A Phase 3 study to determine the Efficacy and Safety of Losmapimod in Treating Patients with FSHD
Research type
Research Study
Full title
A Phase 3 Global, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Patients with Facioscapulohumeral Muscular Dystrophy
IRAS ID
1005829
Contact name
Amin Boateng
Contact email
Sponsor organisation
Fulcrum Therapeutics
Eudract number
2022-000389-16
Clinicaltrials.gov Identifier
Research summary
Facioscapulohumeral Muscular Dystrophy (FSHD), a genetic disease that affects muscle function. FSHD involves progressive muscle weakness that affects the face, shoulders, arms, trunk and also weakness of lower limbs and hips. FSHD causes significant physical disability. The sponsor of this study, Fulcrum Therapeutics, is developing an investigational compound called losmapimod to target the root cause of FSHD.
This study will assess how losmapimod helps to stabilise and/or improve muscle function, how participants feel while taking losmapimod, and its safety and tolerability.
The purpose of the 48-week placebo-controlled period in this study is to evaluate whether losmapimod may slow down the progression of the disease in individuals with FSHD1 and FSHD2. Placebo controlled means that a substance that is not active (placebo) that looks like the study drug will be used. This is done so that the study findings can be looked at fairly.
About 230 adults of the age between 18 and 65 years will take part in this study. This will include about 210 adults with FSHD1 and 20 adults with FSHD2. This study will be conducted at approximately 35 sites in North America and Europe.REC name
East Midlands - Derby Research Ethics Committee
REC reference
22/EM/0147
Date of REC Opinion
25 Aug 2022
REC opinion
Further Information Favourable Opinion