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

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: A Randomized, Double-Blind, Placebo-Controlled, Dose-Titration, Safety, Tolerability, and Pharmacokinetics Study Followed by an Open-Label Safety and Efficacy Evaluation of SRP-4045 in Advanced-Stage

Brief 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.

Detailed Summary:

This is a randomized, placebo-controlled dose-titration study to assess safety, tolerability, and pharmacokinetics of 4 dose levels of SRP-4045 in genotypically confirmed advanced-stage DMD patients with deletions amenable to exon 45 skipping.

After completion of the dose-titration portion of the study and SRP-4045 is determined to be safe, all patients will be evaluated on open-label SRP-4045 for the duration of the study.

Safety, including adverse event monitoring, routine laboratory assessments, and cardiac testing will be monitored through the duration of the dose-titration and open-label portions of the study.

Clinical efficacy will be assessed at regularly scheduled study visits via quality of life questionnaires and tests of pulmonary and upper extremity function through the duration of the dose-titration and open-label portions of the trial.

Sponsor: Sarepta Therapeutics

Current Primary Outcome: Incidence of adverse events [ Time Frame: Approximately 12 Weeks (double-blind dose titration) ]

Original Primary Outcome: Same as current

Current Secondary Outcome: Drug concentration in plasma [ Time Frame: Approximately 12 Weeks (double-blind dose titration) ]

Original Secondary Outcome: Same as current

Information By: Sarepta Therapeutics

Dates:
Date Received: August 10, 2015
Date Started: October 2015
Date Completion: April 2018
Last Updated: May 15, 2017
Last Verified: May 2017