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Rilzabrutinib for Sickle Cell Disease

This study is testing a drug called rilzabrutinib to see if it is safe and effective for people with sickle cell disease (SCD). Researchers want to find out if rilzabrutinib can reduce the number of pain crises, called vaso-occlusive crises (VOCs), that people with SCD experience. You might be able to join if you are between 10 and 65 years old, have been diagnosed with SCD, and have had between 2 and 10 VOCs in the past year. The study will compare rilzabrutinib to a placebo (a pill with no active medicine) over 52 weeks. The study is currently recruiting 192 participants.

Study design
This is a randomized, double-blind study, meaning participants will be randomly assigned to receive either rilzabrutinib or a placebo, and neither you nor your doctor will know which you are receiving. It plans to enroll 192 participants.
What's involved
You would participate in a 52-week double-blind treatment period. If you complete this part, you may be able to continue into an open-label long-term extension period.
Compensation
Not stated in the trial record.
Follow-up
The main goal of the study is measured at Week 52, but participants who complete the initial treatment period can continue into a long-term extension.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06975865

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

Recruiting
PHASE3Ages 10–65InterventionalTreatment
Sanofi
~192 participants
Updated 2026-07-29 on ClinicalTrials.gov
What's tested:RilzabrutinibPlacebo

At a glance

Recruiting sites
53 of 53 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Annualized rate of clinical VOC
Measured over At Week 52

Conditions

Sickle Cell Disease

Explore related trials

Sickle Cell Disease

Where it's being run

53 sites across 35 states
France4
Israel4
Belgium3
Greece3
Turkey (Türkiye)3
Florida2
New York2
Brazil2

Who to contact

Trial Transparency email recommended (Toll free for US & Canada)
[email protected]
800-633-1610 · ext. option 6
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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

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.

What this trial measures

  • Annualized rate of clinical VOCAt Week 52

    Calculated from total number of clinical VOC incidents and total number of days during the observation period