NCT06975865 The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease
| NCT ID | NCT06975865 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | Sanofi |
| Condition | Sickle Cell Disease |
| Study Type | INTERVENTIONAL |
| Enrollment | 192 participants |
| Start Date | 2025-08-12 |
| Primary Completion | 2027-07-23 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 3 trials are large pivotal studies comparing the treatment to current standard of care or placebo. Your participation directly contributes to the evidence needed for regulatory approval.
This trial targets 192 participants in total. It began in 2025-08-12 with a primary completion date of 2027-07-23.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, flexible-adaptive, 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.
Eligibility Criteria
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.
Contact & Investigator
Trial Transparency email recommended (Toll free for US & Canada)
✉ contact-us@sanofi.com📞 800-633-1610
Frequently Asked Questions
Who can join the NCT06975865 clinical trial?
This trial is open to participants of all sexes, aged 10 Years or older, up to 65 Years, studying Sickle Cell Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06975865 trial and what does that mean for participants?
Phase 3 trials are large-scale studies comparing the new treatment to existing standards of care or a placebo. They provide the evidence needed for regulatory approval. This trial targets 192 participants.
Is NCT06975865 currently recruiting?
Yes, NCT06975865 is actively recruiting participants. Contact the research team at contact-us@sanofi.com for enrollment information.
Where is the NCT06975865 trial being conducted?
This trial is being conducted at Birmingham, United States, Phoenix, United States, Fresno, United States, Coral Springs, United States and 11 additional locations.
Who is sponsoring the NCT06975865 clinical trial?
NCT06975865 is sponsored by Sanofi. The trial plans to enroll 192 participants.
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