NCT06975865

The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease

A 52-week, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group, Flexible-adaptive, Group Sequential Study to Evaluate the Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 192 (estimated)
• Sponsor: Sanofi · Industry
• Sex: All
• Age: 10 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, flexible-adaptive, group-sequential study (Part A), followed by an open-label LTE period (Part B) to investigate the efficacy, and safety of rilzabrutinib in participants with sickle-cell disease (SCD). Study details include: * Study duration: a 52-week double-blind period (Part A), followed by an open-label LTE period (Part B). Double-blind period has two parts, 50% (adult only) until the interim analysis (a proof-concept part analogous to a phase 2b study), and 50% (adult and children) after the interim analysis. Only the participants who complete double-blind treatment period (Part A) are eligible to continue to the LTE period. The duration of the LTE period (Part B) will be from the first-participant-in (FPI)-LTE (Part B) until the last participant who enters the LTE has completed 52 weeks. * Treatment duration: 52-week double-blind period (Part A); LTE period (Part B) from the (FPI until the last participant who enters the LTE has completed 52 weeks. * Visit frequency: Week visits based on the Schedule of Assessments.

Conditions

• Sickle Cell Disease

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: 2025-08-12
• Primary completion: 2027-07-23
• Completion: 2028-12-29
• First posted: 2025-05-16
• Last updated: 2026-04-13

Locations

54 sites across 15 countries: United States, Belgium, Brazil, France, Germany, Ghana, Greece, Israel, Italy, Netherlands, Oman, Spain, Tanzania, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study