A Study to Assess the Efficacy and Safety of Efgartigimod IV in Adult Participants With Primary Immune Thrombocytopenia

Recruiting now · Phase 3 · Has a placebo group

Conditions studied: Primary Immune Thrombocytopenia (ITP)

In brief

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

Key facts

Study ID

NCT06544499

Run by

argenx

People needed

69

Starts

2024-10-18

Expected to finish

2028-06-01

Last updated by the study team

2026-05-01

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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.

Where it is running

• University of Southern California Norris Comprehensive Cancer Center — Los Angeles, California, United States (enrolling)
• Mayo Clinic Hospital Scottsdale — Phoenix, Arizona, United States (enrolling)
• University of Colorado Hospital — Aurora, Colorado, United States (enrolling)
• Yale University School of Medicine — New Haven, Connecticut, United States (enrolling)
• Sharp Memorial Hospital — Oceanside, California, United States (enrolling)
• The University of Chicago Medicine — Chicago, Illinois, United States (enrolling)
• The University of Iowa Hospitals & Clinics — Iowa City, Iowa, United States (enrolling)
• Tulane University — New Orleans, Louisiana, United States (enrolling)
• Henry Ford Health — Detroit, Michigan, United States (enrolling)
• University of Minnesota — Minneapolis, Minnesota, United States (enrolling)
• Regional Cancer Care Associates, LLC (RCCA) — Little Silver, New Jersey, United States (enrolling)
• Georgetown University Hospital — Washington D.C., District of Columbia, United States (enrolling)
• Duke University Medical Center — Durham, North Carolina, United States (enrolling)
• Brody School of Medicine at East Carolina University — Greenville, North Carolina, United States (enrolling)
• University Hospitals Cleveland Medical Center — Cleveland, Ohio, United States (enrolling)
• Ohio State University Hospital East — Columbus, Ohio, United States (enrolling)
• INTEGRIS Cancer Institute of Oklahoma — Oklahoma City, Oklahoma, United States (enrolling)
• Allgemeines Krankenhaus der Stadt Linz — Linz, Austria (enrolling)
• Ordensklinikum Linz GmbH Elisabethinen — Linz, Austria (enrolling)
• Klinikum Kreuzschwestern Wels — Wels, Austria (enrolling)
• Acibadem City clinic Tokuda hospital — Sofia, Bulgaria (enrolling)
• UMHAT Aleksandrovska — Sofia, Bulgaria (enrolling)
• University Multi-Profile Hospital for Active Treatment (UMHAT) St. Ivan Rilski — Sofia, Bulgaria (enrolling)
• National Specialized Hospital for Active Treatment of Hematology Diseases — Sofia, Bulgaria (enrolling)
• UMHAT SofiaMed — Sofia, Bulgaria (enrolling)

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.