RightFit
RecruitingPhase 3

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

A 52-week, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group, Flexible-adaptive, Group Sequential Study to Evaluate the Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
10 Years to 65 Years
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests how well rilzabrutinib works and how safe it is for people ages 10 to 65 with sickle-cell disease. For 52 weeks, participants receive either rilzabrutinib or a placebo (a treatment with no active drug). Those who finish this part may join a longer treatment period.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Have a diagnosis of sickle-cell disease and be 10 to 65 years old.
  • Have 2 to 10 recorded pain crises caused by blocked blood flow in the 12 months before screening.
  • If not taking hydroxyurea or L-glutamine at screening, plan not to take them during the study.
  • If taking hydroxyurea or L-glutamine, have taken it for at least 6 months. The dose based on body weight must be stable for at least 3 months before screening, with plans to keep it the same unless safety requires a change.
  • Have an ECOG activity score of 2 or less: be able to care for yourself and be out of bed for more than half the day.
  • Follow local rules for birth control during clinical studies.
  • For ages 10 to 17, a parent or legal guardian must give written permission before any study procedures.

Who can't join

  • Have a history of lymphoma or leukemia (blood cancers), or another cancer within 5 years. Some removed skin cancers are allowed if there has been no sign of spread for 3 years.
  • Have a heart problem or abnormal ECG (heart electrical test) that the study doctor considers important.
  • Have had a stroke or an abnormal ultrasound test of blood flow in the brain.
  • Have active or uncontrolled hepatitis B or C (liver infections), including treatment with antiviral drugs at screening.
  • Have HIV infection.
  • Have a history of active or latent tuberculosis (TB, including infection without symptoms).
  • Have a positive molecular test for COVID-19.
  • Take or have taken crizanlizumab (ADAKVEO) within 90 days before screening, or voxelotor (OXBRYTA) within 30 days before screening.

Simplified for readability. The official criteria below are what the study team uses.

Official 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.

Study sites (21)

AL

AZ

CA

DC

FL

IL

  • University of Illinois-Chicago - College of Medicine- Site Number : 8400054 · Chicago, IL 60612More trials in Chicago

IN

LA

  • Louisiana State University Health Sciences Center - Shreveport- Site Number : 8400037 · Shreveport, LA 71130More trials in Shreveport

MD

  • University of Maryland School of Medicine - Baltimore- Site Number : 8400041 · Baltimore, MD 21201More trials in Baltimore

MI

MS

NY

OR

SC

TN

  • University of Tennessee Health Science Center- Site Number : 8400045 · Memphis, TN 38103More trials in Memphis

TX

VA

  • VCU Massey Cancer Center: Dalton Oncology Clinic- Site Number : 8400012 · Richmond, VA 23298More trials in Richmond