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Acute LeukemiaPhase 1/2cAMeLot-1NCT04811560

A Phase 1/2 Study of Bleximenib in Participants With Acute Leukemia (cAMeLot-1)

A Phase 1/2 study testing Bleximenib in people with Acute Leukemia, run by Janssen Research & Development, LLC at 102 sites in 13 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

The purpose of this study is to determine the recommended Phase 2 dose(s) (RP2D[s]) of bleximenib in phase 1 Part 1 (Dose Escalation) and to determine the safety and tolerability at RP2D in Phase 1 Part 2 (Dose expansion). Read more about this study

Who is it for?
Ages 2 years and older
Conditions the study is for
Acute Leukemia
What is being tested?
Bleximenib
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
420 participants
Where
102 sitesin 13 countries
Study dates
19 May 2021to 22 Sep 2030
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

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 2 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Phase 1:
  • Age 2 years to less than (<) 18 years of age (pediatric cohort only), all other cohorts 18 years and above
  • Relapsed or refractory (R/R) acute leukemia and has exhausted, or is ineligible for, available therapeutic options
  • Acute leukemia harboring histone-lysine N-methyltransferase 2A (KMT2A), nucleophosmin 1 gene (NPM1) or nucleoporin 98 gene or nucleoporin 214 gene (NUP98 or NUP214) alterations
  • Phase: 2
  • Participants greater than 18 years are eligible
Show all 13Show fewer
  • Must have had an initial diagnosis of acute myeloid leukemia (AML) per the WHO 2022 classification criteria and have relapsed/refractory disease
  • AML harboring KMT2A-r (gene rearrangement/translocation) or NPM1 mutations only
  • For Both Phase 1 and 2:
  • Pretreatment clinical laboratory values meeting the following criteria: (a) Hematology: white blood cell (WBC) count <= 20*10^9/liter (L) and (b) renal function; For adult participants, estimated or measured glomerular filtration rate >= 30 milliliter per minute (mL/min) per four variable MDRD equation. For pediatric participants an estimated or measured glomerular filtration rate >=40 mL/min per the CKiD (Chronic Kidney Disease in Children) Schwartz formula
  • Eastern Cooperative Oncology Group (ECOG) performance status grade of 0, 1 or 2. Pediatric participants only: Performance status >=70 by Lansky scale (for participants < 16 years of age) or >=70 Karnofsky scale (for participants >=16 years of age)
  • A female of childbearing potential must have a negative highly sensitive serum beta-human chorionic gonadotropin at screening and within 48 hours prior to the first dose of study treatment
  • Participant must agree to all protocol required contraception requirements and avoid sperm or egg donations or freezing for future reproductive use while on study and for 90 days (males) or 6 months (females) after the last dose of study treatment

Who cannot join

  • Acute promyelocytic leukemia, diagnosis of Down syndrome associated leukemia or juvenile myelomonocytic leukemia according to World Health Organization (WHO) 2016 criteria
  • Active central nervous system (CNS) disease
  • Prior solid organ transplantation
  • QTc according to Fridericia's formula (QTcF) for males >= 450 millisecond (msec) or for females >= 470 msec. Participants with a family history of Long QT syndrome are excluded
  • Exclusion criteria related to stem cell transplant: a. Received prior treatment with allogenic bone marrow or stem cell transplant <=3 months before the first dose of study treatment ; b. Has evidence of graft versus host disease; c. Received donor lymphocyte infusion <=1 month before the first dose of study treatment; d. Requires immunosuppressant therapy (exception: daily doses <=10 milligrams (mg) prednisone or equivalent are allowed for adrenal replacement)
  • Prior cancer immunotherapy within 4 weeks prior to enrollment or blinatumomab within 2 weeks prior to enrollment. Additional prior cancer therapies must not be given within 4 weeks prior to enrollment or 5 half-lives of the agent (whichever is shorter)

Trial locations and contacts

102 sites in 13 countries. Contact the study team or a site near you to ask about taking part.

