The Efficacy and Safety of Rilzabrutinib in Patients Aged 10 to 65 Years With Sickle-cell Disease
Purpose
This is a 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 (a proof-concept part analogous to a phase 2b study), 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.
Condition
- Sickle Cell Disease
Eligibility
- Eligible Ages
- Between 10 Years and 65 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
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.
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Rilzabrutinib |
Rilzabrutinib |
|
|
Placebo Comparator Placebo |
Placebo |
|
Recruiting Locations
Birmingham 4049979, Alabama 4829764 35233
More Details
- Status
- Recruiting
- Sponsor
- Sanofi
Study Contact
Trial Transparency email recommended (Toll free for US & Canada)800-633-1610
contact-us@sanofi.com