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Clinical Trials in the UK / NCT06975865
Recruiting Phase 3

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

NCT06975865 · tracked via the Priya Life Science UK tracker
Sponsor
Sanofi
Phase
Phase 3
Started
2025-08-12
Last updated
2026-07-29

Condition(s) studied

Sickle Cell Disease

Investigational drug(s) / intervention(s)

RilzabrutinibPlacebo

Rilzabrutinib: Pharmaceutical form:Tablet -Route of administration:Oral

Placebo: Pharmaceutical form:Tablet -Route of administration:Oral

Study 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.

Eligibility

Sex
ALL
Min age
10 Years
Max age
65 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (53)

FacilityCityRegionStatus
University of Alabama at Birmingham- Site Number : 8400003 Birmingham Alabama Recruiting
Phoenix Children's Hospital- Site Number : 8400028 Phoenix Arizona Recruiting
University of California San Francisco- Site Number : 8400040 Fresno California Recruiting
Oncology & Hematology Associates of West Broward- Site Number : 8400029 Coral Springs Florida Recruiting
Sylvester Comprehensive Cancer Center- Site Number : 8400020 Miami Florida Recruiting
University of Illinois-Chicago - College of Medicine- Site Number : 8400054 Chicago Illinois Recruiting
Indiana University Health Riley Hospital for Children- Site Number : 8400056 Indianapolis Indiana Recruiting
Louisiana State University Health Sciences Center - Shreveport- Site Number : 8400037 Shreveport Louisiana Recruiting
University of Maryland School of Medicine - Baltimore- Site Number : 8400041 Baltimore Maryland Recruiting
University of Michigan Health System - Ann Arbor- Site Number : 8400035 Ann Arbor Michigan Recruiting
Southern Specialty Research- Site Number : 8400059 Flowood Mississippi Recruiting
Richmond University Medical Center- Site Number : 8400038 Staten Island New York Recruiting
Montefiore Medical Center - Moses Campus- Site Number : 8400057 The Bronx New York Recruiting
Prisma Health- Site Number : 8400051 Greenville South Carolina Recruiting
Baylor College of Medicine- Site Number : 8400055 Houston Texas Recruiting
VCU Massey Cancer Center: Dalton Oncology Clinic- Site Number : 8400012 Richmond Virginia Recruiting
Investigational Site Number : 0560003 Brussels Belgium Recruiting
Investigational Site Number : 0560002 Brussels Belgium Recruiting
Investigational Site Number : 0560001 Leuven Belgium Recruiting
Hospital Santa Izabel- Site Number : 0760006 Salvador Estado de Bahia Recruiting
Universidade Federal de Goias- Site Number : 0760002 Goiânia Goiás Recruiting
Fundação Faculdade Regional de Medicina de São José do Rio Preto- Site Number : 0760001 São José do Rio Preto São Paulo Recruiting
Pontifícia Universidade Católica do Rio de Janeiro- Site Number : 0760009 Rio de Janeiro Brazil Recruiting
Hospital Samaritano De Sao Paulo- Site Number : 0760005 São Paulo Brazil Recruiting
Investigational Site Number : 2500002 Créteil France Recruiting
Investigational Site Number : 2500005 Marseille France Recruiting
Investigational Site Number : 2500001 Paris France Recruiting
Investigational Site Number : 2500004 Toulouse France Recruiting
Investigational Site Number : 2760002 Essen Germany Recruiting
Investigational Site Number : 2760004 Stuttgart Germany Recruiting
Investigational Site Number : 3000001 Athens Greece Recruiting
Investigational Site Number : 3000003 Athens Greece Recruiting
Investigational Site Number : 3000002 Pátrai Greece Recruiting
Investigational Site Number : 3760001 Afula Israel Recruiting
Investigational Site Number : 3760002 Afula Israel Recruiting
Investigational Site Number : 3760005 Haifa Israel Recruiting
Investigational Site Number : 3760006 Haifa Israel Recruiting
Azienda Ospedaliero Universitaria Careggi SOD Ematologia-Site Number : 3800006 Florence Firenze Recruiting
Investigational Site Number : 3800004 Milan Milano Recruiting
Azienda Ospedaliera Universitaria, Università della Campania "Luigi Vanvitelli" Napoli-Site Number : 3800005 Naples Napoli Recruiting

+ 13 more sites — see the full list on the official registry below.

More Sanofi trials in the UK

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06975865 on ClinicalTrials.gov ↗ ← All trials in the UK