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Clinical Trials in the USA / NCT06544499
Recruiting Phase 3

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

NCT06544499 · tracked via the Priya Life Science USA tracker
Sponsor
argenx
Phase
Phase 3
Started
2024-10-18
Last updated
2026-05-01

Condition(s) studied

Primary Immune Thrombocytopenia (ITP)

Investigational drug(s) / intervention(s)

Efgartigimod IVPlacebo IV

Efgartigimod IV: Intravenous infusion of efgartigimod

Placebo IV: Intravenous infusion of placebo

Study 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

Eligibility

Sex
ALL
Min age
18 Years
Max age
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (94)

FacilityCityRegionStatus
Mayo Clinic Hospital Scottsdale Phoenix Arizona Recruiting
University of Southern California Norris Comprehensive Cancer Center Los Angeles California Recruiting
Sharp Memorial Hospital Oceanside California Recruiting
University of Colorado Hospital Aurora Colorado Recruiting
Yale University School of Medicine New Haven Connecticut Recruiting
Georgetown University Hospital Washington D.C. District of Columbia Recruiting
The University of Chicago Medicine Chicago Illinois Recruiting
The University of Iowa Hospitals & Clinics Iowa City Iowa Recruiting
Tulane University New Orleans Louisiana Recruiting
Henry Ford Health Detroit Michigan Recruiting
University of Minnesota Minneapolis Minnesota Recruiting
Regional Cancer Care Associates, LLC (RCCA) Little Silver New Jersey Recruiting
Clinical Research Alliance Inc. Westbury New York Terminated
Duke University Medical Center Durham North Carolina Recruiting
Brody School of Medicine at East Carolina University Greenville North Carolina Recruiting
University Hospitals Cleveland Medical Center Cleveland Ohio Recruiting
Ohio State University Hospital East Columbus Ohio Recruiting
INTEGRIS Cancer Institute of Oklahoma Oklahoma City Oklahoma Recruiting
Allgemeines Krankenhaus der Stadt Linz Linz Austria Recruiting
Ordensklinikum Linz GmbH Elisabethinen Linz Austria Recruiting
Klinikum Kreuzschwestern Wels Wels Austria Recruiting
Acibadem City clinic Tokuda hospital Sofia Bulgaria Recruiting
UMHAT Aleksandrovska Sofia Bulgaria Recruiting
University Multi-Profile Hospital for Active Treatment (UMHAT) St. Ivan Rilski Sofia Bulgaria Recruiting
National Specialized Hospital for Active Treatment of Hematology Diseases Sofia Bulgaria Recruiting
UMHAT SofiaMed Sofia Bulgaria Recruiting
University Hospital Prof. Dr. Stoyan Kirkovich AD Stara Zagora Bulgaria Recruiting
Beijing YouYi Hospital-Beijing Friendship Hospital Capital Medical University Beijing China Recruiting
Peking University Third Hospital Beijing China Recruiting
The First Affiliated Hospital Of Bengbu Medical College Bengbu China Recruiting
The Affiliated Hospital of Chengde Medical University Chengde China Recruiting
The Affiliated Hospital of Inner Mongolia Medical University Hohhot China Recruiting
The Affiliated Huaian No.1 People's Hospital of Nanjing Medical University Huai'an China Recruiting
Huizhou Central People's Hospital Huizhou China Recruiting
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College Tianjin China Recruiting
Union Hospital Tongji Medical College of Huazhong University of Science and Technology (HUST) Wuhan China Recruiting
Wuxi People's Hospital (Wuxi No.1 People's Hospital) Wuxi China Recruiting
Affiliated Hospital of Jiangsu University Zhenjiang China Recruiting
University Hospital Center Zagreb Zagreb Croatia Recruiting
Fakultni Nemocnice Ostrava Ostrava Czechia Recruiting

+ 54 more sites — see the full list on the official registry below.

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06544499 on ClinicalTrials.gov ↗ ← All trials in the USA