Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy

Completed · Phase 2

Conditions studied: Muscular Dystrophy, Duchenne

In brief

The primary objective of this study is to explore safety and tolerability of eteplirsen in participants with advanced stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.

Key facts

Study ID

NCT02286947

Run by

Sarepta Therapeutics, Inc.

People needed

24

Starts

2014-11-01

Expected to finish

2018-03-23

Last updated by the study team

2020-03-30

Who can join

Age: 7 and older, up to 21. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Male 7 - 21 years of age
• Diagnosis of DMD with a mutation that is amenable to exon 51 skipping, confirmed by a genetic report
• Stable dose of oral corticosteroids for at least 24 weeks or has not received corticosteroids for at least 24 weeks
• Non-ambulatory, or incapable of walking ≥300 meters on the 6-Minute Walk Test (6MWT).
• Score of ≤4 on the Brooke Score for Arms and Shoulders.
• Stable cardiac and pulmonary function
• Use of contraceptives for sexually active males throughout the study
• Willing to provide consent and comply with the study

You may not qualify if…

• Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks that may have an effect on muscle strength or function (e.g., growth hormone, anabolic steroids).
• Previous treatment with SMT C1100/BMN 195 at any time.
• Previous treatment with drisapersen (PRO051) within the last 6 months.
• Participation in any other DMD interventional clinical study within 12 weeks
• Major change in physiotherapy regimen within the past 3 months
• Major surgery within 3 months
• Presence of other clinically significant illness
• Use of an aminoglycoside antibiotic within 12 weeks or the need for this antibiotic or statin during study
• Forced vital capacity % predicted [FVC % predicted] <40%, or requiring daytime ventilation.
• Require antiarrhythmic and/or antidiuretic therapy for heart failure.
• Have a left ventricular ejection fraction (LVEF) of <40%.
• Prior or ongoing medical condition that could adversely affect the safety of the patient, make it unlikely that the course of treatment would be completed, or impair the assessment of study results.

Where it is running

• Ronald Reagan UCLA Medical Center — Los Angeles, California, United States
• University of California, Davis Medical Center — Sacramento, California, United States
• University of Iowa Children's Hospital — Iowa City, Iowa, United States
• Kennedy Krieger Institute — Baltimore, Maryland, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• St. Louis Children's Hospital — St Louis, Missouri, United States
• University of Rochester Medical Center — Rochester, New York, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Seattle Children's Hospital — Seattle, Washington, United States

Full record on ClinicalTrials.gov

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