Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy

• Status: Completed
• Trial end date: 2017-08-16
• Target enrollment: 0
• Participant gender: Male

Summary

The primary objective of this study is to assess the ongoing efficacy, safety, and tolerability of an additional 212 weeks of treatment with eteplirsen injection in Duchenne muscular dystrophy (DMD) subjects who have successfully completed the 28 week eteplirsen study: Study 4658-us-201. This study will also evaluate the correlation between biomarkers for DMD and the clinical status of participating DMD subjects.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

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

Criteria

Inclusion Criteria:

A subject must meet all of the following criteria to be eligible for this study.

1. The subject and/or their parent/legal guardian are willing and able to provide signed informed consent.

2. The subject has successfully completed 28 weeks of treatment in Study 4658-US-201.

3. The subject has a parent(s) or legal guardian(s) who is able to understand and comply with all of the study procedure requirements.

Exclusion Criteria:

A subject who meets any of the following criteria will be excluded from this study.

1. The subject has a prior or ongoing medical condition that, in the Investigator's opinion, could adversely affect the safety of the subject or make it unlikely that the course of treatment or follow-up would be completed or impair the assessment of study results.