Time Zero to 6 Hours (AUC6) for Encorafenib and Its Metabolite LHY746
Time frame: Cohort 1: Pre-dose (0 hr), 0.75, 1.5, 2.5, 3.5, 5.5, and 6 hrs post-dose on Day 15 of Cycle 1; Cohort 2: Predose (0 hr), 1, 2, 3, 4, and 6 hrs post-dose on Days 1 and 15 of Cycle 1
SLI: Area Under the Concentration-time Profile From Time Zero to the Time Tau (AUCtau) of Encorafenib and Its Metabolite LHY746
Time frame: Cohort 1: Pre-dose (0 hr), 0.75, 1.5, 2.5, 3.5, 5.5, 7.5, and 48 hrs post-dose on Day 15 of Cycle 1; Cohort 2: Pre-dose (0 hr), 1, 2, 3, 4, 6, 8, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI: Time to Maximum Plasma Concentration (Tmax) of Encorafenib and Its Metabolite LHY746
Time frame: Cohort 1: Pre-dose (0 hr), 0.75, 1.5, 2.5, 3.5, 5.5, 7.5, and 48 hrs post-dose on Day 15 of Cycle 1; Cohort 2: Pre-dose (0 hr), 1, 2, 3, 4, 6, 8, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI: Apparent Total Clearance (CL/F) of Encorafenib
Time frame: Cohort 1: Pre-dose (0 hr), 0.75, 1.5, 2.5, 3.5, 5.5, 7.5, and 48 hrs post-dose on Day 15 of Cycle 1; Cohort 2: Pre-dose (0 hr), 1, 2, 3, 4, 6, 8, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI, Cohort 1: Cmax of Irinotecan and Its Metabolite SN-38
Time frame: Cohort 1: Pre-dose (0 hr), 0.75, 1.5, 2.5, 3.5, 5.5, 7.5, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI, Cohort 1: AUClast of Irinotecan and Its Metabolite SN-38
Time frame: Cohort 1: Pre-dose (0 hr), 0.75, 1.5, 2.5, 3.5, 5.5, 7.5, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI, Cohort 1: Apparent Terminal Elimination Half-Life (t1/2) of Irinotecan and Its Metabolite SN-38
Time frame: Cohort 1: Pre-dose (0 hr), 0.75, 1.5, 2.5, 3.5, 5.5, 7.5, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI, Cohort 1: CL/F of Irinotecan
Time frame: Cohort 1: Pre-dose (0 hr), 0.75, 1.5, 2.5, 3.5, 5.5, 7.5, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI, Cohort 2: Cmax of Oxaliplatin
Time frame: Cohort 2: Pre-dose (0 hr), 1, 2, 3, 4, 6, 8, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI, Cohort 2: AUClast of Oxaliplatin
Time frame: Cohort 2: Pre-dose (0 hr), 1, 2, 3, 4, 6, 8, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI, Cohort 2: CL/F of Oxaliplatin
Time frame: Cohort 2: Pre-dose (0 hr), 1, 2, 3, 4, 6, 8, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI, Cohort 1: Ratio of AUCinf on Cycle 1 Day 15 as Compared to Cycle 1 Day 1 for Irinotecan and Its Metabolite SN-38
Time frame: Cohort 1: Pre-dose (0 hr), 0.75, 1.5, 2.5, 3.5, 5.5, 7.5, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI, Cohort 1: Ratio of AUClast on Cycle 1 Day 15 as Compared to Cycle 1 Day 1 for Irinotecan and Its Metabolite SN-38
Time frame: Cohort 1: Pre-dose (0 hr), 0.75, 1.5, 2.5, 3.5, 5.5, 7.5, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI, Cohort 1: Ratio of Cmax on Cycle 1 Day 15 as Compared to Cycle 1 Day 1 for Irinotecan and Its Metabolite SN-38
Time frame: Cohort 1: Pre-dose (0 hr), 0.75, 1.5, 2.5, 3.5, 5.5, 7.5, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI, Cohort 2: Ratio of AUClast on Cycle 1 Day 15 as Compared to Cycle 1 Day 1 for Oxaliplatin
Time frame: Cohort 2: Pre-dose (0 hr), 1, 2, 3, 4, 6, 8, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
SLI, Cohort 2: Ratio of Cmax on Cycle 1 Day 15 as Compared to Cycle 1 Day 1 for Oxaliplatin
Time frame: Cohort 2: Pre-dose (0 hr), 1, 2, 3, 4, 6, 8, and 48 hrs post-dose on Days 1 and 15 of Cycle 1
Phase 3: OS for Arm B vs Arm C - FAS
Time frame: From date of first dose to death due to any cause or censoring date, whichever occurred first (maximum up to 37.25 months)
Phase 3: ORR by Derived Investigator Assessment for Arm B vs Arm C - FAS ORR Subset
Time frame: From date of randomization until documented PD, or start of subsequent anticancer therapy, or death, whichever occurred first (maximum up to 24.71 months)
Phase 3: ORR as Assessed by BICR - FAS
Time frame: From date of randomization until documented PD by BICR, or start of subsequent anticancer therapy, or death, whichever occurred first (maximum up to 37.25 months)
Phase 3: DOR as Assessed by BICR for Arm B Versus Arm C - FAS ORR Subset
Time frame: From date of first radiographic evidence of response (CR or PR) to earliest documented PD or death due to any cause, whichever occurred first (maximum up to 24.71 months)
Phase 3: DOR by Derived Investigator Assessment for Arm B Versus Arm C - FAS ORR Subset
Time frame: From date of first radiographic evidence of response (CR or PR) to earliest documented PD or death due to any cause, whichever occurred first (maximum up to 24.71 months)
