Phase 3 Study of Edasalonexent in Duchenne Muscular Dystrophy
Research type
Research Study
Full title
A RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED, GLOBAL PHASE 3 STUDY OF EDASALONEXENT IN PEDIATRIC PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY
IRAS ID
251874
Contact name
Gary McCullagh
Contact email
Sponsor organisation
Catabasis Pharmaceuticals, Inc.
Eudract number
2018-000464-29
Duration of Study in the UK
1 years, 4 months, 31 days
Research summary
This is a randomized, double-blind, placebo-controlled, Phase 3, multisite study of edasalonexent in pediatric patients with DMD. Approximately 126 patients will be enrolled.
Eligible patients will be ambulatory boys from ≥4.0 to <8.0 years of age with a confirmed diagnosis of DMD who have not used steroids within 24 weeks prior to treatment initiation (Day 1).
The study includes a 52-week, double-blind, placebo-controlled period comparing 1 dose level of edasalonexent with placebo, followed by a 2-week follow-up.
Following completion of the treatment period and Primary Endpoint evaluation, patients may elect to continue in the open-label extension study, pending approval by appropriate ethics committees and regulatory authorities. Patients who choose not to participate should complete the safety follow-up within 2 weeksREC name
East Midlands - Derby Research Ethics Committee
REC reference
19/EM/0003
Date of REC Opinion
20 Feb 2019
REC opinion
Further Information Favourable Opinion