Primary Immune Thrombocytopenia (ITP)Phase 3advance NEXTNCT06544499
A Study to Assess the Efficacy and Safety of Efgartigimod IV in Adult Participants With Primary Immune Thrombocytopenia
A Phase 3 study testing Efgartigimod IV in people with Primary Immune Thrombocytopenia (ITP), run by argenx at 94 sites in 17 countries.
Trial summary
The main purpose of this study is to look at the effect (efficacy) and safety of efgartigimod IV in participants with primary immune thrombocytopenia (ITP). After an up to 2 weeks screening period, eligible participants will be randomized in a 2:1 ratio to receive either efgartigimod IV or placebo IV, respectively… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Immune Thrombocytopenia (ITP)
- What is being tested?
- Efgartigimod IV
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 69 participants
- Where
- 94 sitesin 17 countries
- Study dates
- 18 Oct 2024to Jun 2028
- Phase
- Phase 3
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Is at least 18 years of age and the local legal age of consent for clinical studies when signing the informed consent form (ICF).
- Has documented baseline mean platelet count of <30 x 10^9/L before randomization
- Has a documented duration of primary immune thrombocytopenia (ITP) of more than 12 months on the date of informed consent form (ICF) signature.
- Has documented prior ITP treatment with at least 1 of the following treatments: corticosteroids, intravenous immunoglobulin (IVIg), anti-D immunoglobulin, thrombopoietin receptor agonist (TPO-RAs), or rituximab.
- Has documented insufficient response to a prior ITP treatment (the specific criteria can be found in the protocol).
- Has documented prior response defined as 1 platelet count of ≥50 × 109/L to at least 1 of the following ITP treatments in the 3 years before the date of ICF signature: prednisone, dexamethasone, other or nonspecified corticosteroids, IVIg, or anti-D immunoglobulin
Who cannot join
- Other than the indication under study, known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of ITP, confound the results of the study or put the participant at undue risk.
- Secondary ITP
- Nonimmune thrombocytopenia
- Autoimmune hemolytic anemia
- ITP-associated critical or severe bleeding The complete list of criteria can be found in the protocol.
Trial locations and contacts
94 sites in 17 countries. Contact the study team or a site near you to ask about taking part.
94 sites in 17 countries
United States18 sites, 17 recruiting
- Recruiting
University of Colorado Hospital
Aurora, Colorado, 80045
Gemlyn George, MD, 303-724-1513, gianni.solis@cuanschutz.edu
- Recruiting
The University of Chicago Medicine
Chicago, Illinois, 60637
Michael Drazer, MD, 773-834-9316, antojean.xavior@bsd.uchicago.edu
- Recruiting
University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106
Koen van Besien, MD, 216-844-8123, donna.kane1@uhhospitals.org
- Recruiting
Ohio State University Hospital East
Columbus, Ohio, 43203
Marina Beltrami Moreira, MD, 614-293-5176, Nives.Quaye@osumc.edu
- Recruiting
Henry Ford Health
Detroit, Michigan, 48202
Vrushali Dabak, MD, 313-556-8729, agopala7@hfhs.org
- Recruiting
Duke University Medical Center
Durham, North Carolina, 27710
Ara Metjian, MD, 919-681-2668, kristin.byrne@duke.edu
- Recruiting
Brody School of Medicine at East Carolina University
Greenville, North Carolina, 27834
