Tests rilzabrutinib in people with sickle cell disease
Tests rilzabrutinib against placebo in people aged 10 to 65 with sickle cell disease.
Key facts
- Who runs it
- Sanofi
- Where
- Madrid, SpainSee the 2 sitesAlso in 13 other countries, 61 sites
- Trial code
- NCT06975865
- EudraCT 2024-518645-17
- U1111-1311-1896
- Registry title
- The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease
- Status
- Recruiting
- Phase
- Phase 3
- People taking part
- 192 people, planned
- Started
- 12 Aug 2025
- Expected to end
- 29 Dec 2028
- Type of study
- Interventional, 2 groupsOne group gets a placebo (a dummy treatment), for comparison.
- Treatments tested
- Rilzabrutinib
- Placebo
- Record updated
- 15 October 2026
Who can take part
AI summary
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for people aged 10 to 65 with sickle-cell disease who have had 2 to 10 documented pain crises (VOC) in the past 12 months.
- Age
- 10 to 65 years. For ages 10 to under 18, a parent or legal guardian must give written consent.
- Sex
- All sexes can join.
- Disease stage
- Diagnosed sickle-cell disease with between 2 and 10 documented clinical VOC episodes within 12 months of screening. ECOG performance status grade 2 or lower.
- Earlier treatment
- People not taking hydroxyurea or L-glutamine and not planning to, or people who have taken them for at least 6 months. Those taking them must have been on a stable weight-based dose for at least 3 months and keep it during the study. No crizanlizumab (ADAKVEO) within 90 days and no voxelotor (OXBRYTA) within 30 days before screening.
Main reasons for not taking part
- Lymphoma, leukemia or any other cancer in the past 5 years (some resected skin cancers excepted)
- Clinically relevant heart abnormality
- History of stroke or abnormal transcranial doppler
- Uncontrolled or active hepatitis B or hepatitis C infection, or HIV infection
- History of active or latent tuberculosis, or a positive COVID-19 molecular test
The registry criteria, word for word
Show all 14 criteriaShow less
Who can take part
- 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
Who cannot take part
- Participants with uncontrolled or active HBV infection and/or HCV infection including those receiving antiviral therapy at the time of screening
- 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
- 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
- HIV infection
- A history of active or latent tuberculosis (TB)
- Positive COVID-19 molecular test
Check if you might fit
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 10 and 65 years old?
Where in Spain
Also in 13 other countries, 61 sites
- MadridHospital not named in the registryTaking people
- MadridHospital not named in the registryTaking people
About the trial
What the trial wants to find out
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).
Read full original descriptionShow less
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.
The 2 groups in the trial
- RilzabrutinibTreatment being tested
- PlaceboPlacebo, for comparison
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorData and sources
- Registry record
- Listed on ClinicalTrials.gov.
- Official title
- 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
- Plain-language title
- Tests rilzabrutinib in people with sickle cell diseaseGenerated from the registry record. The registry title is the official one.
- AI summary
- From the trial's eligibility criteria. Read by machine on 12 November 2026.
- Fit questions
- Built from NCT06975865, who can take part
- Record updated
- 15 October 2026