EPI-743 for Mitochondrial Respiratory Chain Diseases
- Conditions
- Mitochondrial Diseases
- Registration Number
- NCT01370447
- Lead Sponsor
- PTC Therapeutics
- Brief Summary
This study will evaluate the safety and efficacy of EPI-743 in participants with severe mitochondrial respiratory chain diseases who are considered to be within 90 days of end-of-life care.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- NO_LONGER_AVAILABLE
- Sex
- All
- Target Recruitment
- Not specified
Not provided
Not provided
Study & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (16)
UCSD
🇺🇸San Diego, California, United States
Akron Children's Hospital
🇺🇸Akron, Ohio, United States
Emory University
🇺🇸Decatur, Georgia, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
MUSC
🇺🇸North Charleston, South Carolina, United States
UTH
🇺🇸Houston, Texas, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
CHOC Children's Clinic
🇺🇸Orange, California, United States
UCLA
🇺🇸Los Angeles, California, United States
Lucille Packard Children's Hospital
🇺🇸Palo Alto, California, United States
CUMC (Columbia University)
🇺🇸New York, New York, United States
Stanford Children's Health
🇺🇸Stanford, California, United States
Carolinas Medical Center
🇺🇸Charlotte, North Carolina, United States
Cleveland Clinic
🇺🇸Cleveland, Ohio, United States
Naval Hospital, Bremerton
🇺🇸Bremerton, Washington, United States
Baylor College of Medicine
🇺🇸Houston, Texas, United States