A Study to Assess the Efficacy and Safety of Efgartigimod IV in Adult Participants With Primary Immune Thrombocytopenia
Purpose
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
Condition
- Primary Immune Thrombocytopenia (ITP)
Eligibility
- Eligible Ages
- Over 18 Years
- Eligible Sex
- All
- Accepts 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.
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
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) |
|
Recruiting Locations
Mayo Clinic Hospital Scottsdale
Phoenix, Arizona 85054
Phoenix, Arizona 85054
University of Southern California Norris Comprehensive Cancer Center
Los Angeles, California 90033
Los Angeles, California 90033
Sharp Memorial Hospital
Oceanside, California 92056
Oceanside, California 92056
University of Colorado Hospital
Aurora, Colorado 80045
Aurora, Colorado 80045
Yale University School of Medicine
New Haven, Connecticut 06510
New Haven, Connecticut 06510
Georgetown University Hospital
Washington D.C., District of Columbia 20007
Washington D.C., District of Columbia 20007
The University of Chicago Medicine
Chicago, Illinois 60637
Chicago, Illinois 60637
The University of Iowa Hospitals & Clinics
Iowa City, Iowa 52242
Iowa City, Iowa 52242
Tulane University
New Orleans, Louisiana 70112
New Orleans, Louisiana 70112
Henry Ford Health
Detroit, Michigan 48202
Detroit, Michigan 48202
University of Minnesota
Minneapolis, Minnesota 55422
Minneapolis, Minnesota 55422
Regional Cancer Care Associates, LLC (RCCA)
Little Silver, New Jersey 07739
Little Silver, New Jersey 07739
Duke University Medical Center
Durham, North Carolina 27710
Durham, North Carolina 27710
Brody School of Medicine at East Carolina University
Greenville, North Carolina 27834
Greenville, North Carolina 27834
University Hospitals Cleveland Medical Center
Cleveland, Ohio 44106
Cleveland, Ohio 44106
Ohio State University Hospital East
Columbus, Ohio 43203
Columbus, Ohio 43203
INTEGRIS Cancer Institute of Oklahoma
Oklahoma City, Oklahoma 73109
Oklahoma City, Oklahoma 73109
More Details
- NCT ID
- NCT06544499
- Status
- Recruiting
- Sponsor
- argenx