Efficacy, Safety and Pharmacokinetics of Rilzabrutinib in Patients With Warm Autoimmune Hemolytic Anemia (wAIHA)

Warm Autoimmune Hemolytic Anemia (wAIHA) Clinical Trial

Official title: A Multicenter, Open-label, Phase IIb Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Rilzabrutinib in Patients With Warm Autoimmune Hemolytic Anemia

Status Active, not recruiting

Clinical Trial Summary

This is a single group treatment, Phase 2, open-label, study to evaluate the efficacy, safety and pharmacokinetics of rilzabrutinib in adult patients with wAIHA.

All participants will receive rilzabrutinib orally. The screening period is up to 28 days, followed by a treatment period of 24 weeks for Part A. Participants who complete Part A and are deemed eligible for Part B will continue to receive the study medication for 52 weeks following the Last Patient In (LPI-Part B). There will be a 7-day safety follow-up period after receiving the last dose of study drug either in Part A (for those not eligible for Part B or early terminated) or Part B. The estimated total duration of the study is approximately 137 weeks (Parts A and B), including the follow-up period. For participants deemed ineligible for Part B, the total length of the study will be 29 weeks (Part A only), including screening and the follow-up period. In Part B, participants who temporarily stop rilzabrutinib treatment and maintain a durable response from W50 to W74, will have their EOS visit at Week

75. In this case, participation will be for 79 weeks including the screening period.

Clinical Trial Description

n/a

Related Conditions & MeSH terms

• Anemia
• Anemia, Hemolytic
• Anemia, Hemolytic, Autoimmune
• Hemolysis
• Warm Autoimmune Hemolytic Anemia (wAIHA)

• NCT number: NCT05002777
• Study type: Interventional
• Source: Sanofi
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: December 7, 2021
• Completion date: May 28, 2025