Study of Eteplirsen in DMD Patients

Completed · Phase 3

Conditions studied: Duchenne Muscular Dystrophy (DMD)

In brief

The main objective of this study is to provide evidence of efficacy of eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51. Additional objectives include evaluation of safety, biomarkers and the long-term effects of eteplirsen up to 96 weeks, followed by a safety extension (not to exceed 48 weeks).

Key facts

Study ID

NCT02255552

Run by

Sarepta Therapeutics, Inc.

People needed

109

Starts

2014-11-17

Expected to finish

2019-06-14

Last updated by the study team

2021-01-25

Who can join

Age: 7 and older, up to 16. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Male 7-16 years old
• Diagnosed with DMD, genotypically confirmed
• Stable dose of corticosteroids for at least 24 weeks
• Have intact right and left alternative upper muscle groups
• Mean 6MWT greater than 300m (primary analysis on 300 to 450 meters)
• Stable pulmonary and cardiac function: predicted FVC equal to or greater than 50% and LVEF of greater than 50%

You may not qualify if…

• Previous treatment with drisapersen or any other RNA antisense agent or any gene therapy within the last 6 months
• Participation in any other DMD interventional clinical study within 12 weeks
• Major surgery within 3 months
• Presence of other clinically significant illness
• Major change in the physical therapy regime within 3 months
• Other inclusion/exclusion criteria apply.

Where it is running

• Neuromuscular Research Center — Phoenix, Arizona, United States
• David Geffen School of Medicine at UCLA — Los Angeles, California, United States
• University of California, Davis Medical Center — Sacramento, California, United States
• Rady Children's Hospital, U.C. San Diego — San Diego, California, United States
• Stanford University School of Medicine/Medical Center — Stanford, California, United States
• Children's Hospital Colorado — Aurora, Colorado, United States
• Connecticut Children's Medical Center — Hartford, Connecticut, United States
• Children's National Health System — Washington D.C., District of Columbia, United States
• The University of Florida, Powell Gene Therapy Center — Gainesville, Florida, United States
• NW FL Clinical Research Group, LLC — Gulf Breeze, Florida, United States
• Nemours Children's Hospital — Orlando, Florida, United States
• Rare Disease Research Center — Atlanta, Georgia, United States
• Emory University — Atlanta, Georgia, United States
• Ann and Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
• University of Iowa Children's Hospital — Iowa City, Iowa, United States
• University of Kansas Medical Center — Kansas City, Kansas, United States
• Kennedy Krieger Institute — Baltimore, Maryland, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• Children's Hospital Boston — Boston, Massachusetts, United States
• Children's Hospital of Michigan — Detroit, Michigan, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• St. Louis Children's Hospital — St Louis, Missouri, United States
• Dartmouth-Hitchcock Medical Center — Lebanon, New Hampshire, United States
• Columbia University Medical Center — New York, New York, United States
• University of Rochester Clinical Research Center — Rochester, New York, United States

Full record on ClinicalTrials.gov

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