A Phase 2B/3 Open-label Study of EPI-743 in Children with Leigh Syndrome
Phase 2
- Conditions
- eigh Syndrome
- Registration Number
- JPRN-jRCT2080222300
- Lead Sponsor
- Sumitomo Dainippon Pharma Co., Ltd.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Sex
- All
- Target Recruitment
- 5
Inclusion Criteria
- Clinical and MRI diagnosis of Leigh syndrome
etc.
Exclusion Criteria
- Allergy to EPI-743 or sesame oil
- Allergy to vitamin E
- Diagnosis of any other concurrent inborn error of metabolism
etc.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method ewcastle Pediatric Mitochondrial Disease Scale(NPMDS) (Sections 1-3)<br>Amount of change in NPMDS(Sections 1-3) total score of each evaluation period from baseline
- Secondary Outcome Measures
Name Time Method Barry-Albright Dystonia Scale<br>Gross Motor Function Measure<br>Need for tracheostomy<br>Number of deaths<br>Total number of hospitalizations<br>Glutathione cycle biomarkers <br>Adverse events<br>etc.<br>Amount of change in Barry-Albright Dystonia Scale total score of each evaluation period from baseline<br>etc.