Darla Liles, MD, 252-744-3617, wootenk16@ecu.edu
- Recruiting
The University of Iowa Hospitals & Clinics
Iowa City, Iowa, 52242
Steven Lentz, MD, 319-384-9643, darby-donovan@uiowa.edu
- Recruiting
Regional Cancer Care Associates, LLC (RCCA)
Little Silver, New Jersey, 07739
Iuliana Shapira, MD, 732-530-8666 X 1430, melanie.gonzalez@regionalcancercare.org
- Recruiting
University of Southern California Norris Comprehensive Cancer Center
Los Angeles, California, 90033
Caroline I Piatek, MD, 323-865-0371, duran_c@med.usc.edu
- Recruiting
University of Minnesota
Minneapolis, Minnesota, 55422
Marshall Mazepa, MD, 6126269520, wrig1316@umn.edu
- Recruiting
Yale University School of Medicine
New Haven, Connecticut, 06510
Yevgeniya Foster, MD, 203-623-5753, loren.rodriguez@yale.edu
- Recruiting
Tulane University
New Orleans, Louisiana, 70112
Maissaa Janbain, MD, 504-988-5433, cscott@tulane.edu
- Recruiting
Sharp Memorial Hospital
Oceanside, California, 92056
Catherine Quinn, MD, 760-272-4749, Christina.Spencer@profoundresearch.com
- Recruiting
INTEGRIS Cancer Institute of Oklahoma
Oklahoma City, Oklahoma, 73109
Bashar Alasad, MD, 405-636-7951, kellie.larsen@integrishealth.org
- Recruiting
Mayo Clinic Hospital Scottsdale
Phoenix, Arizona, 85054
Surbhi Shah, MD, 480-574-1853, ramirez.alexandria@mayo.edu
- Recruiting
Georgetown University Hospital
Washington D.C., District of Columbia, 20007
Catherine Broome, MD, 202-687-0116, js4936@georgetown.edu
- Terminated
Clinical Research Alliance Inc.
Westbury, New York, 11590
Italy12 sites, 12 recruiting
- Recruiting
Azienda ULSS 7 Pedemontana
Bassano del Grappa, 36061
Eros Di Bona, MD, +390424889434, eros.dibona@aulss7.veneto.it
- Recruiting
Universita Degli Studi Di Firenze - Azienda Ospedaliero-Universitaria Careggi (AOUC)
Florence, 50134
Valentina Carrai, MD, +390557949183, carraiv@aou-careggi.toscana.it
- Recruiting
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico
Milan, 20122
Andrea Artoni, MD, +390255035280, Andrea.artoni@policlinico.mi.it
clinicaltrials@argenx.com
- Recruiting
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico
Milan, 20122
Bruno Fattizzo, MD, +390255033477, bruno.fattizzo@policlinico.mi.it
clinicaltrials@argenx.com
- Recruiting
ASST Grande Ospedale Metropolitano Niguarda
Milan, 20162
Monica Carpenedo, MD, +390264442668, Monica.carpenedo@ospedaleniguarda.it
- Recruiting
AO Maggiore Della Carita
Novara, 28100
Andrea Patriarca, MD, +3903213733092, andrea.patriarca@uniupo.it
- Recruiting
U.O.C. Ematologia Ospedale Santa Maria delle Croci
Ravenna, 48121
Claudia Cellini, MD, +390544285752, claudia.cellini@auslromagna.it
- Recruiting
Arcispedale Santa Maria Nuova (ASMN) Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS)
Reggio Emilia, 42123
Barbara Gamberi, MD, +390522296626, Barbara.Gamberi@ausl.re.it
clinicaltrials@argenx.com
- Recruiting
A.O.U. Citta della Salute e della Scienza di Torino
Torino, 10126
Alessandra Borchiellini, MD, +390116335329, aborchiellini@cittadellasalute.to.it
- Recruiting
AO Ordine Mauriziano di Torino
Torino, 10128
Piera Sivera, MD, +390115082224, psivera@mauriziano.it
- Recruiting
Azienda Sanitaria Universitaria Integrata
Trieste, 34125
Francesco Zaja, MD, +390403992015, francesco.zaja@asugi.sanita.fvg.it
- Recruiting
ASST Sette Laghi Ospedale di circolo e Fondazione Macchi
Varese, 21100
Domenica Caramazza, MD, +39332278115, domenica.caramazza@asst-settelaghi.it
clinicaltrials@argenx.com
