Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)
ConditionFriedreich's Ataxia
Trial statusNot yet recruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age7-21
SponsorPTC Therapeutics
About this trial
The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.
Eligibility criteria
Qualifiers
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.
Disqualifiers
Individuals with clinical diagnosis of FA who have point mutations, deletions, or other non-GAA expansion mutations.
Allergy to vatiquinone, sesame oil, gelatin (bovine and/or porcine), titanium dioxide, or red iron oxide.
Pregnant or lactating participants or those sexually active participants who are unwilling to comply with proper birth control methods; females of childbearing potential must have a negative pregnancy test at Screening and during the Baseline Visit.
Comorbidities that may confound study results (for example, fat malabsorption syndrome, other mitochondrial disorder) in the opinion of the investigator.
Trial design
Treatments tested in this trial
- Vatiquinone
Treatment groups
120 Participants
are divided into 1 treatment groupLocations
Sponsors and collaborators
Source ClinicalTrials.gov