The Efficacy and Safety of Rilzabrutinib in Patients Aged 10 to 65 Years With Sickle-cell Disease
NCT ID: NCT06975865
Last Updated: 2025-12-19
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
192 participants
INTERVENTIONAL
2025-08-12
2028-12-29
Brief Summary
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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.
Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Rilzabrutinib
Rilzabrutinib
Rilzabrutinib
Pharmaceutical form:Tablet -Route of administration:Oral
Placebo
Placebo
Placebo
Pharmaceutical form:Tablet -Route of administration:Oral
Interventions
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Rilzabrutinib
Pharmaceutical form:Tablet -Route of administration:Oral
Placebo
Pharmaceutical form:Tablet -Route of administration:Oral
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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 of age: the parent(s)/legal guardian(s) must provide written informed consent prior to any study-related procedures being performed.
Exclusion Criteria
* 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.
10 Years
65 Years
ALL
No
Sponsors
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Sanofi
INDUSTRY
Responsible Party
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Locations
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University of Alabama at Birmingham- Site Number : 8400003
Birmingham, Alabama, United States
Oncology & Hematology Associates of West Broward- Site Number : 8400029
Coral Springs, Florida, United States
Indiana University School of Medicine - Riley Hospital for Children- Site Number : 8400056
Indianapolis, Indiana, United States
Louisiana State University Health Sciences Center - Shreveport- Site Number : 8400037
Shreveport, Louisiana, United States
University of Michigan Health System - Ann Arbor- Site Number : 8400035
Ann Arbor, Michigan, United States
Richmond University Medical Center- Site Number : 8400038
Staten Island, New York, United States
VCU Massey Cancer Center: Dalton Oncology Clinic- Site Number : 8400012
Richmond, Virginia, United States
Investigational Site Number : 0560003
Brussels, , Belgium
Investigational Site Number : 0560002
Brussels, , Belgium
Investigational Site Number : 0560001
Leuven, , Belgium
Hospital Santa Izabel- Site Number : 0760006
Salvador, Estado de Bahia, Brazil
Universidade Federal da Bahia - Site Number : 0760009
Salvador, Estado de Bahia, Brazil
Universidade Federal de Goias- Site Number : 0760002
Goiânia, Goiás, Brazil
Fundação Faculdade Regional de Medicina de São José do Rio Preto- Site Number : 0760001
São José do Rio Preto, São Paulo, Brazil
Hospital Samaritano De Sao Paulo- Site Number : 0760005
São Paulo, , Brazil
Investigational Site Number : 2500002
Créteil, , France
Investigational Site Number : 2500005
Marseille, , France
Investigational Site Number : 2500001
Paris, , France
Investigational Site Number : 2500004
Toulouse, , France
Investigational Site Number : 2880004
Kintampo, , Ghana
Investigational Site Number : 2880002
Navrongo, , Ghana
Investigational Site Number : 3000001
Athens, , Greece
Investigational Site Number : 3000003
Athens, , Greece
Investigational Site Number : 3760001
Afula, , Israel
Investigational Site Number : 3760002
Afula, , Israel
Investigational Site Number : 3760005
Haifa, , Israel
Investigational Site Number : 3760006
Haifa, , Israel
Investigational Site Number : 3800004
Milan, Milano, Italy
Azienda Ospedaliera Universitaria San Luigi Gonzaga, SSD Microcitemie Malattie Rare Ematologiche-Site Number : 3800007
Orbassano, Torino, Italy
Azienda Ospedaliero Universitaria Careggi SOD Ematologia-Site Number : 3800006
Florence, Tuscany, Italy
Azienda Ospedaliera Ospedali Riuniti Villa Sofia - Cervello-Site Number : 3800002
Palermo, , Italy
IRCCS Ospedale Pediatrico Bambino Gesù-Site Number : 3800001
Roma, , Italy
Centro Ricerche Cliniche Verona s.r.l. presso Ospedale G.B. Rossi Borgo Roma-Site Number : 3800003
Verona, , Italy
Investigational Site Number : 5280002
Rotterdam, , Netherlands
Investigational Site Number : 7240002
Madrid, , Spain
Investigational Site Number : 7240001
Madrid, , Spain
Investigational Site Number : 8340003
Mwanza, , Tanzania
Investigational Site Number : 7920001
Adana, , Turkey (Türkiye)
Investigational Site Number : 7920002
Adana, , Turkey (Türkiye)
Investigational Site Number : 7920003
Mersin, , Turkey (Türkiye)
Investigational Site Number : 8260002
London, Harrow, United Kingdom
Investigational Site Number : 8260001
London, , United Kingdom
Countries
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Central Contacts
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Trial Transparency email recommended (Toll free for US & Canada)
Role: CONTACT
Phone: 800-633-1610
Email: [email protected]
Facility Contacts
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Rosa Maria Catena De Maria
Role: primary
Vincenzo Voi
Role: backup
Valentina Carrai
Role: primary
Silvia Querceto
Role: backup
Rosario Di Maggio
Role: primary
Alessandro Inzerillo
Role: backup
Centro Trial Oncoematologico
Role: primary
Lucia De Franceschi
Role: primary
Related Links
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EFC17872 Plain Language Results Summary
Other Identifiers
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2024-518645-17
Identifier Type: REGISTRY
Identifier Source: secondary_id
U1111-1311-1896
Identifier Type: OTHER_GRANT
Identifier Source: secondary_id
EFC17872
Identifier Type: -
Identifier Source: org_study_id