Evaluation of Long-Term Efficacy of Vatiquinone for the Treatment of Friedreich's Ataxia
PTC Therapeutics
Summary
The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.
Eligibility
- Age range
- 7–21 years
- Sex
- All
- Healthy volunteers
- No
Key Inclusion Criteria: * mFARS ≥20 to ≤70 at Screening (4 weeks prior to Day 1) and Baseline (Day 1). * Must be ambulatory as defined by a E7 score of 4 or less on the USS at Screening and Baseline. * Documentation that participants reached maximum score on items E4, E5, and E3b on the USS of mFARS at Screening and Baseline. * FA diagnosis (homozygous for guanine-adenine-adenine \[GAA\] repeat expansion in intron-1 of the frataxin gene), confirmed and documented with GAA repeat length for both alleles by clinical genetic testing. * Difference in the mFARS score between Screening and Baseline…