RECRUITING Phase 3 ClinicalTrials.gov

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

Sponsor: Sanofi • ClinicalTrials.gov ID: NCT06975865

Registry updated: 2026-10-15

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Description

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.

Interventions

Rilzabrutinib (Drug)
Pharmaceutical form:Tablet -Route of administration:Oral
Placebo (Drug)
Pharmaceutical form:Tablet -Route of administration:Oral

Eligibility criteria

Inclusion Criteria: * 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. Exclusion Criteria: * 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.

Conditions

Sickle Cell Disease

Locations

University of Alabama at Birmingham- Site Number : 8400003 — Birmingham, Alabama, United States
Phoenix Children's Hospital- Site Number : 8400028 — Phoenix, Arizona, United States
University of California San Francisco- Site Number : 8400040 — Clovis, California, United States
David Geffen School of Medicine at UCLA- Site Number : 8400032 — Los Angeles, California, United States
MedStar Georgetown University Hospital- Site Number : 8400036 — Washington D.C., District of Columbia, United States
Oncology & Hematology Associates of West Broward- Site Number : 8400029 — Coral Springs, Florida, United States
Sylvester Comprehensive Cancer Center- Site Number : 8400020 — Miami, Florida, United States
University of Illinois-Chicago - College of Medicine- Site Number : 8400054 — Chicago, Illinois, United States
Indiana University Health Riley Hospital for Children- Site Number : 8400056 — Indianapolis, Indiana, United States
Louisiana State University Health Sciences Center - Shreveport- Site Number : 8400037 — Shreveport, Louisiana, United States
University of Maryland School of Medicine - Baltimore- Site Number : 8400041 — Baltimore, Maryland, United States
University of Michigan Health System - Ann Arbor- Site Number : 8400035 — Ann Arbor, Michigan, United States
Southern Specialty Research- Site Number : 8400059 — Flowood, Mississippi, United States
Richmond University Medical Center- Site Number : 8400038 — Staten Island, New York, United States
Montefiore Medical Center - Moses Campus- Site Number : 8400057 — The Bronx, New York, United States
Oregon Health & Science University (OHSU)- Site Number : 8400043 — Portland, Oregon, United States
Prisma Health- Site Number : 8400051 — Greenville, South Carolina, United States
University of Tennessee Health Science Center- Site Number : 8400045 — Memphis, Tennessee, United States
Baylor College of Medicine- Site Number : 8400055 — Houston, Texas, United States
UT Physicians - Memorial Hermann- Site Number : 8400052 — Houston, Texas, United States
VCU Massey Cancer Center: Dalton Oncology Clinic- Site Number : 8400012 — Richmond, Virginia, United States
Investigational Site Number : 0560003 — Brussels, Belgium
Investigational Site Number : 0560002 — Brussels, Belgium
Investigational Site Number : 0560001 — Leuven, Belgium
Investigational Site Number : 0560004 — Liège, Belgium
Hospital Santa Izabel- Site Number : 0760006 — Salvador, Estado de Bahia, Brazil
Fundação Bahiana de Infectologia- Site Number : 0760009 — Salvador, Estado de Bahia, Brazil
Universidade Federal de Goias- Site Number : 0760002 — Goiânia, Goiás, Brazil
Fundação Faculdade Regional de Medicina de São José do Rio Preto- Site Number : 0760001 — São José do Rio Preto, São Paulo, Brazil
Hospital Samaritano De Sao Paulo- Site Number : 0760005 — São Paulo, Brazil
Investigational Site Number : 2500002 — Créteil, France
Investigational Site Number : 2500005 — Marseille, France
Investigational Site Number : 2500001 — Paris, France
Investigational Site Number : 2500004 — Toulouse, France
Investigational Site Number : 2760005 — Berlin, Germany
Investigational Site Number : 2760002 — Essen, Germany
Investigational Site Number : 2760004 — Stuttgart, Germany
Investigational Site Number : 3000001 — Athens, Greece
Investigational Site Number : 3000003 — Athens, Greece
Investigational Site Number : 3000002 — Pátrai, Greece
Investigational Site Number : 3760001 — Afula, Israel
Investigational Site Number : 3760002 — Afula, Israel
Investigational Site Number : 3760005 — Haifa, Israel
Investigational Site Number : 3760006 — Haifa, Israel
Investigational Site Number : 3800006 — Florence, Firenze, Italy
Investigational Site Number : 3800008 — Genoa, Genova, Italy
Investigational Site Number : 3800004 — Milan, Milano, Italy
Investigational Site Number : 3800005 — Naples, Napoli, Italy
Investigational Site Number : 3800001 — Rome, Roma, Italy
Investigational Site Number : 3800007 — Orbassano, Torino, Italy
Investigational Site Number : 3800002 — Palermo, Italy
Investigational Site Number : 3800003 — Verona, Italy
Investigational Site Number : 5280002 — Rotterdam, Netherlands
Investigational Site Number : 5120001 — Muscat, Oman
Investigational Site Number : 6820003 — Riyadh, Saudi Arabia
Investigational Site Number : 7240001 — Madrid, Madrid, Comunidad de, Spain
Investigational Site Number : 7240002 — Madrid, Spain
Investigational Site Number : 7920001 — Adana, Turkey (Türkiye)
Investigational Site Number : 7920002 — Adana, Turkey (Türkiye)
Investigational Site Number : 7920003 — Mersin, Turkey (Türkiye)
Investigational Site Number : 8260002 — London, Harrow, United Kingdom
Investigational Site Number : 8260001 — London, London, City of, United Kingdom
Investigational Site Number : 8260005 — Manchester, United Kingdom

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Official Source

Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

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