Rilzabrutinib for Sickle Cell Disease

(LIBRA Trial)

Recruiting at 1 trial location

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Sanofi

Must be taking: Hydroxyurea, L-glutamine

Must not be taking: Crizanlizumab, Voxelotor

Disqualifiers: Lymphoma, Leukemia, Stroke, HIV, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: Unknown

Duration: 52 weeks

192 Participants Needed

Trial Summary

What is the purpose of this trial?

This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled, parallel-group, 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, 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

Eligibility Criteria

This trial is for patients aged 10 to 65 with Sickle-cell Disease who've had 2-10 acute clinical VOC episodes in the past year. They should have an ECOG status of ≤2, use contraception, and either not be on hydroxyurea/L-glutamine or have been on a stable dose for at least 6 months.

Inclusion Criteria

I have been diagnosed with sickle cell disease.

I've had 2 to 10 episodes of acute pain crises in the last year.

I can do most activities, but I am unable to work.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either rilzabrutinib or placebo during a 52-week double-blind period

52 weeks

Weekly visits based on the Schedule of Assessments

Open-label extension (LTE)

Participants who complete the double-blind period may continue to receive rilzabrutinib in an open-label extension

52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Rilzabrutinib

Trial Overview The study tests Rilzabrutinib's effectiveness and safety against a placebo in SCD patients over a year. It starts with a double-blind phase where neither doctors nor participants know who gets the drug or placebo, followed by an open-label period where everyone knows.

Participant Groups

Group I: Rilzabrutinib Experimental Treatment 1 Intervention

Rilzabrutinib

Group II: Placebo Placebo Group 1 Intervention

Placebo

Who Is Running the Clinical Trial?

Sanofi

Lead Sponsor

Trials

2,246

Recruited

4,085,000+

Paul Hudson

Sanofi

Chief Executive Officer since 2019

Degree in Economics from Manchester Metropolitan University

Christopher Corsico

Sanofi

Chief Medical Officer

MD from Cornell University, MPH in Chronic Disease Epidemiology from Yale University