A Pilot Study of Efgartigimod for Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP)

Launched by UNIVERSITY OF MINNESOTA · Feb 13, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a medication called efgartigimod to see if it can help patients with a rare autoimmune disorder known as immune-mediated thrombotic thrombocytopenic purpura (iTTP). This condition can cause serious problems like low platelet counts and organ damage due to blood clots. The trial aims to find out if efgartigimod can safely increase the levels of a protein called ADAMTS13, which helps regulate blood clotting, and if this increase can help prevent relapses of iTTP.

To be eligible for this study, participants need to be at least 18 years old and have a history of iTTP with specific test results showing low ADAMTS13 activity and the presence of related antibodies. They must also be in clinical remission, meaning their platelet count is normal for at least 90 days. Participants can expect to receive efgartigimod and will be monitored for changes in their ADAMTS13 levels during the treatment. The trial is not yet recruiting, but it is an important step in exploring a new potential treatment for patients facing this challenging condition.

Gender

ALL

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