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Looking for participantsPhase3

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

Sponsor: Sanofi

NCT ID: NCT06975865

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
Rilzabrutinib (drug), Placebo (drug)
How long the study runs
Study runs about 40 months (dates as stated)
About the drug or intervention
Rilzabrutinib — drug: Pharmaceutical form:Tablet -Route of administration:Oral · Placebo — drug: Pharmaceutical form:Tablet -Route of administration:Oral
Patient visit burden
Not specified by the sponsor

In plain English

This study, run by Sanofi, is looking at how well a medicine called rilzabrutinib works and how safe it is for people aged 10 to 65 with sickle cell disease. It focuses on people who have had repeated painful episodes caused by their condition.

Who can take part

  • You have been diagnosed with sickle cell disease.
  • You have had between 2 and 10 documented painful sickle cell episodes (called vaso-occlusive crises) within 12 months before screening.
  • If you take hydroxyurea and/or L-glutamine, you must have taken them for at least 6 months, and been on a stable dose based on your weight for at least 3 months before screening, and plan to keep taking the same dose during the study (unless there is a safety reason to change).
  • You are able to carry out day-to-day activities reasonably well (Eastern Cooperative Oncology Group performance status grade 2 or lower).
  • Men and women must use contraception in line with local rules for people taking part in clinical studies.
  • If you are aged 10 to under 18, a parent or legal guardian must give written informed consent before any study procedures.

Who may not be able to

  • You have had lymphoma, leukaemia, or any cancer in the past 5 years, unless it was a skin cancer (basal cell or squamous cell) fully removed with no spread for the past 3 years.
  • You have a heart problem that the study doctor considers important, or unusual findings on a heart trace test (electrocardiogram).
  • You have had a stroke, or a history of abnormal transcranial doppler (a scan that checks blood flow in the brain).
  • You have uncontrolled or active hepatitis B or hepatitis C infection, including taking antiviral treatment for these at screening.
  • You have HIV infection.
  • You have a history of active or hidden (latent) tuberculosis (TB).
  • You have a positive COVID-19 molecular test.
  • You are taking, or have taken, crizanlizumab (Adakveo) within the past 90 days or voxelotor (Oxbryta) within the past 30 days before screening.

What taking part involves

  • • Not stated — ask the trial team.

Time commitment: Not stated — ask the trial team.

Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.

Type of study
Testing a treatment
Ages
10 Years to 65 Years
Who
All
Number of participants
192
Started
2025-08-12
Last checked
2026-07

Plain English Summary

What is this study?

  • • Testing a new treatment for sickle cell disease
  • • Phase3 - 192 participants
  • • 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)

Who can take part?

  • • Ages 10 Years to 65 Years
  • • Diagnosed with sickle cell disease

Where?

  • • London - Investigational Site Number : 8260002
  • • London - Investigational Site Number : 8260001

This is a simplified summary. Always discuss with your doctor before making any decisions.

About This Trial

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.

Sickle Cell Disease

How this trial compares with your answers

Answer 2 more questions to improve match

What we know so far

Condition· Matched your search
Age· Tell us your age for better matching
Gender· Tell us your sex for better matching

Still need:

  • • Tell us your age for better matching
  • • Tell us your sex for better matching

Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 10 Years - 65 Years
  • Who can join: All genders

Treatment history

Treatments you must have had:

  • ✓ been on a stable weight-based dose level (mg/kg) for at least 3 months

What the study is looking for

  • ✓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...
  • ✓Participants with activity scale (ECOG) performance status grade 2 or lower.
  • ✓Contraceptive use by men and women should be consistent with local regulations regarding the methods of...
  • ✓For participants ≥10 to \<18 years of age: the parent(s)/legal guardian(s) must provide written agreement to take part...

Who cannot take part

  • ✗Clinically relevant heart 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...
  • ✗HIV infection.
  • ✗A history of active or latent tuberculosis (TB)
See the full 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.

Where Is This Study? (2 UK sites)

Investigational Site Number : 8260002

London NW10 7NS, United Kingdom

Recruiting

Investigational Site Number : 8260001

London SE5 9RL, United Kingdom

Recruiting

How to Get in Touch

Trial Transparency email recommended (Toll free for US & Canada)

Sponsor contact

CONTACT

800-633-1610 contact-us@sanofi.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-07