The Efficacy and Safety of Rilzabrutinib in Patients Aged 10 to 65 Years With Sickle-cell Disease (LIBRA)
Investigating the Efficacy and Safety of an Investigational Medication in Sickle-cell Disease
Study Overview
This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled, parallel-group, group-sequential study (Part A), followed by an open-label LTE period (Part B) to investigate the efficacy, and safety of rilzabrutinib in participants with sickle-cell disease (SCD).
Study details include:
- Study duration: a 52-week double-blind period (Part A), followed by an open-label LTE period (Part B). Double-blind period has two parts, 50% (adult only) until the interim analysis, and 50% (adult and children) after the interim analysis. Only the participants who complete double-blind treatment period (Part A) are eligible to continue to the LTE period. The duration of the LTE period (Part B) will be from the first-participant-in (FPI)-LTE (Part B) until the last participant who enters the LTE has completed 52 weeks.
- Treatment duration: 52-week double-blind period (Part A); LTE period (Part B) from the (FPI until the last participant who enters the LTE has completed 52 weeks
- Visit frequency: Week visits based on the Schedule of Assessments
Eligibility Criteria
You may be eligible for this study if you meet the following criteria:
- Conditions: Sickle Cell Disease
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Age: 10 years - 65 years
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Gender: All
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.
This study investigates the efficacy and safety of an investigational medication in participants with sickle-cell disease (SCD). Sickle-cell disease is a genetic condition where red blood cells, which normally look like discs, become crescent-shaped. These sickle-shaped cells can block blood flow and cause pain and other serious problems. The study is divided into two phases: a double-blind phase where neither participants nor researchers know who receives the investigational medication or a placebo, and an open-label phase where all participants receive the investigational medication.
Participants will undergo various study procedures, including regular visits for assessments and monitoring. The double-blind phase involves receiving either the investigational medication or a placebo, which is an inactive substance that looks like the investigational medication but does not contain any medicine. After this phase, participants may continue into the open-label phase where they will receive the investigational medication. The study aims to monitor the effects on sickle-cell disease symptoms and overall safety.
- Who can participate: Participants aged 10 to 65 years with a diagnosis of sickle-cell disease and who have experienced between 2 to 10 episodes of acute clinical VOC in the past year are eligible. Participants must not have certain medical conditions such as recent cancer or active infections like HIV or hepatitis. Stable use of hydroxyurea or L-glutamine is permitted.
- Study details: Participants will be required to attend regular visits for assessments and monitoring. During the double-blind phase, participants will receive either the investigational medication or a placebo. Those who complete this phase may continue to an open-label phase where all will receive the investigational medication.
- Study timelines: The study will last 52 weeks for the double-blind period, followed by an open-label period of 52 weeks.
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