NCT07086976

A Study to Investigate the Efficacy, Safety, and Pharmacokinetics of Oral Rilzabrutinib Compared With Placebo in Participants 18 Years of Age and Older With Warm Autoimmune Hemolytic Anemia

A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study With an Open-label Period and Long-term Extension to Assess the Efficacy and Safety of Rilzabrutinib in Participants With Warm Autoimmune Hemolytic Anemia (wAIHA)

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 90 (estimated)
• Sponsor: Sanofi · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a parallel-group, Phase 3, double-blind, 2-arm study to investigate the efficacy, safety, PK and PD of oral rilzabrutinib in achieving durable Hb response (DHR) compared with placebo in approximately 90 male and female participants ≥ 18 years of age with a confirmed diagnosis of primary wAIHA. Following a 4-week screening period, eligible participants will be randomized in a 2:1 ratio to receive rilzabrutinib or placebo in primary analysis period (PAP) for a duration of up to 24 weeks. All participants who completed PAP will then continue in open-label period (OLP) to receive rilzabrutinib for a duration of 28 weeks. Upon the completion of OLP, only participants who demonstrate Hb increase during the last 8 weeks of OLP per specified criteria in the protocol will be eligible to continue in long-term extension (LTE) of the study. The duration of the LTE period will be from the first-participant-in (FPI)-LTE until the last participant completes 52 weeks in LTE. The safety follow-up period of this study following treatment completion or discontinuation will be 2 weeks.

Conditions

• Autoimmune Haemolytic Anaemia

Interventions

Type	Name	Description
DRUG	rilzabrutinib	Pharmaceutical form:tablet-Route of administration:oral
DRUG	placebo	Pharmaceutical form:tablet-Route of administration:oral

Timeline

• Start date: 2025-08-18
• Primary completion: 2028-05-31
• Completion: 2029-12-26
• First posted: 2025-07-25
• Last updated: 2026-03-31

Locations

91 sites across 17 countries: United States, Argentina, Austria, Brazil, China, Czechia, Denmark, Germany, Greece, Hungary, Israel, Italy, Japan, Netherlands, Poland, Spain, Sweden

Regulatory

• FDA-regulated drug study