China11 sites, 11 recruiting
- Recruiting
Beijing YouYi Hospital-Beijing Friendship Hospital Capital Medical University
Beijing, 100050
Zhao Wang, MD, 13910623679, wangzhao@ccmu.edu.cn
- Recruiting
Peking University Third Hospital
Beijing, 100191
Hongmei Jing, MD, 18311319952, iqihui450052@sina.com
- Recruiting
The First Affiliated Hospital Of Bengbu Medical College
Bengbu, 233004
Feng Zhang, MD, 13855291898, 13855291898@163.com
- Recruiting
The Affiliated Hospital of Chengde Medical University
Chengde, 067020
Zhihua Zhang, MD, 857-350-4834, zzhangzhihua@163.com
- Recruiting
The Affiliated Hospital of Inner Mongolia Medical University
Hohhot, 010050
Da Gao, MD, 18547144520, 173064266@qq.com
- Recruiting
The Affiliated Huaian No.1 People's Hospital of Nanjing Medical University
Huai'an, 223300
Chunling Wang, MD, 13770498080, 21347725@qq.com
- Recruiting
Huizhou Central People's Hospital
Huizhou
Guowei Li, MD, 18719190842, 547081777@qq.com
- Recruiting
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College
Tianjin, 300020
Lei Zhang, MD, 18502220788, chenyunfei@ihcams.ac.cn
- Recruiting
Union Hospital Tongji Medical College of Huazhong University of Science and Technology (HUST)
Wuhan
Heng Mei, MD, 13212794115, xu_min1015@163.com
- Recruiting
Wuxi People's Hospital (Wuxi No.1 People's Hospital)
Wuxi, 214023
Yun Zhuang, MD, 15261533080, rmleiyang2023@163.com
- Recruiting
Affiliated Hospital of Jiangsu University
Zhenjiang, 212001
Xiaoming Fei, MD, 13952846267, luomingmm@126.com
United Kingdom8 sites, 8 recruiting
- Recruiting
Queen Elizabeth Hospital Birmingham - University Hospitals Birmingham NHS Foundation Trust
Birmingham, B15 2GW
Charles Percy, MD, 44 121 371 4351, hayley.ellis@uhb.nhs.uk
- Recruiting
Bradford Royal Infirmary - Bradford Teaching Hospitals NHS Foundation
Bradford, BD9 6RJ
Samuel Ackroyd, MD, 441274383383, Stephen.Cox@bthft.nhs.uk
clinicaltrials@argenx.com
- Recruiting
University Hospitals Coventry and Warwickshire NHS Trust
Coventry, CV2 2DX
Benjamin Bailiff, MD, +44 2476 967151, benjamin.bailiff@uhcw.nhs.uk
- Recruiting
Glasgow Royal Infirmary - North Glasgow University Hospital Division
Glasgow, G4 0ET
Catherine Bagot, MD, 441412013770, Jane.Ireland@nhs.scot
- Recruiting
University Hospitals of Leicester NHS Trust-Leicester Royal Infirmary
Leicester, LE1 5WW
Mamta Garg, MD, +44 1162584739, vernadine.roque@mhs.net
- Recruiting
Barts and the London Pathology & Pharmacy Building - Barts Health NHS Trust
London, E1 2ES
Frederick Chen, MD, +44 2032460261, s.mamsa@nhs.net
- Recruiting
Hammersmith Hospital, Imperial College School Of Medicine - Imperial College Healthcare NHS Trust
London, W12 0HS
Nichola Cooper, MD, +44 20 331 35711, tung.le1@nhs.net
- Recruiting
Royal Cornwall Hospital (RCH)
Truro, TR1 3LJ
Adam Forbes, MD, +44 1872 253075, rch-tr.HaematologyTrials@nhs.net
Poland7 sites, 7 recruiting
- Recruiting
Szpital Uniwersytecki nr 2 im. Dr Jana Biziela w Bydgoszczy
Bydgoszcz, 85-168
Marta Sobas, MD, +48795149026, marta.sobas@cm.umk.pl
- Recruiting
Pratia Onkologia Katowice
Katowice, 40-519
Sebastian Grosicki, MD, +48501714019, kontakt.onkologia.katowice@pratia.com
- Recruiting
Wojewodzkie Wielospecjalistyczne Centrum Onkologii i Traumatologii im. M. Kopernika w Lodzi
Lodz, 93-513
Jacek Trelinski, MD, +48426895191, Sekretariat.hemat@kopernik.lodz.pl
clinicaltrials@argenx.com
- Recruiting
NZOZ Neuromed M. i M. Nastaj Sp.P.
