Efgartigimod treatment for immune-mediated thrombotic thrombocytopenic purpura

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• University of Minnesota
  Minneapolis, Minnesota, United States Recruiting

Study contacts

• Principal investigator: Marshall Mazepa, MD — University of Minnesota
• Study coordinator: Diondra Howard
• Email: howar709@umn.edu
• Phone: 651-208-7476

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Long-term outcomes for people with immune TTP in the French national TMA cohort

  This project uses past medical records to see long-term health outcomes and complications in people in France who had immune thrombotic thrombocytopenic purpura (iTTP).

  Recruiting Assistance Publique - Hôpitaux de Paris · Paris · NCT07205861

• cTTP patients treated with recombinant ADAMTS13: what happened after the early access program

  This study looks at people with congenital TTP who received recombinant ADAMTS13 to see how treatment changes their health before and after use.

  Recruiting Takeda · Cologne, North Rhine-Westphalia and 7 other locations · NCT07429942

• Adzynma in people with congenital TTP: a look at safety and antibody risk

  This study checks whether people with congenital TTP who have already received Adzynma develop neutralizing antibodies against Adzynma within six months of starting treatment.

  Recruiting at 6 sites Takeda · Vienna and 6 other locations · NCT07353099

• Understanding the immune response in Thrombotic Thrombocytopenic Purpura

  This study is trying to understand how certain immune cells behave in people with Thrombotic Thrombocytopenic Purpura to see if they can help predict relapses and improve treatment.

  Recruiting University Hospital, Rouen · Rouen · NCT06945861

• Survey of a treatment for congenital thrombotic thrombocytopenic purpura

  This study is testing a new treatment called recombinant ADAMTS13 to see if it helps people with congenital thrombotic thrombocytopenic purpura feel better and stay healthy over 18 months.

  Recruiting Takeda · Tokyo, Tokyo · NCT06441578

• Registry for patients with immune-mediated thrombotic thrombocytopenic purpura in Italy

  This study is tracking patients with immune-mediated thrombotic thrombocytopenic purpura in Italy to see how the disease affects them over time and what their treatment outcomes are.

  Recruiting Fondazione Luigi Villa · Milan, MI · NCT06376786

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.