Status:
COMPLETED
Study of Eteplirsen in DMD Patients
Lead Sponsor:
Sarepta Therapeutics, Inc.
Conditions:
Duchenne Muscular Dystrophy (DMD)
Eligibility:
MALE
7-16 years
Phase:
PHASE3
Brief Summary
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 ...
Detailed Description
This is an open-label, multi-center study to evaluate the efficacy and safety of eteplirsen in patients with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable t...
Eligibility Criteria
Inclusion
- 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%
Exclusion
- 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.
Key Trial Info
Start Date :
November 17 2014
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 14 2019
Estimated Enrollment :
109 Patients enrolled
Trial Details
Trial ID
NCT02255552
Start Date
November 17 2014
End Date
June 14 2019
Last Update
January 25 2021
Active Locations (37)
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1
Neuromuscular Research Center
Phoenix, Arizona, United States, 85028
2
David Geffen School of Medicine at UCLA
Los Angeles, California, United States, 90095
3
University of California, Davis Medical Center
Sacramento, California, United States, 95817
4
Rady Children's Hospital, U.C. San Diego
San Diego, California, United States, 92130