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RECRUITING

NCT06975865

PHASE3

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

Sponsor: Sanofi

View on ClinicalTrials.gov

Summary

This is a 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 (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.

Official title: A 52-week, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group, Group Sequential Study to Evaluate the Efficacy and Safety of Rilzabrutinib in Patients Aged 10 to 65 Years With Sickle-cell Disease

Key Details

Gender

All

Age Range

10 Years - 65 Years

Study Type

INTERVENTIONAL

Enrollment

192

Start Date

2025-08-12

Completion Date

2028-12-29

Last Updated

2026-04-02

Healthy Volunteers

Conditions

Sickle Cell Disease

Interventions

DRUG

Rilzabrutinib

Pharmaceutical form:Tablet -Route of administration:Oral

DRUG

Placebo

Pharmaceutical form:Tablet -Route of administration:Oral

Inclusion Criteria: * Participants who have been diagnosed with SCD. * Participants who have had between ≥2 to ≤10 episodes of documented acute clinical VOC within 12 months of the screening visit. * 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.

Locations (53)

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

Fresno, California, 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 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

Baylor College of Medicine- Site Number : 8400055

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

Hospital Santa Izabel- Site Number : 0760006

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

Pontifícia Universidade Católica do Rio de Janeiro- Site Number : 0760009

Rio de Janeiro, 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 : 2760002

Essen, Germany

Investigational Site Number : 2760004

Stuttgart, Germany

Investigational Site Number : 2880004

Kintampo, Ghana

Investigational Site Number : 2880002

Navrongo, Ghana

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

Azienda Ospedaliero Universitaria Careggi SOD Ematologia-Site Number : 3800006

Florence, Firenze, Italy

Investigational Site Number : 3800004

Milan, Milano, Italy

Azienda Ospedaliera Universitaria, Università della Campania "Luigi Vanvitelli" Napoli-Site Number : 3800005

Naples, Napoli, Italy

IRCCS Ospedale Pediatrico Bambino Gesù-Site Number : 3800001

Rome, Roma, Italy

Azienda Ospedaliera Universitaria San Luigi Gonzaga, SSD Microcitemie Malattie Rare Ematologiche-Site Number : 3800007

Orbassano, Torino, Italy

Azienda Ospedaliera Ospedali Riuniti Villa Sofia - Cervello-Site Number : 3800002

Palermo, Italy

Centro Ricerche Cliniche Verona s.r.l. presso Ospedale G.B. Rossi Borgo Roma-Site Number : 3800003

Verona, Italy

Investigational Site Number : 5280002

Rotterdam, Netherlands

Investigational Site Number : 5120001

Muscat, Oman

Investigational Site Number : 7240002

Madrid, Spain

Investigational Site Number : 7240001

Madrid, Spain

Investigational Site Number : 8340003

Mwanza, Tanzania

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