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

Recruiting now · Phase 3 · Has a placebo group

Conditions studied: Sickle Cell Disease

In brief

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.

Key facts

Study ID

NCT06975865

Run by

Sanofi

People needed

192

Starts

2025-08-12

Expected to finish

2028-12-29

Last updated by the study team

2026-07-29

Who can join

Age: 10 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Participants who have been diagnosed with SCD.
• Participants who have had between ≥2 and ≤10 episodes of documented clinical VOC within 12 months of the screening events.
• Participants who are either not on hydroxyurea and/or L-glutamine at the Screening Visit and does not plan to receive them during the course of the study or has received HU and/or L-glutamine for a minimum of 6 months. Participants on hydroxyurea and/or L-glutamine must have been on a stable weight-based dose level (mg/kg) for at least 3 months prior to the Screening Visit, with the intent to continue at the same weight-based dose level for the duration of the study, except for safety reasons.
• Participants with Eastern Cooperative Oncology Group (ECOG) performance status grade 2 or lower.
• Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
• For participants ≥10 to <18 years of age: the parent(s)/legal guardian(s) must provide written informed consent prior to any study-related procedures being performed.

You may not qualify if…

• Participants are excluded from the study if any of the following criteria apply: Participants with medical history of lymphoma, leukemia, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for the past 3 years.
• Clinically relevant cardiac abnormality, in the opinion of the Investigator or electrocardiogram (ECG) findings.
• Participants with history of stroke, or history of abnormal transcranial doppler.
• Participants with uncontrolled or active HBV infection and/or HCV infection including those receiving antiviral therapy at the time of screening.
• HIV infection.
• A history of active or latent tuberculosis (TB)
• Positive COVID-19 molecular test.
• Participant is taking or has received crizanlizumab (ADAKVEO®) within 90 days and/or voxelotor (OXBRYTA®) within 30 days prior to the Screening visit.
• The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Where it is running

• Phoenix Children's Hospital- Site Number : 8400028 — Phoenix, Arizona, United States (enrolling)
• University of California San Francisco- Site Number : 8400040 — Fresno, California, United States (enrolling)
• Oncology & Hematology Associates of West Broward- Site Number : 8400029 — Coral Springs, Florida, United States (enrolling)
• Sylvester Comprehensive Cancer Center- Site Number : 8400020 — Miami, Florida, United States (enrolling)
• University of Illinois-Chicago - College of Medicine- Site Number : 8400054 — Chicago, Illinois, United States (enrolling)
• Indiana University Health Riley Hospital for Children- Site Number : 8400056 — Indianapolis, Indiana, United States (enrolling)
• Louisiana State University Health Sciences Center - Shreveport- Site Number : 8400037 — Shreveport, Louisiana, United States (enrolling)
• University of Maryland School of Medicine - Baltimore- Site Number : 8400041 — Baltimore, Maryland, United States (enrolling)
• University of Michigan Health System - Ann Arbor- Site Number : 8400035 — Ann Arbor, Michigan, United States (enrolling)
• Southern Specialty Research- Site Number : 8400059 — Flowood, Mississippi, United States (enrolling)
• Richmond University Medical Center- Site Number : 8400038 — Staten Island, New York, United States (enrolling)
• Montefiore Medical Center - Moses Campus- Site Number : 8400057 — The Bronx, New York, United States (enrolling)
• Prisma Health- Site Number : 8400051 — Greenville, South Carolina, United States (enrolling)
• Baylor College of Medicine- Site Number : 8400055 — Houston, Texas, United States (enrolling)
• VCU Massey Cancer Center: Dalton Oncology Clinic- Site Number : 8400012 — Richmond, Virginia, United States (enrolling)
• Investigational Site Number : 0560003 — Brussels, Belgium (enrolling)
• Investigational Site Number : 0560002 — Brussels, Belgium (enrolling)
• Investigational Site Number : 0560001 — Leuven, Belgium (enrolling)
• Hospital Santa Izabel- Site Number : 0760006 — Salvador, Estado de Bahia, Brazil (enrolling)
• Universidade Federal de Goias- Site Number : 0760002 — Goiânia, Goiás, Brazil (enrolling)
• Fundação Faculdade Regional de Medicina de São José do Rio Preto- Site Number : 0760001 — São José do Rio Preto, São Paulo, Brazil (enrolling)
• Pontifícia Universidade Católica do Rio de Janeiro- Site Number : 0760009 — Rio de Janeiro, Brazil (enrolling)
• Hospital Samaritano De Sao Paulo- Site Number : 0760005 — São Paulo, Brazil (enrolling)
• Investigational Site Number : 2500002 — Créteil, France (enrolling)
• University of Alabama at Birmingham- Site Number : 8400003 — Birmingham, Alabama, United States (enrolling)

Full record on ClinicalTrials.gov

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