Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients

Completed · Phase 1 · Has a placebo group

Conditions studied: Duchenne Muscular Dystrophy

In brief

This is a first-in-human dose-titration and open-label extension study to assess safety, tolerability, and pharmacokinetics of SRP-4045 in advanced-stage Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon 45 skipping.

Key facts

Study ID

NCT02530905

Run by

Sarepta Therapeutics, Inc.

People needed

12

Starts

2015-10-08

Expected to finish

2018-10-03

Last updated by the study team

2021-05-17

Who can join

Age: 7 and older, up to 21. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Genotypically confirmed DMD (amenable to exon 45 skipping).
• Stable cardiac and pulmonary function.
• Limited or no ambulation.
• On a stable dose of oral corticosteroids for at least 24 weeks OR has not received corticosteroids for at least 24 weeks.

You may not qualify if…

• Current or previous treatment with the experimental agents SMT C1100 (BMN-195) or PRO045.
• Other experimental treatment in the past 12 weeks.
• If on cardiac medication, must be on a stable dose for the past 12 weeks.
• Major surgery within the past 3 months.
• Other inclusion/exclusion criteria apply.

Where it is running

• David Geffen School of Medicine at UCLA — Los Angeles, California, United States
• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
• Kennedy Krieger Institute — Baltimore, Maryland, United States

Full record on ClinicalTrials.gov

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