Last updated 6 days ago
Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)
120 patients around the world
Available in Brazil, United States
PTC Therapeutics
1Research sites
120Patients around the world
This study is for people with
Ataxia
Friedreich's ataxia
Requirements for the patient
To 21 Years
All Gender
Medical requirements
- 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 of no more than 4 points.Ability to abstain from strong cytochrome P450 (CYP) 3A4 inducers/inhibitors (for example, ketoconazole, rifampin, St. John's wort, grapefruit juice) for at least 4 weeks prior to Baseline and for the duration of the study.
- 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.Participation in an interventional clinical study or received investigational drug (other than SKYCLARYS® [omaveloxolone]) within 60 days prior to Screening. Participants may be screened after the exclusionary period of 60 days has passed.Current use of omaveloxolone. Previous use of omaveloxolone will be allowed if.Use was less than 3 cumulative months and participants have been off treatment for >30 days.Use was less than 6 cumulative months and participants have been off treatment for >90 days.Previous or concurrent use of other investigational treatment for FA.Participation in any cell or gene therapy-based treatment for FA.Participation in an ongoing study for vatiquinone or current or previous use of vatiquinone.Illicit drug use 30 days prior to Screening and during the study.
Sites
Hospital de Clínicas - Universidade Estadual de Campinas - UNICAMP
StudyPROVE-FA
SponsorPTC Therapeutics
Study typeInterventional
Conditions
Friedreich's ataxia
Requirements
To 21 YearsAll Gender
Unique study IDclinicaltrials.gov
NCT07681713
