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

• Status: Completed
• Trial end date: 2018-10-03
• Target enrollment: 0
• Participant gender: Male

Summary

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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Sarepta Therapeutics; Sarepta Therapeutics, Inc.

Criteria

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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.