Recruiting
Phase 3

Rilzabrutinib

Sponsor:

Sanofi

Code:

NCT06975865

Conditions

Sickle Cell Disease

Eligibility Criteria

Sex: All

Age: 10 - 65

Healthy Volunteers: Not accepted

Interventions

Rilzabrutinib

Placebo

Study Details

Brief 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.

Conditions

Sickle Cell Disease

Study ID

NCT06975865

Start date

Aug 12, 2025

Status verified date

Jul, 2026

Completion date

Dec 29, 2028

Anticipated

Primary completion date

Jul 23, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 10 - 65

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

192 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Rilzabrutinib

Rilzabrutinib

placebo comparator: Placebo

Placebo

Interventions

Rilzabrutinib

Pharmaceutical form:Tablet -Route of administration:Oral

Placebo

Pharmaceutical form:Tablet -Route of administration:Oral

Primary outcome measure

  • Annualized rate of clinical VOC [ Time Frame: At Week 52 ]

Central Contacts and Locations

Central contacts

Trial Transparency email recommended (Toll free for US & Canada)

800-633-1610contact-us@sanofi.com

Locations

University of Alabama at Birmingham- Site Number : 8400003

Recruiting

Birmingham, Alabama, United States, 35233

Phoenix Children's Hospital- Site Number : 8400028

Recruiting

Phoenix, Arizona, United States, 85016

University of California San Francisco- Site Number : 8400040

Recruiting

Fresno, California, United States, 93701

Oncology & Hematology Associates of West Broward- Site Number : 8400029

Recruiting

Coral Springs, Florida, United States, 33065

Sylvester Comprehensive Cancer Center- Site Number : 8400020

Recruiting

Miami, Florida, United States, 33136

University of Illinois-Chicago - College of Medicine- Site Number : 8400054

Recruiting

Chicago, Illinois, United States, 60612

Indiana University Health Riley Hospital for Children- Site Number : 8400056

Recruiting

Indianapolis, Indiana, United States, 46202

Louisiana State University Health Sciences Center - Shreveport- Site Number : 8400037

Recruiting

Shreveport, Louisiana, United States, 71130

University of Maryland School of Medicine - Baltimore- Site Number : 8400041

Recruiting

Baltimore, Maryland, United States, 21201

University of Michigan Health System - Ann Arbor- Site Number : 8400035

Recruiting

Ann Arbor, Michigan, United States, 48109

Southern Specialty Research- Site Number : 8400059

Recruiting

Flowood, Mississippi, United States, 39232

Richmond University Medical Center- Site Number : 8400038

Recruiting

Staten Island, New York, United States, 10310

Montefiore Medical Center - Moses Campus- Site Number : 8400057

Recruiting

The Bronx, New York, United States, 10467

Prisma Health- Site Number : 8400051

Recruiting

Greenville, South Carolina, United States, 29605

Baylor College of Medicine- Site Number : 8400055

Recruiting

Houston, Texas, United States, 77030

VCU Massey Cancer Center: Dalton Oncology Clinic- Site Number : 8400012

Recruiting

Richmond, Virginia, United States, 23298

More Information

Sponsor

Sanofi

Last update posted

Jul 29, 2026

Last verified

Jul, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Sanofi on 2026-07-29.