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

Completed · Phase 2

Conditions studied: Duchenne Muscular Dystrophy (DMD)

In brief

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.

Key facts

Study ID

NCT01540409

Run by

Sarepta Therapeutics, Inc.

People needed

12

Starts

2012-02-27

Expected to finish

2017-08-16

Last updated by the study team

2020-03-30

Who can join

Age: 7 and older, up to 13. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• A subject must meet all of the following criteria to be eligible for this study.
• The subject and/or their parent/legal guardian are willing and able to provide signed informed consent.
• The subject has successfully completed 28 weeks of treatment in Study 4658-US-201.
• The subject has a parent(s) or legal guardian(s) who is able to understand and comply with all of the study procedure requirements.

You may not qualify if…

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

Where it is running

• Miller Children's Hospital — Long Beach, California, United States
• University of Florida Clinical Research Center — Gainesville, Florida, United States
• Rush University Medical Center — Chicago, Illinois, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• Washington University Medical School — St Louis, Missouri, United States
• Summerwood Pediatrics/Infusacare Medical Services — Liverpool, New York, United States
• Levine Children's Hospital — Charlotte, North Carolina, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Children's Hospital of Pittsburgh of UPMC — Pittsburgh, Pennsylvania, United States
• Children's Specialty Group, Pediatric Neurology — Norfolk, Virginia, United States
• Osceola Medical Center — Osceola, Wisconsin, United States

Full record on ClinicalTrials.gov

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