Study Contact contact

844-434-4210

Participate-In-This-Study1@its.jnj.com

102 sites in 13 countries

United States26 sites, 22 recruiting

  • University of New Mexico

    Albuquerque, New Mexico, 87131

    Recruiting

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

    Recruiting

  • Dana Farber Cancer Institute

    Boston, Massachusetts, 02115

    Recruiting

  • Roswell Park Comprehensive Cancer Center

    Buffalo, New York, 14263

    Recruiting

  • University of Chicago

    Chicago, Illinois, 60637

    Recruiting

  • Baylor University Medical Center

    Dallas, Texas, 75246

    Recruiting

  • Karmanos Cancer Institute

    Detroit, Michigan, 48201 2013

    Completed

  • City of Hope

    Duarte, California, 91010

    Recruiting

  • Start Midwest

    Grand Rapids, Michigan, 49546

    Recruiting

  • Houston Methodist Hospital

    Houston, Texas, 77030-2740

    Recruiting

  • MD Anderson

    Houston, Texas, 77030

    Recruiting

  • St Francis Hospital & Health Centers Indiana Blood and Marrow Transplantation

    Indianapolis, Indiana, 46237

    Recruiting

  • University of Tennessee Medical Center

    Knoxville, Tennessee, 37920

    Recruiting

  • Norton Cancer Institute

    Louisville, Kentucky, 40207

    Completed

  • Medical College of WI at Froedtert

    Milwaukee, Wisconsin, 53226

    Recruiting

  • NYU Langone Medical Center

    New York, New York, 10016

    Recruiting

  • University of California Irvine Medical Center

    Orange, California, 92868

    Completed

  • Fox Chase Cancer Center

    Philadelphia, Pennsylvania, 19111

    Completed

  • Oregon Health And Science University

    Portland, Oregon, 97239

    Recruiting

  • Virginia Commonwealth University

    Richmond, Virginia, 23298

    Recruiting

  • San Antonio Methodist TX Transplant Physicians Group

    San Antonio, Texas, 78229

    Recruiting

  • University of California San Francisco

    San Francisco, California, 94143

    Recruiting

  • UCSF Benioff Children's Hospital

    San Francisco, California, 94158

    Recruiting

  • Swedish Cancer Institute

    Seattle, Washington, 98104

    Recruiting

  • Maine Health

    South Portland, Maine, 04106

    Recruiting

  • Montefiore Medical Center

    The Bronx, New York, 10467

    Recruiting

Other trials in United States

France13 sites, 12 recruiting

  • Hopital Jean Minjoz

    Besançon, 25000

    Recruiting

  • Institut Paoli Calmettes

    Marseille, 13009

    Recruiting

  • CHU de Nantes hotel Dieu

    Nantes, 44093

    Recruiting

  • Hopital Saint Louis

    Paris, 75010

    Recruiting

  • Hopital trousseau- APHP

    Paris, 75012

    Recruiting

  • Centre Hospitalier Universitaire (CHU) de Bordeaux Hopital HautLeveque Centre Francois Magendie

    Pessac, 33604

    Completed

  • CHU Lyon Sud

    Pierre-Bénite, 69310

    Recruiting

  • Hôpitaux Universitaires de Strasbourg - Hôpital de Hautepierre

    Strasbourg, 67098

    Recruiting

  • Institut Universitaire du Cancer Toulouse Oncopole

    Toulouse, 31059

    Recruiting

  • CHU Bretonneau

    Tours, 37044

    Recruiting

  • CHRU de Nancy - Hopitaux de Brabois

    Vandœuvre-lès-Nancy, 54500

    Recruiting

  • CHRU Nancy Brabois

    Vandœuvre-lès-Nancy, 54511

    Recruiting

  • Gustave Roussy

    Villejuif, 94805

    Recruiting

Other trials in France

China12 sites, 11 recruiting

  • Peking University First Hospital

    Beijing, 100034

    Recruiting

  • The First Hospital of Jilin University

    Changchun, 130021

    Recruiting

  • West China Hospital Si Chuan University

    Chengdu, 610041

    Recruiting

  • Nanfang Hospital of Southern Medical Hospital

    Guangzhou, 510515

    Completed

  • First Affiliated Hospital Medical School of Zhejiang University

    Hangzhou, 310009

    Recruiting

  • Qilu Hospital of Shandong University

    Jinan, 250012

    Recruiting

  • The First Affiliated Hospital of NanChang University

    Nanchang, 330006

    Recruiting

  • Zhongda Hospital Southeast University

    Nanjing, 210009

    Recruiting

  • Institute of Hematology and Blood Diseases Hospital

    Tianjin, 301609

    Recruiting

  • The First Affiliated Hospital of Wenzhou Medical University

    Wenzhou, 325000

    Recruiting

  • Union Hospital Tongji Medical College of Huazhong University of Science and Technology

