Efgartigimod for Thrombotic Thrombocytopenic Purpura

Overseen By Diondra Howard

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: University of Minnesota

Disqualifiers: Pregnancy, Lactation, cTTP, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 13

Duration: 60 days

15 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Research Team

Marshall Mazepa, MD

Principal Investigator

University of Minnesota

Eligibility Criteria

This trial is for individuals who have had immune-mediated Thrombotic thrombocytopenic purpura (iTTP) and are currently in remission with specific levels of ADAMTS13 activity. They must be at least 6 months past their last rituximab or similar treatment, not have changed oral immunosuppressant doses recently, and agree to use effective birth control.

Inclusion Criteria

I have signed the consent form for the trial.

I am not pregnant and agree to use effective birth control during the study.

My ADAMTS13 activity levels were between 30% and 70% on two tests at least a week apart.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive efgartigimod therapy to increase ADAMTS13 activity

60 days

Regular visits for treatment administration and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

8 weeks

Follow-up visits to assess ADAMTS13 activity and antibody titers

Treatment Details

Interventions

• Efgartigimod

Trial Overview The study tests efgartigimod's ability to increase ADAMTS13 activity temporarily in iTTP patients. It aims to show that this drug can safely prevent relapses by reducing harmful antibodies. Participants will receive efgartigimod therapy and then be monitored for changes in ADAMTS13 levels after stopping the treatment.

Participant Groups

Group I: iTTP patients Experimental Treatment 1 Intervention

participants with a history of iTTP in clinical remission but with ADAMTS13 deficiency (\>30% but \< 70% activity)

Who Is Running the Clinical Trial?

University of Minnesota

Lead Sponsor

Trials

1,459

Recruited

1,623,000+