Sickle Cell DiseasePhase 3LIBRANCT06975865
The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease
A Phase 3 study testing Rilzabrutinib in people with Sickle Cell Disease, run by Sanofi at 63 sites in 14 countries.
Trial summary
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 more about this study
- Who is it for?
- Ages 10 to 65 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Rilzabrutinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 192 participants
- Where
- 63 sitesin 14 countries
- Study dates
- 12 Aug 2025to 29 Dec 2028
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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 join
- 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)
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- 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.
Trial locations and contacts
63 sites in 14 countries. Contact the study team or a site near you to ask about taking part.
Trial Transparency email recommended (Toll free for US & Canada) contact
63 sites in 14 countries
United States21 sites, 21 recruiting
- Recruiting
University of Michigan Health System - Ann Arbor- Site Number : 8400035
Ann Arbor, Michigan, 48109
- Recruiting
University of Maryland School of Medicine - Baltimore- Site Number : 8400041
Baltimore, Maryland, 21201
- Recruiting
University of Alabama at Birmingham- Site Number : 8400003
Birmingham, Alabama, 35233
- Recruiting
University of Illinois-Chicago - College of Medicine- Site Number : 8400054
Chicago, Illinois, 60612
- Recruiting
University of California San Francisco- Site Number : 8400040
Clovis, California, 93611
- Recruiting
Oncology & Hematology Associates of West Broward- Site Number : 8400029
Coral Springs, Florida, 33065
- Recruiting
Southern Specialty Research- Site Number : 8400059
Flowood, Mississippi, 39232
- Recruiting
Prisma Health- Site Number : 8400051
Greenville, South Carolina, 29605
- Recruiting
Baylor College of Medicine- Site Number : 8400055
Houston, Texas, 77030
- Recruiting
UT Physicians - Memorial Hermann- Site Number : 8400052
Houston, Texas, 77030
- Recruiting
Indiana University Health Riley Hospital for Children- Site Number : 8400056
Indianapolis, Indiana, 46202
- Recruiting
David Geffen School of Medicine at UCLA- Site Number : 8400032
Los Angeles, California, 90095
- Recruiting
University of Tennessee Health Science Center- Site Number : 8400045
Memphis, Tennessee, 38103
- Recruiting
Sylvester Comprehensive Cancer Center- Site Number : 8400020
Miami, Florida, 33136
- Recruiting
Phoenix Children's Hospital- Site Number : 8400028
Phoenix, Arizona, 85016
- Recruiting
Oregon Health & Science University (OHSU)- Site Number : 8400043
Portland, Oregon, 97239
- Recruiting
VCU Massey Cancer Center: Dalton Oncology Clinic- Site Number : 8400012
Richmond, Virginia, 23298
- Recruiting
Louisiana State University Health Sciences Center - Shreveport- Site Number : 8400037
Shreveport, Louisiana, 71130
- Recruiting
Richmond University Medical Center- Site Number : 8400038
Staten Island, New York, 10310
- Recruiting
Montefiore Medical Center - Moses Campus- Site Number : 8400057
The Bronx, New York, 10467
- Recruiting
MedStar Georgetown University Hospital- Site Number : 8400036
Washington D.C., District of Columbia, 20007
Italy8 sites, 8 recruiting
- Recruiting
Investigational Site Number : 3800006
Florence, Firenze, 50134
- Recruiting
Investigational Site Number : 3800008
Genoa, Genova, 16128
- Recruiting
Investigational Site Number : 3800004
Milan, Milano, 20122
- Recruiting
Investigational Site Number : 3800005
Naples, Napoli, 80138
- Recruiting
Investigational Site Number : 3800007
Orbassano, Torino, 10043
- Recruiting
Investigational Site Number : 3800002
Palermo, 90146
- Recruiting
Investigational Site Number : 3800001
Rome, Roma, 00165
- Recruiting
Investigational Site Number : 3800003
Verona, 37134
Brazil5 sites, 5 recruiting
- Recruiting
Universidade Federal de Goias- Site Number : 0760002
Goiânia, Goiás, 74605-020
- Recruiting
Hospital Santa Izabel- Site Number : 0760006
Salvador, Estado de Bahia, 40050-410
- Recruiting
Fundação Bahiana de Infectologia- Site Number : 0760009
Salvador, Estado de Bahia, 40110-160
- Recruiting
Fundação Faculdade Regional de Medicina de São José do Rio Preto- Site Number : 0760001
São José do Rio Preto, São Paulo, 15090-000
- Recruiting
Hospital Samaritano De Sao Paulo- Site Number : 0760005
São Paulo, 01232-010
Belgium4 sites, 4 recruiting
- Recruiting
Investigational Site Number : 0560003
Brussels, 1020
- Recruiting
Investigational Site Number : 0560002
