Actively Recruiting
The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease
Led by Sanofi · Updated on 2026-05-01
192
Participants Needed
54
Research Sites
176 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosed with sickle-cell disease.
- Had between 2 and 10 documented clinical vaso-occlusive crisis episodes within 12 months before screening.
- Either not on hydroxyurea and/or L-glutamine and not planning to start them during the study, or have taken hydroxyurea and/or L-glutamine for at least 6 months.
- If taking hydroxyurea and/or L-glutamine, must have been on a stable weight-based dose for at least 3 months before screening and plan to continue the same dose during the study, except for safety reasons.
- Eastern Cooperative Oncology Group (ECOG) performance status grade 2 or lower.
- Men and women must use contraception according to local regulations during the study.
- For participants aged 10 to under 18 years, a parent or legal guardian must give written informed consent before any study procedures.
You will not qualify if you...
- History of lymphoma, leukemia, or any malignancy within the past 5 years except for basal cell or squamous epithelial skin carcinomas resected with no metastatic disease for 3 years.
- Clinically relevant cardiac abnormalities or abnormal ECG findings.
- History of stroke or abnormal transcranial doppler.
- Uncontrolled or active hepatitis B or C infection, including those receiving antiviral therapy.
- HIV infection.
- History of active or latent tuberculosis.
- Positive COVID-19 molecular test.
- Use of crizanlizumab within 90 days or voxelotor within 30 days before screening.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 54 locations
1
University of Alabama at Birmingham- Site Number : 8400003
Birmingham, Alabama, United States, 35233
Actively Recruiting
2
Phoenix Children's Hospital- Site Number : 8400028
Phoenix, Arizona, United States, 85016
Actively Recruiting
3
University of California San Francisco- Site Number : 8400040
Fresno, California, United States, 93701
Actively Recruiting
4
Oncology & Hematology Associates of West Broward- Site Number : 8400029
Coral Springs, Florida, United States, 33065
Actively Recruiting
5
Sylvester Comprehensive Cancer Center- Site Number : 8400020
Miami, Florida, United States, 33136
Actively Recruiting
6
University of Illinois-Chicago - College of Medicine- Site Number : 8400054
Chicago, Illinois, United States, 60612
Actively Recruiting
7
Indiana University Health Riley Hospital for Children- Site Number : 8400056
Indianapolis, Indiana, United States, 46202
Actively Recruiting
8
Louisiana State University Health Sciences Center - Shreveport- Site Number : 8400037
Shreveport, Louisiana, United States, 71130
Actively Recruiting
9
University of Maryland School of Medicine - Baltimore- Site Number : 8400041
Baltimore, Maryland, United States, 21201
Actively Recruiting
10
University of Michigan Health System - Ann Arbor- Site Number : 8400035
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
11
Southern Specialty Research- Site Number : 8400059
Flowood, Mississippi, United States, 39232
Actively Recruiting
12
Richmond University Medical Center- Site Number : 8400038
Staten Island, New York, United States, 10310
Actively Recruiting
13
Baylor College of Medicine- Site Number : 8400055
Houston, Texas, United States, 77030
Actively Recruiting
14
VCU Massey Cancer Center: Dalton Oncology Clinic- Site Number : 8400012
Richmond, Virginia, United States, 23298
Actively Recruiting
15
Investigational Site Number : 0560003
Brussels, Belgium, 1020
Actively Recruiting
16
Investigational Site Number : 0560002
Brussels, Belgium, 1070
Actively Recruiting
17
Investigational Site Number : 0560001
Leuven, Belgium, 3000
Actively Recruiting
18
Hospital Santa Izabel- Site Number : 0760006
Salvador, Estado de Bahia, Brazil, 40050-410
