NCT06975865 · Sanofi
The Efficacy and Safety of Rilzabrutinib in Patients Aged 10 to 65 Years With Sickle-cell Disease
(LIBRA)
What this study is about
This is a conducted at multiple hospitals, randomly assigned, where neither patients nor doctors know which treatment is given, compared against an inactive treatment, parallel-group, group-sequential study (Part A), followed by an where both patients and doctors know the treatment given LTE period (Part B) to investigate the effectiveness, and safety of rilzabrutinib in participants with sickle-cell disease (SCD). Study details include: * Study duration: a 52-week where neither patients nor doctors know which treatment is given period (Part A), followed by an where both patients and doctors know the treatment given LTE period (Part B). where neither patients nor doctors know which treatment is given period has two parts, 50% (adult only) until the interim analysis (a proof-concept part
View original scientific description
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).
Interventions
DRUG
Rilzabrutinib
Pharmaceutical form:Tablet -Route of administration:Oral
DRUG
Placebo
Pharmaceutical form:Tablet -Route of administration:Oral
Primary outcome measures
Annualized rate of clinical VOC
Time frame: At Week 52
Calculated from total number of clinical VOC incidents and total number of days during the observation period
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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
Where
- Birmingham, Alabama
- Phoenix, Arizona
- Fresno, California
- Coral Springs, Florida
- Miami, Florida
- Chicago, Illinois
- Indianapolis, Indiana
- Shreveport, Louisiana
- Baltimore, Maryland
- Ann Arbor, Michigan
- Flowood, Mississippi
- Staten Island, New York
And 4 more locations — see the full list below.
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jun 23, 2026 · Source of record for eligibility and locations