    Wuhan, 430022

    Recruiting

  • Henan Cancer Hospital

    Zhengzhou, 450003

    Recruiting

Other trials in China

Japan11 sites

  • Kyushu University Hospital

    Fukuoka, 812-0054

    Active, not recruiting

  • Fukushima Medical University Hospital

    Fukushima, 960 1295

    Active, not recruiting

  • Kanazawa University Hospital

    Kanazawa, 920 8641

    Active, not recruiting

  • National Cancer Center Hospital East

    Kashiwa, 277-8577

    Active, not recruiting

  • Kobe City Medical Center General Hospital

    Kobe, 650 0047

    Completed

  • Gunmaken Saiseikai Maebashi Hospital

    Maebashi, 371-0821

    Completed

  • Nagoya University Hospital

    Nagoya, 466-8560

    Completed

  • Hokkaido University Hospital

    Sapporo, 060-8648

    Completed

  • NTT Medical Center Tokyo

    Tokyo, 141-8625

    Active, not recruiting

  • Yamagata University Hospital

    Yamagata, 990 2331

    Completed

  • University of Fukui Hospital

    Yoshida, 910-1193

    Completed

Other trials in Japan

Spain9 sites, 9 recruiting

  • Hosp Univ Vall D Hebron

    Barcelona, 08035

    Recruiting

  • Hosp Clinic de Barcelona

    Barcelona, 08036

    Recruiting

  • Hosp. Gral. Univ. Gregorio Maranon

    Madrid, 28007

    Recruiting

  • Hospital Infantil Universitario Nino Jesus

    Madrid, 28009

    Recruiting

  • Hosp Univ Fund Jimenez Diaz

    Madrid, 28040

    Recruiting

  • Clinica Univ. de Navarra

    Pamplona, 31008

    Recruiting

  • Hosp Clinico Univ de Salamanca

    Salamanca, 37007

    Recruiting

  • Hosp. Virgen Del Rocio

    Seville, 41013

    Recruiting

  • Hosp. Clinico Univ. de Valencia

    Valencia, 46010

    Recruiting

Other trials in Spain

United Kingdom8 sites, 8 recruiting

  • University Hospital of Wales

    Cardiff, CF14 4XW

    Recruiting

  • Leeds Teaching Hospitals NHS Trust

    Leeds, LS9 7TF

    Recruiting

  • The Clatterbridge Cancer Centre

    Liverpool, L7 8YA

    Recruiting

  • Guys and St Thomas NHS Foundation Trust

    London, SE1 9RT

    Recruiting

  • University College London Hospitals

    London, W1T 7HA

    Recruiting

  • The Christie Nhs Foundation Trust

    Manchester, M20 4BX

    Recruiting

  • Oxford University Hospitals NHS Trust

    Oxfordshire, OX3 7LE

    Recruiting

  • University Hospitals Plymouth NHS Trust

    Plymouth, PL6 8DH

    Recruiting

Other trials in United Kingdom

Brazil6 sites, 6 recruiting

  • IDOR - Regional Brasilia

    Brasília, 70390-140

    Recruiting

  • Liga Norte Riograndense Contra O Cancer

    Natal, 59062 000

    Recruiting

  • Ministerio da Saude Instituto Nacional do Cancer

    Rio de Janeiro, 20230 130

    Recruiting

  • Fundacao Faculdade de Medicina - Instituto do Cancer do Estado de Sao Paulo

    São Paulo, 01246 000

    Recruiting

  • IDOR - Regional Sao Paulo

    São Paulo, 01401 002

    Recruiting

  • Fundacao Antonio Prudente A C Camargo Cancer Center

    São Paulo, 01509 900

    Recruiting

Other trials in Brazil

Israel4 sites, 4 recruiting

  • Carmel Medical Center

    Haifa, 3436212

    Recruiting

  • Hadassah University Hospita Ein Kerem

    Jerusalem, 9112001

    Recruiting

  • Sheba Medical Center

    Ramat Gan, 52621

    Recruiting

  • Tel Aviv Sourasky Medical Center

    Tel Aviv, 6423906

    Recruiting

Other trials in Israel

Australia3 sites, 3 recruiting

  • Monash Medical Centre

    Clayton, 3168

    Recruiting

  • Royal Perth Hospital

    Perth, 6000

    Recruiting

  • Gold Coast University Hospital

    Southport, 4211

    Recruiting

Other trials in Australia

Canada3 sites, 3 recruiting

  • Hopital Maisonneuve Rosemont

    Montreal, Quebec, H1T 2M4

    Recruiting

  • Princess Margaret Cancer Centre University Health Network

    Toronto, Ontario, M5G 1X6

    Recruiting

  • Vancouver Coastal Health

    Vancouver, British Columbia, V5Z 1M9

    Recruiting

