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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 France tracker
Sponsor
Phase
Phase 3
Started
2025-08-12
Last updated
2026-10-15

Condition(s) studied

Sickle Cell Disease

Investigational drug(s) / intervention(s)

Rilzabrutinib →Placebo

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)

  • Annualized rate of clinical VOC — At Week 52
    Calculated from total number of clinical VOC incidents and total number of days during the observation period

Trial sites (63)

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 Clovis California Recruiting
David Geffen School of Medicine at UCLA- Site Number : 8400032 Los Angeles California Recruiting
MedStar Georgetown University Hospital- Site Number : 8400036 Washington D.C. District of Columbia 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
Oregon Health & Science University (OHSU)- Site Number : 8400043 Portland Oregon Recruiting
Prisma Health- Site Number : 8400051 Greenville South Carolina Recruiting
University of Tennessee Health Science Center- Site Number : 8400045 Memphis Tennessee Recruiting
Baylor College of Medicine- Site Number : 8400055 Houston Texas Recruiting
UT Physicians - Memorial Hermann- Site Number : 8400052 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
Investigational Site Number : 0560004 Liège Belgium Recruiting
Hospital Santa Izabel- Site Number : 0760006 Salvador Estado de Bahia Recruiting
Fundação Bahiana de Infectologia- Site Number : 0760009 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
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 : 2760005 Berlin Germany 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

+ 23 more sites — see the full list on the official registry below.

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 France