Lublin, 20-064
Tomasz Gromek, MD, +48791430975, gromek.neuromed@gmail.com
clinicaltrials@argenx.com
- Recruiting
Uniwersytecki Szpital Kliniczny Nr 4 w Lublinie Centrum Innowacyjnych Terapii
Lublin, 20-090
Marta Morawska, MD, +48814485180, mmorawska79@gmail.com
- Recruiting
Szpital Specjalistyczny im. Jedrzeja Sniadeckiego w Nowym Saczu
Nowy Sącz, 33-300
Szymon Fornagiel, MD, +48501079942, szf@wp.pl
clinicaltrials@argenx.com
- Recruiting
Wojewodzki Szpital Zespolony im. Ludwika Rydygiera w Toruniu
Torun, 87-100
Marcin Rymko, MD, +48566793745, hematologia@wszz.torun.pl
Bulgaria6 sites, 6 recruiting
- Recruiting
Acibadem City clinic Tokuda hospital
Sofia, 1407
Ismail Amine, MD, +359884993594, dramine66@gmail.com
- Recruiting
UMHAT Aleksandrovska
Sofia, 1431
Evgueniy Hadjiev, MD, +359898901090, e_hadji58@yahoo.com
- Recruiting
University Multi-Profile Hospital for Active Treatment (UMHAT) St. Ivan Rilski
Sofia, 1431
Atanas Radinoff, MD, +359884933151, aradinoff@hotmail.com
- Recruiting
National Specialized Hospital for Active Treatment of Hematology Diseases
Sofia, 1797
Tanya Yankova, MD, +359898719792, t.yankova@hematology.bg
- Recruiting
UMHAT SofiaMed
Sofia, 1797
Tzvetan Alaikov, MD, +359888627955, dr.alaikov@gmail.com
- Recruiting
University Hospital Prof. Dr. Stoyan Kirkovich AD
Stara Zagora, 6003
Maria Todorova, MD, +359896414650, dr.maria.dtodorova@gmail.com
Spain6 sites, 6 recruiting
- Recruiting
Complejo Hospitalario Universitario A Coruna
A Coruña, 15006
Michael Calvino Suarez, MD, +34981178000, Michael.Calvino.Suarez@sergas.es
- Recruiting
Instituto Catalan de Oncologia - Hospital Duran i Reynals
L'Hospitalet de Llobregat, 08908
Silvana Ana Novelli Canales, MD, +34932607733, snovelli@iconcologia.net
clinicaltrials@argenx.com
- Recruiting
Hospital Universitario Ramon y Cajal
Madrid, 28034
Francisco Javier Lopez Jimenez, MD, +34913368367, jljimenez@salud.madrid.org
- Recruiting
Hospital General Universitario Morales Meseguer
Murcia, 30008
Maria Luisa Lozano Almela, MD, +34968360900, mllozano@um.es
- Recruiting
University Hospital Quironsalud Madrid
Pozuelo de Alarcón, 28223
Maria Aranzazu Alonso Alonso, MD, +34914521900, aranzazu.alonso@quironsalud.es
- Recruiting
Hospital Clinico Universitario - University of Valencia
Valencia, 46010
Maria Luisa Calabuig Munoz, MD, +34961973500, marisacalabuig@yahoo.es
Serbia5 sites, 5 recruiting
- Recruiting
Clinical Center of Serbia
Belgrade, 11000
Mirjana Mitrovic, MD, +381 63 314 278, mirjanamitrovic777@gmail.com
- Recruiting
University Clinical Centre of Kragujevac
Kragujevac, 34000
Danijela Jovanovic, MD, 381641437733, daziv81@yahoo.com
- Recruiting
Clinical Center of Nis
Niš, 18000
Ivan Tijanic, MD, 381641198028, tijanic.ivan@yahoo.com
- Recruiting
University Clinical Hospital Center Zemun
Zemun, 11080
Zorica Cvetkovic, MD, 381641302624, zcvetkovic06@gmail.com
- Recruiting
General Hospital Djordje Joanovic
Zrenjanin, 23101
Nebojsa Rajic, MD, 381638342859, nebojsavrajic@gmail.com
clinicaltrials@argenx.com
