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

NCT06975865

RecruitingPhase 3

Sponsor: Sanofi

Conditions: Sickle Cell Disease

Interventions: Rilzabrutinib, Placebo

Countries: United States, Belgium, Brazil, France, Germany, Greece, Israel, Italy

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 overview

Sex: ALL

Age: 10 Years to 65 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

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.
Locations (53)
  • Birmingham, Alabama, United States
  • Phoenix, Arizona, United States
  • Fresno, California, United States
  • Coral Springs, Florida, United States
  • Miami, Florida, United States
  • Chicago, Illinois, United States
  • Indianapolis, Indiana, United States
  • Shreveport, Louisiana, United States
  • Baltimore, Maryland, United States
  • Ann Arbor, Michigan, United States
  • Flowood, Mississippi, United States
  • Staten Island, New York, United States
  • The Bronx, New York, United States
  • Greenville, South Carolina, United States
  • Houston, Texas, United States
  • Richmond, Virginia, United States
  • Brussels, Belgium
  • Brussels, Belgium
  • Leuven, Belgium
  • Salvador, Estado de Bahia, Brazil
  • Goiânia, Goiás, Brazil
  • São José do Rio Preto, São Paulo, Brazil
  • Rio de Janeiro, Brazil
  • São Paulo, Brazil
  • Créteil, France
  • Marseille, France
  • Paris, France
  • Toulouse, France
  • Essen, Germany
  • Stuttgart, Germany
  • + 23 more on CT.gov