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Evaluating Rilzabrutinib for Sickle Cell Disease in Patients Aged 10 to 65

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

Phase 3 Interventional Sanofi · NCT06975865

This study is testing a new medication called rilzabrutinib to see if it can help people aged 10 to 65 with sickle cell disease have fewer pain crises and improve their overall health.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment192 (estimated)
Ages10 Years to 65 Years
SexAll
SponsorSanofi Industry-sponsored
Drugs / interventionscrizanlizumab, rilzabrutinib
Locations54 sites (Birmingham, Alabama and 53 other locations)
Trial IDNCT06975865 on ClinicalTrials.gov

What this trial studies

This Phase 3 clinical trial investigates the efficacy and safety of rilzabrutinib in patients aged 10 to 65 years with sickle cell disease. The study consists of a 52-week double-blind period, where participants are randomly assigned to receive either the drug or a placebo, followed by an open-label long-term extension period for those who complete the initial phase. The trial aims to assess the frequency of vaso-occlusive crises and overall health outcomes in participants. It includes both adults and children, with specific eligibility criteria regarding previous treatments and health status.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 10 to 65 years diagnosed with sickle cell disease who have experienced between 2 to 10 vaso-occlusive crises in the past year.

Not a fit: Patients who are currently on hydroxyurea or L-glutamine and do not meet the study's specific treatment criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly reduce the frequency of painful crises and improve the quality of life for patients with sickle cell disease.

How similar studies have performed: Previous studies have shown promise in treating sickle cell disease with novel therapies, but this specific approach with rilzabrutinib is relatively novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
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.

Where this trial is running

Birmingham, Alabama and 53 other locations

+4 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Sickle Cell Disease
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.