Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients
|Study Design:||Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|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 Patients With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping|
- Incidence of adverse events [ Time Frame: Approximately 12 Weeks (double-blind dose titration) ]
- Drug concentration in plasma [ Time Frame: Approximately 12 Weeks (double-blind dose titration) ]
|Study Start Date:||October 2015|
|Estimated Study Completion Date:||April 2018|
|Primary Completion Date:||March 2016 (Final data collection date for primary outcome measure)|
Experimental: SRP-4045 (double-blind dose titration)
Approximately 8 genotypically confirmed DMD patients amenable to exon 45 skipping
Placebo Comparator: Placebo (double-blind dose titration)
Approximately 4 genotypically confirmed DMD patients amenable to exon 45 skipping.
Experimental: SRP-4045 (open label)
Approximately 12 DMD patients who completed the double-blind dose titration part of the study.
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02530905
|United States, California|
|David Geffen School of Medicine at UCLA|
|Los Angeles, California, United States, 90095|
|United States, Illinois|
|Ann & Robert H. Lurie Children's Hospital of Chicago|
|Chicago, Illinois, United States, 60611|
|United States, Maryland|
|Kennedy Krieger Institute|
|Baltimore, Maryland, United States, 21205|
|Study Director:||Genevieve Laforet, MD, PhD||Sarepta Therapeutics|
|Study Chair:||Edward Kaye, MD||Sarepta Therapeutics|