Status:
COMPLETED
Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy
Lead Sponsor:
Sarepta Therapeutics, Inc.
Conditions:
Muscular Dystrophy, Duchenne
Eligibility:
MALE
7-21 years
Phase:
PHASE2
Brief Summary
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.
Detailed Description
This is an open-label, multi-center study to explore the safety and tolerability of eteplirsen injection in participants with advanced stage DMD with confirmed genetic mutations amenable to treatment ...
Eligibility Criteria
Inclusion
- 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
Exclusion
- 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.
Key Trial Info
Start Date :
November 1 2014
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 23 2018
Estimated Enrollment :
24 Patients enrolled
Trial Details
Trial ID
NCT02286947
Start Date
November 1 2014
End Date
March 23 2018
Last Update
March 30 2020
Active Locations (9)
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1
Ronald Reagan UCLA Medical Center
Los Angeles, California, United States, 90095
2
University of California, Davis Medical Center
Sacramento, California, United States, 95817
3
University of Iowa Children's Hospital
Iowa City, Iowa, United States, 52242
4
Kennedy Krieger Institute
Baltimore, Maryland, United States, 21205