Romania4 sites, 4 recruiting
- Recruiting
Spitalul Clinic Coltea, Clinica de Hematologie
Bucharest, 030171
Gabriela Borsaru, MD, +0213874100, secretariat@coltea.ro
clinicaltrials@argenx.com
- Recruiting
Spitalul Clinic Colentina
Bucharest, 20125
Georgeta Daniela Georgescu, MD, +0213173245, secretariat@spitalulcolentina.ro
clinicaltrials@argenx.com
- Recruiting
Spitalul Clinic Judetean de Urgenta Sibiu (SCJS) (Clinical County Hospital Sibiu)
Sibiu, 550245
Alina Catana, MD, +0269215050, secretariat@scjus.ro
clinicaltrials@argenx.com
- Recruiting
Spitalul Clinic Judetean de Urgenta Targu-Mures
Târgu Mureş, 540136
Ioan Macarie, MD, +0372653100, secretariat@spitalmures.ro
clinicaltrials@argenx.com
Austria3 sites, 3 recruiting
- Recruiting
Allgemeines Krankenhaus der Stadt Linz
Linz, 4020
Clemens Schmitt, MD, +4357680831610, clemens.schmitt@keplerklinkum.at
- Recruiting
Ordensklinikum Linz GmbH Elisabethinen
Linz, 4020
Robert Milanov, MD, +4373276764457, robert.milanov@ordensklinikum.at
- Recruiting
Klinikum Kreuzschwestern Wels
Wels, 4600
Sonja Heibl, MD, +43724241592201, sonja.heibl@klinikum-wegr.at
France3 sites, 3 recruiting
- Recruiting
CHU d'Amiens-Picardie - Hopital SUD
Amiens, 80054
Clement Gourguechon, MD, +33322455914, gourguechon.clement@chu-amiens.fr
- Recruiting
Centre Hospitalier Universitaire (CHU) - Hopital Henri Mondor
Créteil, 94010
Marc Michel, MD, +33149812076, Marc.michel2@aphp.fr
- Recruiting
Centre Hospitalier Universitaire Dijon Bourgogne Medecine Interne et Immunologie Clinique
Dijon, 21079
Bernard Bonnotte, MD, +33380293432, bernard.bonnotte@chu-dijon.fr
Czechia2 sites, 2 recruiting
- Recruiting
Fakultni Nemocnice Ostrava
Ostrava, 708 52
Ivo Demel, MD, +420597374576, ivo.demel@fno.cz
- Recruiting
Fakultni nemocnice Kralovske Vinohrady
Prague, 100 34
Olga Cerna, MD, +420267162887, olga.cerna@fnkv.cz
Germany2 sites, 2 recruiting
- Recruiting
University Hospital Carl Gustav Carus
Dresden, 01307
Karolin Trautmann, MD, +493514582311, Karolin.Trautmann@ukdd.de
clinicaltrials@argenx.com
- Recruiting
Universitaetsklinikum Essen (AoR)
Essen, 45147
Alexander Roeth, MD, +4920172384763, studienzentrum-haemaSZT@uk-essen.de
Hungary2 sites, 2 recruiting
- Recruiting
Semmelweis University-Internal Medicine and Haematology
Budapest, 1088
Zsolt Nagy, MD, +36208258660, nagy.zsolt@med.semmelweis-univ.hu
- Recruiting
Gyor-Moson-Sopron Varmegyei Petz Aladar Egyetemi Oktato Korhaz
Győr, 9024
Zsolt Lazar, MD, +3696418244, lazarzsolt1982@gmail.com
Ireland2 sites, 2 recruiting
- Recruiting
Mater Misericordiae University Hospital
Dublin, D07 R2WY
Anne Fortune, MD, +35318034831, CTRU@mater.ie
clinicaltrials@argenx.com
- Recruiting
St James's Hospital - Cancer Clinical Trials Office
Dublin, D08 NHY1
Catherine M Flynn, MD, +35314103756, cancerclinicaltrials@stjames.ie
Portugal2 sites, 2 recruiting
- Recruiting
Unidade Local de Saude de Braga
Braga, 4710
Carlos Alberto Pereira Capela, MD, +351253027100, Carloscapela@med.uminho.pt
- Recruiting
Unidade Local de Saude Gaia e Espinho, E.P.E.