Other trials in Canada

South Korea3 sites, 3 recruiting

  • Seoul National University Hospital

    Seoul, 03080

    Recruiting

  • Asan Medical Center

    Seoul, 05505

    Recruiting

  • Samsung Medical Center

    Seoul, 06351

    Recruiting

Other trials in South Korea

Taiwan3 sites, 3 recruiting

  • China Medical University Hospital

    Taichung, 404327

    Recruiting

  • National Cheng Kung University Hospital

    Tainan, 704

    Recruiting

  • National Taiwan University Hospital

    Taipei, 10043

    Recruiting

Other trials in Taiwan

Belgium1 site, 1 recruiting

  • Ghent University Hospital

    Ghent, 9000

    Recruiting

Other trials in Belgium

About this study

Official title: A Phase 1/2, First-in-Human Study of the Menin-KMT2A (MLL1) Inhibitor Bleximenib in Participants With Acute Leukemia (cAMeLot-1)

The purpose of this study is to determine the recommended Phase 2 dose(s) (RP2D[s]) of bleximenib in phase 1 Part 1 (Dose Escalation) and to determine the safety and tolerability at RP2D in Phase 1 Part 2 (Dose expansion). The purpose of the Phase 2 part of the study is to evaluate the efficacy of bleximenib at the RP2D.

Conditions
Acute Leukemia
Being tested
Bleximenib

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BleximenibDrug

    Also called JNJ-75276617

    Bleximenib is administered orally.

Study groups in NCT04811560
Study groupRoleWhat they receive
BleximenibExperimentalParticipants in Phase 1 Part 1 (dose escalation) will receive bleximenib orally. The dose levels will be escalated based on the dose limiting toxicities (DLT) evaluation by Study Evaluation Team (SET) until the recommended Phase 2 Doses (RP2Ds) have been identified. Participants in Phase 1 Part 2 (dose expansion) will receive bleximenib orally at the RP2D(s) determined in Part 1. In Phase 2 participants will receive bleximenib at the RP2D to evaluate anti-leukemia activity and demonstrate acceptable safety at the RP2D(s).
Receives: Drug: Bleximenib

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Phase 1: Number of Participants with Adverse Events (AEs) as a Measure of Safety and Tolerability

    An AE is any untoward medical occurrence in a participant participating in a clinical study that does not necessarily have a causal relationship with the pharmaceutical/biological agent under study.

    Measured: Up to 4 years and 9 months

  2. Phase 1: Number of Participants with AEs by Severity

    Severity will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0. Severity scale ranges from Grade 1 (Mild) to Grade 5 (Death). Grade 1= Mild, Grade 2= Moderate, Grade 3= Severe, Grade 4= Life-threatening and Grade 5= Death related to adverse event.

    Measured: Up to 4 years and 9 months

  3. Phase 1: Part 1: Percentage of Participants with Dose-Limiting Toxicity (DLT)

    Percentage of participants with DLT will be assessed accordingly to national cancer institute common terminology criteria for adverse events (NCI-CTCAE) version 5.

    Measured: Up to 28 days Cycle 1

  4. Phase 2: Rate of Complete Remission or Complete Remission with Partial Hematologic Recovery (CR/CRh)

    Rate of CR/CRh is defined as the percentage of participants achieving a CR or CRh at any time post-treatment.

    Measured: Up to 4 years and 9 months

Secondary outcomes (11)

  1. Phase 1 and 2: Plasma Concentration of Bleximenib

    Plasma concentration of bleximenib will be reported.

    Measured: Up to 4 years and 9 months

  2. Phase 1 and 2: Overall Response Rate (ORR)

    ORR is defined as the percentage of participants who achieve any response.

    Measured: Up to 4 years and 9 months

  3. Phase 1: Duration of Response (DOR)

    DOR will be calculated among responders from the date of initial documentation of a response to the date of first documented evidence of relapse, as defined in the disease-specific response criteria, or death due to any cause, whichever occurs first.