Phase 3: PFS as Assessed by BICR - FAS
Time frame: From date of randomization to earliest documentation of PD or death or censoring date, whichever occurred first (maximum up to 37.25 months)
Phase 3: OS - FAS
Time frame: From date of first dose to death due to any cause or censoring date, whichever occurred first (maximum up to 37.25 months)
Phase 3: PFS by Derived Investigator Assessment - FAS
Time frame: From date of first dose to death due to any cause or censoring date, whichever occurred first (maximum up to 37.25 months)
Phase 3: TTR as Assessed by BICR for Arm B vs Arm C - FAS ORR Subset
Time frame: From date of first dose to CR or PR (maximum up to 24.71 months [107.37 weeks])
Phase 3: TTR by Derived Investigator Assessment for Arm B vs Arm C - FAS ORR Subset
Time frame: From date of first dose to CR or PR (maximum up to 24.71 months [107.37 weeks])
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 41230651Kopetz S, Tabernero J, Elez E. BREAKWATER Phase III: results for encorafenib and cetuximab plus mFOLFOX6 in first-line BRAF V600E-mutant metastatic colorectal cancer. Future Oncol. 2025 Dec;21(28):3585-3588. doi: 10.1080/14796694.2025.2579882. Epub 2025 Nov 13.
- Derived from this study: PubMed 40444708Elez E, Yoshino T, Shen L, Lonardi S, Van Cutsem E, Eng C, Kim TW, Wasan HS, Desai J, Ciardiello F, Yaeger R, Maughan TS, Morris VK, Wu C, Usari T, Laliberte R, Dychter SS, Zhang X, Tabernero J, Kopetz S; BREAKWATER Trial Investigators. Encorafenib, Cetuximab, and mFOLFOX6 in BRAF-Mutated Colorectal Cancer. N Engl J Med. 2025 Jun 26;392(24):2425-2437. doi: 10.1056/NEJMoa2501912. Epub 2025 May 30.
- Derived from this study: PubMed 39863775Kopetz S, Yoshino T, Van Cutsem E, Eng C, Kim TW, Wasan HS, Desai J, Ciardiello F, Yaeger R, Maughan TS, Beyzarov E, Zhang X, Ferrier G, Zhang X, Tabernero J. Encorafenib, cetuximab and chemotherapy in BRAF-mutant colorectal cancer: a randomized phase 3 trial. Nat Med. 2025 Mar;31(3):901-908. doi: 10.1038/s41591-024-03443-3. Epub 2025 Jan 25.
- Derived from this study: PubMed 36763936Van Cutsem E, Taieb J, Yaeger R, Yoshino T, Grothey A, Maiello E, Elez E, Dekervel J, Ross P, Ruiz-Casado A, Graham J, Kato T, Ruffinelli JC, Andre T, Carriere Roussel E, Klauck I, Groc M, Vedovato JC, Tabernero J. ANCHOR CRC: Results From a Single-Arm, Phase II Study of Encorafenib Plus Binimetinib and Cetuximab in Previously Untreated BRAFV600E-Mutant Metastatic Colorectal Cancer. J Clin Oncol. 2023 May 10;41(14):2628-2637. doi: 10.1200/JCO.22.01693. Epub 2023 Feb 10.
- To obtain contact information for a study center near you, click here.Link provided by the sponsor.
- Individual participant dataSharing planned: Yes. Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
Related trials for Neoplasms
Not yet recruitingPhase 1
Hepatic Artery Infusion of Carfilzomib in Participants With Liver Metastatic Disease Previously Treated With Hepatic Artery Infusion Pump Therapy
Colorectal Neoplasms, Neoplasms
RecruitingPhase 2
Tafasitamab With Acalabrutinib and Venetoclax for the Treatment of Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma
Chronic Lymphocytic Leukemia, Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma
Active, not recruitingPhase 1/2
Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma
Low Grade Glioma, Recurrent Childhood Pilocytic Astrocytoma
Enrolling by invitationPhase 2
A Single-Arm Study of Nimotuzumab Combined With NALIRIFOX as First-Line Treatment for Pancreatic Cancer
Pancreatic Cancer Metastatic
Recruiting
Methylation Profiling in Self-Samples for Identification of (Epi)Genetic Biomarkers for Gynecological Cancers
Gynaecological Cancers, Ovarian Cancer
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Pfizer CT.gov Call Center study director
Pfizer
274 sites in 29 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Encorafenib, Cetuximab and Oxaliplatin in people with Neoplasms, run by Pfizer at 274 sites in 29 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 5 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 274 sites in United States, China, Japan, Germany, Australia and 24 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 11 Jun 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
SLI: Number of Participants With Dose Limiting Toxicity (DLTs) (time frame: Cycle 1 (28 days)). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04607421, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Nov 2026.