Actively Recruiting
A Pilot Study of Efgartigimod for Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP)
Led by University of Minnesota · Updated on 2025-05-29
15
Participants Needed
1
Research Sites
156 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Immune-mediated Thrombotic thrombocytopenic purpura (iTTP) is a rare, autoimmune disorder characterized by life-threatening episodes of thrombocytopenia, microangiopathic hemolytic anemia and organ damage. Patients have an unpredictable course punctuated by relapses associated with autoantibody-mediated (primarily IgG) depletion of ADAMTS13, a key regulator of coagulation. ADAMTS13 deficiency during remission has been associated with increased risk of relapse, but also, and potentially more devastating, ischemic stroke. Until recently, it was presumed that rituximab (a monoclonal antibody targeting B cells) improved relapse-free survival in most patients, but this was based on findings from very small studies. Given concern about stroke and relapse risk, preventive immunosuppression with rituximab has also recently come into practice for patients with falling ADAMTS13 activity (ADAMTS13-relapse). It is expected that following efgartigimod therapy, there will be a rise in ADAMTS13 activity to the normal range that will be sustained during the treatment period. Following withdrawal of therapy, it is expected that most participants will experience a fall in ADAMTS13 activity, demonstrating the safety and efficacy in efgartigimod to reliably but temporarily reduce pathogenic antibodies. This would demonstrate the potential efficacy for efgartigimod as a maintenance therapy to safely prevent relapse of iTTP to be further explored in a larger efficacy study.
CONDITIONS
Official Title
A Pilot Study of Efgartigimod for Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Subject must provide a signed informed consent form
- Subject is 18 years or older at the time of screening
- Subject has a prior history of iTTP defined by ADAMTS13 activity < 10% with ADAMTS13 antibodies or inhibitor, thrombocytopenia (platelet count < 100), and microangiopathic hemolytic anemia (schistocytes on blood smear)
- Subject is in clinical remission from iTTP with normal platelet count for at least 90 days
- Subject has ADAMTS13 activity between < 70% and > 30% on 2 separate occasions at least 7 days apart
- Subject is at least 6 months from last dose of rituximab or other intravenous immunosuppression
- If taking oral immunosuppressants, no change in dose for at least 60 days
- Female subjects of childbearing potential must have a negative pregnancy test and agree to use highly effective birth control during the study
- Sexually active male subjects must agree to use effective contraception during the study
You will not qualify if you...
- Subject has been diagnosed with congenital thrombotic thrombocytopenic purpura (cTTP)
- Subject has used another investigational product within 30 days before enrollment or plans to participate in another investigational study during this study
- Subject cannot understand the nature, scope, or possible consequences of the study
- Subject is pregnant or lactating
- Subject has a known life-threatening allergy to efgartigimod
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
University of Minnesota
Minneapolis, Minnesota, United States, 55455
Actively Recruiting
Research Team
D
Diondra Howard
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here