Vila Nova de Gaia, 4434-502
Teresa Melo, MD, +351227865100, teresa.lencastre@ulsge.min-saude.pt
Croatia1 site, 1 recruiting
- Recruiting
University Hospital Center Zagreb
Zagreb, 10000
Drazen Pulanic, MD, +38512388277, dpulanic@yahoo.com
About this study
Official title: A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Arm Study Followed by an Open-Label Arm to Evaluate the Efficacy and Safety of Efgartigimod IV in Adult Participants With Primary Immune Thrombocytopenia
The main purpose of this study is to look at the effect (efficacy) and safety of efgartigimod IV in participants with primary immune thrombocytopenia (ITP). After an up to 2 weeks screening period, eligible participants will be randomized in a 2:1 ratio to receive either efgartigimod IV or placebo IV, respectively during the double-blinded treatment period (DBTP). At the end of the treatment period (up to 24 weeks), all participants will receive efgartigimod IV during the first 52-week open-label treatment period (OLTP1). At the end of the first OLTP1, participants may begin a second 52-week OLTP2. After the OLTP2, the participants will enter a follow-up period (approximately 8 weeks) while off study drug. The participants will be in the study for up to 138 weeks.
More information can be found here: https://clinicaltrials.argenx.com/advancenext
- Conditions
- Primary Immune Thrombocytopenia (ITP)
- Being tested
- Efgartigimod IV, Placebo IV
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Efgartigimod IVBiological
Intravenous infusion of efgartigimod
Placebo IVOther
Intravenous infusion of placebo
| Study group | Role | What they receive |
|---|---|---|
| Efgartigimod IV | Experimental | Participants receiving efgartigimod IV during the double-blinded treatment period and the open-label treatment period(s) Receives: Biological: Efgartigimod IV |
| Placebo IV | Experimental | Participants receiving placebo IV during the double-blinded treatment period and receiving efgartigimod IV during the open-label treatment period(s) Receives: Biological: Efgartigimod IV; Other: Placebo IV |
What is being measured
The primary outcome is the main question the study is designed to answer.
Extent of disease control, defined as the number of cumulative weeks during the 24-week Double-Blinded Treatment Period with platelet counts of at least 50 × 10^9/L
Measured: Up to 24 weeks
Secondary outcomes (28)
Proportion of participants achieving platelet counts of at least 50 × 10^9/L for at least 4 of the 6 study visits between study weeks 19 and 24 of the DBTP
Measured: Up to 6 weeks
Proportion of participants achieving platelet counts of at least 50 × 10^9/L for at least 6 of the 8 study visits between study weeks 17 and 24 of the DBTP
Measured: Up to 8 weeks
Proportion of participants achieving a platelet counts of at least 50 × 10^9/L for at least 8 of the 12 study visits between weeks 13 and 24 of the DBTP
Measured: Up to 12 weeks
Proportion of participants achieving a platelet count of at least 50 × 10^9/L on at least 4 occasions at any time until study week 12 during the DBTP
Measured: Up to 12 weeks
Time to response, defined as the time to achieve 2 consecutive platelet counts of at least 50 × 10^9/L at any time during the DBTP
Measured: up to 24 weeks
Proportion of participants with an IWG response during the DBTP
International Working Group (IWG)
Measured: Up to 24 weeks
Proportion of participants with initial response, defined as a platelet count of at least 30 × 10^9/L and a 2-fold increase from baseline in platelet count at study week 5 of the DBTP
Measured: Up to 20 weeks
Time to achieve a platelet count of at least 30 × 10^9/L and a 2-fold increase from baseline in platelet count during the DBTP
Measured: Up to 24 weeks
Number of cumulative weeks during the DBTP with platelet counts of at least 30 × 10^9/L and ≥20 × 10^9/L above baseline
Measured: Up to 24 weeks
