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

Completed · Phase 3 · Has a placebo group

Conditions studied: Duchenne Muscular Dystrophy

In brief

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.

Key facts

Study ID

NCT02500381

Run by

Sarepta Therapeutics, Inc.

People needed

228

Starts

2016-09-28

Expected to finish

2025-10-16

Last updated by the study team

2025-11-18

Who can join

Age: 6 and older, up to 13. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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 brachii muscles or 2 alternative upper arm muscle groups
• Mean 6MWT ≥300 meters and ≤450 meters
• Stable pulmonary function: forced vital capacity (FVC) ≥50% predicted

You may not qualify if…

• 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.

Where it is running

• Children's Hospital Los Angeles — Los Angeles, California, United States
• David Geffen School of Medicine, UCLA — Los Angeles, California, United States
• Rady Children's Hospital San Diego/ UCSD — San Diego, California, United States
• Stanford University School of Medicine/Medical Center — Stanford, California, United States
• University of Florida — Gainesville, Florida, United States
• NW Florida Clinical Research Group, LLC — Gulf Breeze, Florida, United States
• Center for Integrative Rare Disease Research (CIRDR) — Atlanta, Georgia, United States
• Ann and Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
• University of Iowa Children's Hospital — Iowa City, Iowa, United States
• University of Kansas, Medical Center — Kansas City, Kansas, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• St. Louis Children's Hospital — St Louis, Missouri, United States
• Las Vegas Clinic — Las Vegas, Nevada, United States
• University of Rochester Clinical Research Center — Rochester, New York, United States
• Cincinnati Children's Hospital Medical Center (CCHMC) — Cincinnati, Ohio, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Shriners Hospital for Children — Portland, Oregon, United States
• Children's Hospital of Pittsburgh of UPMC — Pittsburgh, Pennsylvania, United States
• Children's Medical Center Dallas — Dallas, Texas, United States
• University of Utah — Salt Lake City, Utah, United States
• Children's Hospital of the King's Daughters — Norfolk, Virginia, United States
• Children's Hospital of Wisconsin — Milwaukee, Wisconsin, United States
• DOM Centro de Reumatologia — Buenos Aires, Argentina
• Royal Children's Hospital Melbourne — Parkville, Victoria, Australia
• Neuromuscular Research Center — Phoenix, Arizona, United States

Full record on ClinicalTrials.gov

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