NCT05468320

Caplacizumab and Immunosuppressive Therapy Without Firstline Therapeutic Plasma Exchange in Adults With Immune-mediated Thrombotic Thrombocytopenic Purpura

An Open-label, Single-arm, Multicenter Study to Evaluate the Efficacy and Safety of Caplacizumab and Immunosuppressive Therapy Without Firstline Therapeutic Plasma Exchange in Adults With Immune-mediated Thrombotic Thrombocytopenic Purpura

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 51 (actual)
• Sponsor: Sanofi · Industry
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

This is a single group, treatment, Phase 3, open-label, single-arm study to evaluate the efficacy and safety of caplacizumab and immunosuppressive therapy (IST) without firstline therapeutic plasma exchange (TPE) with primary endpoint of remission in male and female participants aged 18 to 80 years with immune-mediated thrombotic thrombocytopenic purpura (iTTP). The anticipated study duration per participant without a recurrence while on therapy is maximum 24 weeks (ie, approximately 1 day for screening + maximum 12 weeks of treatment for the presenting episode + 12 weeks of follow-up). Participants will have daily assessments during hospitalization and weekly visits for assessments during ongoing treatment with caplacizumab and IST. There will be 3 outpatient visits for assessments during the follow-up period. There will be two additional follow-up visits for participants who do not have ADAMTS13 activity levels of ≥50% at the time of caplacizumab discontinuation.

Detailed description

The anticipated study duration per participant with the presenting episode therefore is a maximum of about 24 weeks (ie, 1 day of screening + maximum 12 weeks of treatment for the presenting episode + 12 weeks of follow-up).

Conditions

• Thrombotic Thrombocytopenic Purpura

Interventions

Type	Name	Description
DRUG	Caplacizumab	Lyophilized powder for solution for injection.
DRUG	Corticosteroids	Solution for injection or Tablet
BIOLOGICAL	anti-CD20 antibody	Solution for injection

Timeline

• Start date: 2022-11-21
• Primary completion: 2024-12-26
• Completion: 2024-12-26
• First posted: 2022-07-21
• Last updated: 2025-12-30
• Results posted: 2025-12-30

Locations

27 sites across 11 countries: United States, Belgium, Canada, Czechia, France, Germany, Italy, Japan, Netherlands, Spain, United Kingdom

Regulatory

• FDA-regulated drug study