Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)


Trial ID: NCT02500381


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

Stanford Investigator(s)

John W. Day, MD, PhD
John W. Day, MD, PhD

Professor of Neurology (Adult Neurology), of Pediatrics (Genetics) and, by courtesy, of Pathology

Kristine Luce

Clinical Professor, Psychiatry and Behavioral Sciences


Inclusion Criteria:

   - Genotypically confirmed DMD, with genetic deletion amenable to exon 45 or exon 53

   - 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.


drug: SRP-4045

drug: SRP-4053

drug: Placebo


Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305