NCT06831058 A Pilot Study of Efgartigimod for Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP)
| NCT ID | NCT06831058 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | University of Minnesota |
| Condition | Immune-mediated Thrombotic Thrombocytopenic Purpura |
| Study Type | INTERVENTIONAL |
| Enrollment | 15 participants |
| Start Date | 2025-05-01 |
| Primary Completion | 2028-05-01 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 15 participants in total. It began in 2025-05-01 with a primary completion date of 2028-05-01.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
Inclusion Criteria: 1. Subject must provide a signed informed consent form 2. Subject is 18 years or older at the time of screening 3. Subject has a prior history of iTTP as defined by the presence of ADAMTS13 activity \< 10% with ADAMTS13 antibodies or inhibitor, thrombocytopenia (platelet count \< 100) and microangiopathic hemolytic anemia (defined by the presence of schistocytes on blood smear) 4. Subject is in clinical remission from iTTP (normal platelet count) for at least 90 days 5. Subject has ADAMTS13 activity \< 70% and \> 30% on 2 separate occasions separate by at least 7 days 6. Subject is at least 6 months from last dose of rituximab or other intravenous immunosuppression 7. If taking other oral immunosuppressants, no change in dose for at least 60 days 8. Female subjects of childbearing potential must present with a negative pregnancy test and agree to employ highly effective birth control measures for duration of study. Sexually active male subjects must agree to use an effective method of contraception for the duration of the study Exclusion Criteria: 1. Subject has been diagnosed with cTTP 2. Subject has been exposed to another investigational product within 30 days prior to enrollment or is scheduled to participate in another clinical study involving investigational product or investigational device during the course of the study 3. Subject is unable to understand the nature, scope, and possible consequences of the study. 4. Subject is pregnant or lactating 5. Subject has a known life-threatening hypersensitivity reaction to efgartigimod
Contact & Investigator
Marshall Mazepa, MD
PRINCIPAL INVESTIGATOR
University of Minnesota
Frequently Asked Questions
Who can join the NCT06831058 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying Immune-mediated Thrombotic Thrombocytopenic Purpura. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06831058 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT06831058 currently recruiting?
Yes, NCT06831058 is actively recruiting participants. Contact the research team at howar709@umn.edu for enrollment information.
Where is the NCT06831058 trial being conducted?
This trial is being conducted at Minneapolis, United States.
Who is sponsoring the NCT06831058 clinical trial?
NCT06831058 is sponsored by University of Minnesota. The principal investigator is Marshall Mazepa, MD at University of Minnesota. The trial plans to enroll 15 participants.