Actively Recruiting
19
Universidade Federal de Goias- Site Number : 0760002
Goiânia, Goiás, Brazil, 74605-020
Actively Recruiting
20
Fundação Faculdade Regional de Medicina de São José do Rio Preto- Site Number : 0760001
São José do Rio Preto, São Paulo, Brazil, 15090-000
Actively Recruiting
21
Pontifícia Universidade Católica do Rio de Janeiro- Site Number : 0760009
Rio de Janeiro, Brazil, 22451-900
Actively Recruiting
22
Hospital Samaritano De Sao Paulo- Site Number : 0760005
São Paulo, Brazil, 01232-010
Actively Recruiting
23
Investigational Site Number : 2500002
Créteil, France, 94010
Actively Recruiting
24
Investigational Site Number : 2500005
Marseille, France, 13885
Actively Recruiting
25
Investigational Site Number : 2500001
Paris, France, 75015
Actively Recruiting
26
Investigational Site Number : 2500004
Toulouse, France, 31100
Actively Recruiting
27
Investigational Site Number : 2760002
Essen, Germany, 45147
Actively Recruiting
28
Investigational Site Number : 2760004
Stuttgart, Germany, 70736
Actively Recruiting
29
Investigational Site Number : 2880004
Kintampo, Ghana
Active, Not Recruiting
30
Investigational Site Number : 2880002
Navrongo, Ghana, UK-0043-7777
Active, Not Recruiting
31
Investigational Site Number : 3000001
Athens, Greece, 115 27
Actively Recruiting
32
Investigational Site Number : 3000003
Athens, Greece, 115 27
Actively Recruiting
33
Investigational Site Number : 3000002
Pátrai, Greece, 265 04
Actively Recruiting
34
Investigational Site Number : 3760001
Afula, Israel, 1834111
Actively Recruiting
35
Investigational Site Number : 3760002
Afula, Israel, 1834111
Actively Recruiting
36
Investigational Site Number : 3760005
Haifa, Israel, 3109601
Actively Recruiting
37
Investigational Site Number : 3760006
Haifa, Israel, 3109601
Actively Recruiting
38
Azienda Ospedaliero Universitaria Careggi SOD Ematologia-Site Number : 3800006
Florence, Firenze, Italy, 50134
Actively Recruiting
39
Investigational Site Number : 3800004
Milan, Milano, Italy, 20122
Actively Recruiting
40
Azienda Ospedaliera Universitaria, Università della Campania "Luigi Vanvitelli" Napoli-Site Number : 3800005
Naples, Napoli, Italy, 80138
Actively Recruiting
41
IRCCS Ospedale Pediatrico Bambino Gesù-Site Number : 3800001
Rome, Roma, Italy, 00165
Actively Recruiting
42
Azienda Ospedaliera Universitaria San Luigi Gonzaga, SSD Microcitemie Malattie Rare Ematologiche-Site Number : 3800007
Orbassano, Torino, Italy, 10043
Actively Recruiting
43
Azienda Ospedaliera Ospedali Riuniti Villa Sofia - Cervello-Site Number : 3800002
Palermo, Italy, 90146
Actively Recruiting
44
Centro Ricerche Cliniche Verona s.r.l. presso Ospedale G.B. Rossi Borgo Roma-Site Number : 3800003
Verona, Italy, 37134
Actively Recruiting
45
Investigational Site Number : 5280002
Rotterdam, Netherlands, 3015 CE
Actively Recruiting
46
Investigational Site Number : 5120001
Muscat, Oman, 123
Actively Recruiting
47
Investigational Site Number : 7240002
Madrid, Spain, 28007
Actively Recruiting
48
Investigational Site Number : 7240001
Madrid, Spain, 28046
Actively Recruiting
49
Investigational Site Number : 8340003
Mwanza, Tanzania, 1462
Active, Not Recruiting
50
Investigational Site Number : 7920001
Adana, Turkey (Türkiye), 01330
Actively Recruiting
51
Investigational Site Number : 7920002
Adana, Turkey (Türkiye), 01330
Actively Recruiting
52
Investigational Site Number : 7920003
Mersin, Turkey (Türkiye), 33330
Actively Recruiting
53
Investigational Site Number : 8260002
London, Harrow, United Kingdom, NW10 7NS
Actively Recruiting
54
Investigational Site Number : 8260001
London, London, City of, United Kingdom, SE5 9RL
Actively Recruiting
Research Team
T
Trial Transparency email recommended (Toll free for US & Canada)
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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