Efgartigimod treatment for immune-mediated thrombotic thrombocytopenic purpura

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

This study is testing a new treatment called efgartigimod to see if it can help people with immune-mediated thrombotic thrombocytopenic purpura stay healthy and prevent relapses.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 15 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: University of Minnesota (other)
• Drugs / interventions: rituximab
• Locations: 1 site (Minneapolis, Minnesota)
• Trial ID: NCT06831058 on ClinicalTrials.gov

What this trial studies

This pilot study investigates the use of efgartigimod, a novel therapeutic agent, for patients with immune-mediated thrombotic thrombocytopenic purpura (iTTP), a rare autoimmune disorder. The study aims to assess the impact of efgartigimod on ADAMTS13 activity levels, which are crucial for coagulation and often deficient in iTTP patients. Participants must have a history of iTTP and be in clinical remission, with specific criteria regarding their ADAMTS13 activity. The study is designed to evaluate the safety and efficacy of this treatment approach in preventing relapses and associated complications.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with a documented history of iTTP who are currently in clinical remission.

Not a fit: Patients who are not in remission or have not met the specific ADAMTS13 activity criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly reduce the risk of relapse and complications such as ischemic stroke in patients with iTTP.

How similar studies have performed: While there have been small studies on rituximab for iTTP, the use of efgartigimod represents a novel approach that has not been extensively tested in this context.

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

Where this trial is running

Minneapolis, Minnesota

• University of Minnesota — Minneapolis, Minnesota, United States (RECRUITING)

Study contacts

• Principal investigator: Marshall Mazepa, MD — University of Minnesota
• Study coordinator: Diondra Howard

Conditions: Immune-mediated Thrombotic Thrombocytopenic Purpura