Brussels, 1070
- Recruiting
Investigational Site Number : 0560001
Leuven, 3000
- Recruiting
Investigational Site Number : 0560004
Liège, 4000
France4 sites, 4 recruiting
- Recruiting
Investigational Site Number : 2500002
Créteil, 94010
- Recruiting
Investigational Site Number : 2500005
Marseille, 13885
- Recruiting
Investigational Site Number : 2500001
Paris, 75015
- Recruiting
Investigational Site Number : 2500004
Toulouse, 31100
Israel4 sites, 4 recruiting
- Recruiting
Investigational Site Number : 3760001
Afula, 1834111
- Recruiting
Investigational Site Number : 3760002
Afula, 1834111
- Recruiting
Investigational Site Number : 3760005
Haifa, 3109601
- Recruiting
Investigational Site Number : 3760006
Haifa, 3109601
Germany3 sites, 3 recruiting
- Recruiting
Investigational Site Number : 2760005
Berlin, 13353
- Recruiting
Investigational Site Number : 2760002
Essen, 45147
- Recruiting
Investigational Site Number : 2760004
Stuttgart, 70736
Greece3 sites, 3 recruiting
- Recruiting
Investigational Site Number : 3000001
Athens, 115 27
- Recruiting
Investigational Site Number : 3000003
Athens, 115 27
- Recruiting
Investigational Site Number : 3000002
Pátrai, 265 04
Turkey (Türkiye)3 sites, 3 recruiting
- Recruiting
Investigational Site Number : 7920001
Adana, 01330
- Recruiting
Investigational Site Number : 7920002
Adana, 01330
- Recruiting
Investigational Site Number : 7920003
Mersin, 33200
United Kingdom3 sites, 3 recruiting
- Recruiting
Investigational Site Number : 8260002
London, Harrow, NW10 7NS
- Recruiting
Investigational Site Number : 8260001
London, London, City of, SE5 9RL
- Recruiting
Investigational Site Number : 8260005
Manchester, M13 9WL
Spain2 sites, 2 recruiting
- Recruiting
Investigational Site Number : 7240001
Madrid, Madrid, Comunidad de, 28046
- Recruiting
Investigational Site Number : 7240002
Madrid, 28007
Netherlands1 site, 1 recruiting
- Recruiting
Investigational Site Number : 5280002
Rotterdam, 3015 CE
Saudi Arabia1 site, 1 recruiting
- Recruiting
Investigational Site Number : 6820003
Riyadh, 12713
About this study
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
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
- Sickle Cell Disease
- Being tested
- Rilzabrutinib, Placebo
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RilzabrutinibDrug
Also called SAR444671
Pharmaceutical form:Tablet -Route of administration:Oral
PlaceboDrug
Pharmaceutical form:Tablet -Route of administration:Oral
| Study group | Role | What they receive |
|---|---|---|
| Rilzabrutinib | Experimental | Rilzabrutinib Receives: Drug: Rilzabrutinib |
| Placebo | Placebo comparator | Placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized rate of clinical VOC
Calculated from total number of clinical VOC incidents and total number of days during the observation period
Measured: At Week 52
Secondary outcomes (10)
Time to first clinical VOC incidence
Measured: Until Week 52
Annualized rate of visits due to SCD-related complications as assessed by the Investigator
Measured: At Week 52
Annualized rate of home-managed VOCs as reported in the Sickle Cell Pain Crisis (SCPC) eDiary
Measured: At Week 52
Change in fatigue as measured by the PROMIS SF v1.0 Fatigue 13a total score (adults)
Measured: From baseline to Week 52
Change in Hb levels
Measured: From baseline to Week 52
Change in fatigue as measured by the PedsQL Multidimensional Fatigue Scale total score (pediatric participants)
Measured: From baseline to Week 52
Incidence of treatment emergent adverse events (TEAEs), including serious adverse events (SAEs), adverse events of special interest (AESIs) and adverse events leading to discontinuation
Measured: Until Week 52
Incidence of potentially clinically significant laboratory, vital signs, and ECG abnormalities
Measured: Until Week 52
Absolute number of simple and exchange blood transfusion
Measured: Until Week 52
Number of days requiring acetaminophen, NSAID and/or short-acting opioid usage
Measured: Until Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 May 2025
- Study start12 Aug 2025
- Primary completion23 Jul 2027 (estimated)
- Study completion29 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by SanofiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 192 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06975865
- Sponsor's protocol ID
- EFC17872
- Other IDs
- 2024-518645-17, U1111-1311-1896
- Acronym
- LIBRA
- Status last verified
- Oct 2026
- First submitted
- 8 May 2025
- Last updated
- 15 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org