Recruiting
Phase 3

Efgartigimod IV

Sponsor:

argenx

Code:

NCT06544499

Conditions

Primary Immune Thrombocytopenia (ITP)

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Efgartigimod IV

Placebo IV

Study Details

Brief 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 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)

Study ID

NCT06544499

Start date

Oct 18, 2024

Status verified date

Apr, 2026

Completion date

Jun, 2028

Anticipated

Primary completion date

Jun, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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.

Study Design

Enrollment

69 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Efgartigimod IV

Participants receiving efgartigimod IV during the double-blinded treatment period and the open-label treatment period(s)

experimental: Placebo IV

Participants receiving placebo IV during the double-blinded treatment period and receiving efgartigimod IV during the open-label treatment period(s)

Interventions

Efgartigimod IV

Intravenous infusion of efgartigimod

Placebo IV

Intravenous infusion of placebo

Primary outcome measure

  • 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 [ Time Frame: Up to 24 weeks ]

Central Contacts and Locations

Central contacts

Sabine Coppieters, MD

857-350-4834Clinicaltrials@argenx.com

Locations

Mayo Clinic Hospital Scottsdale

Recruiting

Phoenix, Arizona, United States, 85054

Contacts

Surbhi Shah, MD

480-574-1853ramirez.alexandria@mayo.edu

University of Southern California Norris Comprehensive Cancer Center

Recruiting

Los Angeles, California, United States, 90033

Contacts

Caroline I Piatek, MD

323-865-0371duran_c@med.usc.edu

Sharp Memorial Hospital

Recruiting

Oceanside, California, United States, 92056

Contacts

Catherine Quinn, MD

760-272-4749Christina.Spencer@profoundresearch.com

University of Colorado Hospital

Recruiting

Aurora, Colorado, United States, 80045

Contacts

Gemlyn George, MD

303-724-1513gianni.solis@cuanschutz.edu

Yale University School of Medicine

Recruiting

New Haven, Connecticut, United States, 06510

Contacts

Yevgeniya Foster, MD

203-623-5753loren.rodriguez@yale.edu

Georgetown University Hospital

Recruiting

Washington D.C., District of Columbia, United States, 20007

Contacts

Catherine Broome, MD

202-687-0116js4936@georgetown.edu

The University of Chicago Medicine

Recruiting

Chicago, Illinois, United States, 60637

Contacts

Michael Drazer, MD

773-834-9316antojean.xavior@bsd.uchicago.edu

The University of Iowa Hospitals & Clinics

Recruiting

Iowa City, Iowa, United States, 52242

Contacts

Steven Lentz, MD

319-384-9643darby-donovan@uiowa.edu

Tulane University

Recruiting

New Orleans, Louisiana, United States, 70112

Contacts

Maissaa Janbain, MD

504-988-5433cscott@tulane.edu

Henry Ford Health

Recruiting

Detroit, Michigan, United States, 48202

Contacts

Vrushali Dabak, MD

313-556-8729agopala7@hfhs.org

University of Minnesota

Recruiting

Minneapolis, Minnesota, United States, 55422

Contacts

Marshall Mazepa, MD

6126269520wrig1316@umn.edu

Regional Cancer Care Associates, LLC (RCCA)

Recruiting

Little Silver, New Jersey, United States, 07739

Contacts

Iuliana Shapira, MD

732-530-8666 X 1430melanie.gonzalez@regionalcancercare.org

Duke University Medical Center

Recruiting

Durham, North Carolina, United States, 27710

Contacts

Ara Metjian, MD

919-681-2668kristin.byrne@duke.edu

Brody School of Medicine at East Carolina University

Recruiting

Greenville, North Carolina, United States, 27834

Contacts

Darla Liles, MD

252-744-3617wootenk16@ecu.edu

University Hospitals Cleveland Medical Center

Recruiting

Cleveland, Ohio, United States, 44106

Contacts

Koen van Besien, MD

216-844-8123donna.kane1@uhhospitals.org

Ohio State University Hospital East

Recruiting

Columbus, Ohio, United States, 43203

Contacts

Marina Beltrami Moreira, MD

614-293-5176Nives.Quaye@osumc.edu

INTEGRIS Cancer Institute of Oklahoma

Recruiting

Oklahoma City, Oklahoma, United States, 73109

Contacts

Bashar Alasad, MD

405-636-7951kellie.larsen@integrishealth.org

More Information

Sponsor

argenx

Last update posted

May 1, 2026

Last verified

Apr, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by argenx on 2026-05-01.