LIBRA
Research type
Research Study
Full title
A 52-week, multicenter, randomized, double-blind, placebo-controlled, parallel-group, group sequential, Phase 3 study to evaluate the efficacy and safety of rilzabrutinib in patients aged 10 to 65 years with sickle-cell disease
IRAS ID
1011036
Contact name
Patrick Maury
Contact email
Sponsor organisation
Sanofi-Aventis Recherche & Développement
Eudract number
2024-518645-17
Research summary
This is a Phase 3, multicentermulticentre, randomised, double-blind, placebo-controlled, parallel-group, group-sequential study (Part A), followed by an open-label long-term extension (LTE) period (Part B) for a new sickle-cell disease experimental drug.
Rilzabrutinib is an experimental medicine that is given orally twice-daily. Rilzabrutinib mediates its effect through blocking a functional protein called Burton’s Tyrosine Kinase (BTK). BTK play a role in immunity such as activation of some blood cells like B-cell, basophil, neutrophil, mast cell and macrophages. Rilzabrutinib is a high-affinity inhibitor of BTK and the treatment with rilzabrutinib may result in reduction of chronic inflammatory state, blood cell adhesion, oxidative stress that can ultimately result in prevention of vaso-occlusive crisis (VOC) and hemolysis. The main objective of this study is to compare the safety and effects of rilzabrutinib with the safety and effects of a placebo in sickle-cell disease.
This study will help the study Sponsor find out if this new treatment approach is better, the same, or worse than the usual treatment approach for your sickle-cell disease and/or placebo. To decide if it is better, the study Sponsor will be looking to see if rilzabrutinib prevents the clinical vaso-occlusive crisis.
Participants will either get rilzabrutinib or placebo for up to 52 weeks (blinded treatment period). Afterwards, for participants who complete the double-blind period they will be given the option to join the LTE part. The maximum duration of the long-term extension (LTE) period will be 12 months from the date of the last participant to enter the LTE. A computer will randomly assign participants who enter the study to the experimental group (ie, rilzabrutinib) or the control group (ie, placebo). Participants have a will have a 2 in 3 chance of being assigned to the experimental group.REC name
East of England - Cambridge Central Research Ethics Committee
REC reference
24/EE/0252
Date of REC Opinion
10 Feb 2025
REC opinion
Further Information Favourable Opinion