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

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• University of Alabama at Birmingham- Site Number : 8400003
  Birmingham, Alabama, United States Recruiting
• Phoenix Children's Hospital- Site Number : 8400028
  Phoenix, Arizona, United States Recruiting
• University of California San Francisco- Site Number : 8400040
  Clovis, California, United States Recruiting
• David Geffen School of Medicine at UCLA- Site Number : 8400032
  Los Angeles, California, United States Recruiting
• MedStar Georgetown University Hospital- Site Number : 8400036
  Washington D.C., District of Columbia, United States Recruiting
• Oncology & Hematology Associates of West Broward- Site Number : 8400029
  Coral Springs, Florida, United States Recruiting

+13 more sites. See ClinicalTrials.gov for the full list.

Study contacts

• Study coordinator: Trial Transparency email recommended (Toll free for US & Canada)
• Email: contact-us@sanofi.com
• Phone: 800-633-1610

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Hematopoietic stem cell editing to treat sickle cell disease

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.