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

Inclusion Criteria:

• Participants who have been diagnosed with SCD.

• Participants who have had between ≥2 to ≤10 episodes of documented acute clinicalVOC within 12 months of the screening visit.

• Participants who are either not on hydroxyurea and/or L-glutamine at the ScreeningVisit and does not plan to receive them during the course of the study or hasreceived HU and/or L-glutamine for a minimum of 6 months. Participants onhydroxyurea 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 tocontinue at the same weight-based dose level for the duration of the study, exceptfor 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 regulationsregarding the methods of contraception for those participating in clinical studies.

• For participants ≥10 to <18 years of age: the parent(s)/legal guardian(s) mustprovide written informed consent prior to any study-related procedures beingperformed.

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 withinthe past 5 years except for basal cell or squamous epithelial carcinomas of the skinthat have been resected with no evidence of metastatic disease for the past 3 years.

• Clinically relevant cardiac abnormality, in the opinion of the Investigator orelectrocardiogram (ECG) findings.

• Participants with history of stroke, or history of abnormal transcranial doppler.

• Participants with uncontrolled or active HBV infection and/or HCV infectionincluding 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/orvoxelotor (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.