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

Launched by SANOFI · May 8, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new medication called rilzabrutinib to see how effective and safe it is for people aged 10 to 65 years who have sickle cell disease (SCD). Sickle cell disease is a condition that affects the shape of red blood cells and can lead to painful episodes called vaso-occlusive crises (VOC). In this trial, participants will first take the medication or a placebo (a treatment with no active ingredient) for 52 weeks without knowing which one they received. After this period, those who complete the initial phase may continue in a longer phase of the study where everyone will know they are receiving rilzabrutinib.

To be eligible for this trial, participants must have a diagnosis of sickle cell disease and have experienced between 2 to 10 episodes of painful crises in the past year. They should not be currently taking certain medications like hydroxyurea or L-glutamine unless they have been on a stable dose for at least three months. Participants under 18 need permission from a parent or guardian to join. Throughout the study, participants will have regular visits to monitor their health. This trial is currently recruiting participants and aims to provide more information about how rilzabrutinib can help manage sickle cell disease.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Participants who have been diagnosed with SCD.
• • Participants who have had between ≥2 to ≤10 episodes of documented acute clinical VOC within 12 months of the screening visit.
• • 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.