Number of cumulative weeks during the DBTP with platelet counts of at least 30 × 10^9/L and at least 20 × 10^9/L above baseline in participants with baseline platelet counts of <15 × 10^9/L
Measured: Up to 24 weeks
Extent of disease control, defined as the percentage of weeks with platelet counts of at least 50 × 10^9/L
Measured: Up to 76 weeks
In participants receiving placebo IV in the DBTP, the extent of disease control, defined as the number of cumulative weeks with platelet counts of at least 50 × 10^9/L
Measured: Up to 76 weeks
Overall platelet count response, defined as the proportion of participants achieving a platelet count of at least 50 × 10^9/L on at least 4 occasions during the first 52 weeks of treatment with efgartigimod IV
Measured: Up to 52 weeks
Mean change from baseline in platelet count at each study visit
Measured: Up to 76 weeks
The percentage of weeks with platelet counts of at least 30 × 10^9/L and at least 20 × 10^9/L above baseline
Measured: Up to 76 weeks
In participants with baseline platelet counts <15 × 10^9/L, the percentage of weeks with platelet counts of at least 30 × 10^9/L and at least 20 × 10^9/L above baseline
Measured: Up to 76 weeks
Proportion of participants who achieve a sustained platelet count response, defined as achieving platelet counts of at least 50 × 10^9/L for at least 4 occasions during 6-week intervals
Measured: Up to 76 weeks
In participants receiving placebo IV in the DBTP, the proportion of participants achieving platelet counts of at least 50 × 10^9/L for at least 8 of the 12 study visits between weeks 13 and 24 of the OLTP1
Measured: Up to 12 weeks
In participants receiving placebo IV in the DBTP, proportion of participants who achieve sustained platelet count response, defined as achieving platelet counts of at least 50 × 10^9/L for at least 6 of 8 visits between weeks 17 and 24 of the OLTP1
Measured: Up to 8 weeks
In participants receiving placebo IV in the DBTP, the extent of disease control, defined as the number of cumulative weeks with platelet counts of at least 50 × 10^9/L during the first 24 weeks of treatment with efgartigimod IV
Measured: Up to 24 weeks
Occurrence rate of rescue ITP therapy
Measured: Up to 76 weeks
Proportion of participants with reduction in concurrent ITP therapy during the OLTP1
Measured: Up to 52 weeks
Incidence and severity of bleeding, assessed by the ITP Bleeding Scale (IBLS)
Measured: Up to 76 weeks
Incidence of AEs (including AEs of clinical interest) and SAEs
AE = adverse event; SAE = serious adverse event; An adverse event of clinical interest is a pre-specified medically significant event that has the potential to be causally associated with the study drug.
Measured: Up to 136 weeks
Incidence of ADA against efgartigimod in serum over time
ADA = antidrug antibodies
Measured: Up to 136 weeks
Incidence of NAb against efgartigimod in serum over time
NAb = Neutralizing antibodies
Measured: Up to 136 weeks
Efgartigimod Cmax over time
Measured: Up to 136 weeks
Percent change from baseline in total IgG levels in serum over time
IgG = immunoglobulin G
Measured: Up to 136 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Aug 2024
- Study start18 Oct 2024
- Primary completionJun 2028 (estimated)
- Study completionJun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Immune Thrombocytopenia (ITP)Studies of the same condition, open and under way.
- More trials run by argenxThe 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
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 69 participants (estimated)
Sponsor and investigators
- Lead sponsor
- argenx
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06544499
- Sponsor's protocol ID
- ARGX-113-2402
- Other IDs
- 2024-515451-38-00
- Acronym
- advance NEXT
- Status last verified
- Apr 2026
- First submitted
- 31 Jul 2024
- Last updated
- 1 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No