    Measured: Up to 4 years and 9 months

  4. Phase 1 and 2: Time To Response (TTR)

    TTR is defined for the responders as the time from the date of the first dose of bleximenib to the date of the first documented response.

    Measured: Up to 4 years and 9 months

  5. Phase 2: Duration of Complete Response (CR)/Complete Remission With Partial Hematologic Recovery (CRh)

    The duration of CR/CRh is defined from the date of first CR or CRh response achieved to the date of first evidence of relapsed disease or death due to any cause, whichever occurs first, for participants who achieve a CR or CRh.

    Measured: Up to 4 years and 9 months

  6. Phase 2: Time To CR/CRh

    Time to CR/CRh is defined for responders as the time from the date of the first dose of bleximenib to the date of first achieving either CR or CRh, depending on which milestone is reached.

    Measured: Up to 4 years and 9 months

  7. Phase 2: Event-free survival (EFS)

    EFS is defined as the time from the date of first dose of study treatment to the date of treatment failure, relapse, or death due to any cause, whichever occurs first.

    Measured: Up to 4 years and 9 months

  8. Phase 2: Overall survival (OS)

    OS is defined from the date of first dose of study treatment to the date of death due to any cause.

    Measured: Up to 4 years and 9 months

  9. Phase 2: Measurable Residual Disease (MRD) Negativity Among Participants Achieving CR/CRh/CRi

    MRD-negative rate is defined as the percentage of participants who are MRD-negative at any timepoint after the first dose of bleximenib in the responders.

    Measured: Up to 4 years and 9 months

  10. Phase 2: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)

    An AE is any untoward medical event that occurs in a participant administered an investigational product, and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product. A Serious AE is an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.

    Measured: Up to 4 years and 9 months

  11. Phase 2: Number of Participants Reporting Transfusion Independence

    Transfusion independence is defined as independence from red blood cells (RBC) and platelet transfusions during any 56-day interval after receiving study treatment.

    Measured: Up to 4 years and 9 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted23 Mar 2021
  2. Study start19 May 2021
  3. Primary completion29 Dec 2028 (estimated)
  4. Study completion22 Sep 2030 (estimated)

Resources

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 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Sequential
Masking
None (open label)
Enrolment
420 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04811560
Sponsor's protocol ID
CR108998
Other IDs
75276617ALE1001, 2023-506581-31-00, 2023
Acronym
cAMeLot-1
Status last verified
Nov 2026
First submitted
22 Mar 2021
Last updated
4 Nov 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: The data sharing policy of Johnson \& Johnson Innovative Medicine is available at innovativemedicine.jnj.com/our-innovation/clinical-trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu

Publications

  • Kwon MC, Thuring JW, Querolle O, Dai X, Verhulst T, Pande V, Marien A, Goffin D, Wenge DV, Yue H, Cutler JA, Jin C, Perner F, Hogeling SM, Shaffer PL, Jacobs F, Vinken P, Cai W, Keersmaekers V, Eyassu F, Bhogal B, Verstraeten K, El Ashkar S, Perry JA, Jayaguru P, Barreyro L, Kuchnio A, Darville N, Krosky D, Urbanietz G, Verbist B, Edwards JP, Cowley GS, Kirkpatrick R, Steele R, Ferrante L, Guttke C, Daskalakis N, Pietsch EC, Wilson DM, Attar R, Elsayed Y, Fischer ES, Schuringa JJ, Armstrong SA, Packman K, Philippar U. Preclinical efficacy of the potent, selective menin-KMT2A inhibitor JNJ-75276617 (bleximenib) in KMT2A- and NPM1-altered leukemias. Blood. 2024 Sep 12;144(11):1206-1220. doi: 10.1182/blood.2023022480.

    PubMed 38905635

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Bleximenib in people with Acute Leukemia, run by Janssen Research & Development, LLC at 102 sites in 13 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 13 inclusion and 6 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: looking for participants now, as of the sponsor's last update on 4 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 102 sites in United States, France, China, Japan, Spain and 8 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 combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Study Contact (844-434-4210, Participate-In-This-Study1@its.jnj.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Phase 1: Number of Participants with Adverse Events (AEs) as a Measure of Safety and Tolerability (time frame: Up to 4 years and 9 months). 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 NCT04811560, which also lists 1 linked publication.

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 4 Nov 2026.

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