Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)
Trial ID or NCT#
NCT02500381
Status
RECRUITING
Purpose
The main objective of this study is to evaluate the efficacy of SRP-4045 (casimersen) and SRP-4053 (golodirsen) compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53, respectively.
Official Title
A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy
Eligibility Criteria
Ages Eligible for Study: 6 Years to 13 Years
Sexes Eligible for Study: Male
Accepts Healthy Volunteers: No
Inclusion Criteria:
- - Genotypically confirmed DMD, with genetic deletion amenable to exon 45 or exon 53 skipping - Stable dose of oral corticosteroids for at least 24 weeks prior to Week 1, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight). - Intact right and left biceps or 2 alternative upper muscle groups - Mean 6MWT ≥300 meters and ≤450 meters - Stable pulmonary function: forced vital capacity (FVC) ≥50% predicted
Exclusion Criteria:
- - Treatment with gene therapy at any time - Previous treatment with SMT C1100 within 1 week prior to Week 1 and previous treatment with PRO045 (BMN 045), PRO053 (BMN 053), or PRO051 (BMN 051) within 24 weeks prior to Week 1 - Current or previous treatment with any other experimental treatment within 12 weeks prior to Week 1 - Major surgery within 3 months prior to Week 1 - Presence of other clinically significant illness Other inclusion/exclusion criteria may apply.
Investigator(s)
John W. Day, MD, PhD
Neuromuscular neurologist,
Neurophysiologist
Professor of Neurology (Adult Neurology), of Pediatrics (Genetics) and, by courtesy, of Pathology
View on ClinicalTrials.gov
