CN001016: Phase 2/3 Study of BMS-986089 in Ambulatory Boys with DMD
Research type
Research Study
Full title
A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys with Duchenne Muscular Dystrophy
IRAS ID
225539
Contact name
Mariacristina Scoto
Contact email
Sponsor organisation
F Hoffmann-La Roche Ltd
Eudract number
2016-001654-18
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
Universal Trial Number (UTN) number, U1111-1181-8752
Duration of Study in the UK
2 years, 7 months, 31 days
Research summary
Duchenne Muscular Dystrophy (DMD) affects 1 in 3600-6000 live male births. DMD is caused by a mutation/change in the gene that makes dystrophin (a protein). Dystrophin protects muscles from stress and damage during activity. Boys with DMD are unable to make enough working dystrophin to protect their muscles and muscle mass progressively decreases. Cardiac and respiratory failure typically begin to develop in adolescence, leading to death in the second or third decade of life. \n\nStandard of care is symptomatic treatment, including medical and physical therapies to improve cardiac and respiratory function, and corticosteroids to improve skeletal muscle strength and function. However, corticosteroids cause significant adverse side effects, including obesity, diabetes, and fracture. \n\nTwo drugs are recently approved for DMD, however both target restricted populations (10.2 % and 13%) with specific mutations therefore the unmet need for safe and effective treatments in DMD remains significant. \n\nBMS-986089 is an anti-myostatin agent which blocks myostatin, a protein involved in regulating muscle mass. Myostatin has a negative effect on muscle growth therefore muscle mass may increase by blocking myostatin. Treatment with BMS-986089 could therefore increase muscle size and strength. \n\nBMS-986089 is expected to preserve and improve muscle strength by increasing the size of existing muscle fibres. A study in healthy volunteers showed that BMS-986089 treatment was associated with increases in thigh muscle volume and total body mass. \n\nPreliminary safety data from a phase 1b/2 study in ambulatory boys (aged ≥5to<11 years on stable corticosteroid treatment), suggests that BMS-986089 is generally safe and well tolerated in this population. \n\nThis study is sponsored by Bristol-Myers Squibb and will evaluate the efficacy, safety and tolerability of weekly subcutaneous doses of BMS-986089 compared to placebo in ambulatory boys with DMD. Approximately 159 participants aged ≥6to<12 will be enrolled from approximately 40 centres worldwide. Participants will be in the study for approximately 96 weeks (double-blind and open-label phases) followed by a 24 week follow-up period.\n
REC name
London - Brighton & Sussex Research Ethics Committee
REC reference
17/LO/1240
Date of REC Opinion
12 Oct 2017
REC opinion
Further Information Favourable Opinion