Phase I/II Study of SRP-4053 in DMD Patients

Completed · Phase 1/Phase 2 · Has a placebo group

Conditions studied: Duchenne Muscular Dystrophy

In brief

This is a first-in-human, multiple-dose 2-part study to assess the safety, tolerability, efficacy, and pharmacokinetics of SRP-4053 in Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon 53 skipping.

Key facts

Study ID

NCT02310906

Run by

Sarepta Therapeutics, Inc.

People needed

39

Starts

2015-01-13

Expected to finish

2019-03-25

Last updated by the study team

2020-10-19

Who can join

Age: 6 and older, up to 15. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Diagnosed with DMD, genotypically confirmed.
• Intact right and left biceps muscles or an alternative upper arm muscle group.
• Stable pulmonary and cardiac function.
• Minimum performance on 6MWT, North Star Ambulatory Assessment, and rise (Gowers) test as specified in the study protocol.
• On a stable dose of corticosteroids for at least 6 months.

You may not qualify if…

• Previous treatment with the experimental agents BMN-195 (SMT C1100) or PRO053.
• Current or previous treatment with any other experimental treatments within 12 weeks prior to study entry.
• Major surgery within the last 3 months.
• Presence of other clinically significant illness.
• Major change in physical therapy regime within the last 3 months.
• Other inclusion and exclusion criteria may apply.

Where it is running

• Boston Children's Hospital — Boston, Massachusetts, United States
• Institute de Myologie — Paris, France
• Policlinico Universitario A Gemelli — Rome, Italy
• Great Ormond Street Hospital for Children NHS Foundation Trust — London, United Kingdom
• Newcastle University Hospital — Newcastle, United Kingdom

Full record on ClinicalTrials.gov

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