Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients
|ClinicalTrials.gov Identifier: NCT02530905|
Recruitment Status : Active, not recruiting
First Posted : August 21, 2015
Last Update Posted : August 23, 2017
|Condition or disease||Intervention/treatment||Phase|
|Duchenne Muscular Dystrophy||Drug: SRP-4045 Drug: Placebo||Phase 1|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||12 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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|
|Study Start Date :||October 2015|
|Actual Primary Completion Date :||March 2016|
|Estimated Study Completion Date :||March 2020|
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.
- 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) ]
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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:||Jon Lu, MD, PhD||Sarepta Therapeutics|
|Study Director:||Catherine Stehman-Breen, MD, MS||Sarepta Therapeutics|