[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100645351":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":21,"centralContacts":22,"locations":21,"responsibleParty":28,"collaborators":21,"id":30,"slug":31,"hasResults":32,"nctId":33,"briefTitle":34,"officialTitle":35,"acronym":36,"eligibilityCriteria":37,"healthyVolunteers":32,"sex":38,"minAge":39,"maxAge":40,"enrollmentInfo":41,"targetDuration":21,"studyType":44,"phases":45,"briefSummary":47,"conditions":48,"keywords":21,"overallStatus":50,"whyStopped":21,"lastUpdateSubmitDate":51,"lastUpdatePostDateStruct":52,"startDateStruct":55,"completionDateStruct":57,"leadSponsor":59,"locationsCount":21},{"fullName":5,"class":6},"PTC Therapeutics","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Vatiquinone","EXPERIMENTAL","Participants will receive vatiquinone capsule at a dose of either 200 milligrams (mg) orally 3 times a day (TID) if weighing ˂25 kilograms (kg) or 400 mg orally TID if weighing ≥25 kg for 24 months.",[13],"Drug: Vatiquinone",[15],{"type":16,"name":9,"description":17,"armGroupLabels":18,"otherNames":19},"DRUG","Vatiquinone will be administered per dose and schedule specified in the arm.",[9],[20],"PTC743",null,[23],{"name":24,"role":25,"phone":26,"phoneExt":21,"email":27},"Patient Advocacy","CONTACT","1-866-562-4620","medinfo@ptcbio.com",{"type":29,"investigatorFullName":21,"investigatorTitle":21,"investigatorAffiliation":21,"oldNameTitle":21,"oldOrganization":21},"SPONSOR","100645351","phase-3-long-term-efficacy-study-of-vatiquinone-for-the-treatment-of-friedreichs-ataxia-fa-100645351",false,"NCT07681713","Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)","Evaluation of Long-Term Efficacy of Vatiquinone for the Treatment of Friedreich's Ataxia","PROVE-FA","Key Inclusion Criteria:\n\n* mFARS ≥20 to ≤70 at Screening (4 weeks prior to Day 1) and Baseline (Day 1).\n* Must be ambulatory as defined by a E7 score of 4 or less on the USS at Screening and Baseline.\n* Documentation that participants reached maximum score on items E4, E5, and E3b on the USS of mFARS at Screening and Baseline.\n* 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.\n* Difference in the mFARS score between Screening and Baseline of no more than 4 points.\n* Ability to abstain from strong cytochrome P450 (CYP) 3A4 inducers\u002Finhibitors (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.\n\nKey Exclusion Criteria:\n\n* Individuals with clinical diagnosis of FA who have point mutations, deletions, or other non-GAA expansion mutations.\n* Allergy to vatiquinone, sesame oil, gelatin (bovine and\u002For porcine), titanium dioxide, or red iron oxide.\n* 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.\n* Comorbidities that may confound study results (for example, fat malabsorption syndrome, other mitochondrial disorder) in the opinion of the investigator.\n* 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.\n* Current use of omaveloxolone. Previous use of omaveloxolone will be allowed if:\n\n  1. Use was less than 3 cumulative months and participants have been off treatment for \\>30 days.\n  2. Use was less than 6 cumulative months and participants have been off treatment for \\>90 days\n* Previous or concurrent use of other investigational treatment for FA.\n* Participation in any cell or gene therapy-based treatment for FA.\n* Participation in an ongoing study for vatiquinone or current or previous use of vatiquinone.\n* Illicit drug use 30 days prior to Screening and during the study.\n\nNote: Other protocol-defined inclusion and exclusion criteria may apply.","ALL","7 Years","21 Years",{"count":42,"type":43},120,"ESTIMATED","INTERVENTIONAL",[46],"PHASE3","The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.",[49],"Friedreich's Ataxia","NOT_YET_RECRUITING","2026-06-26",{"date":53,"type":54},"2026-07-02","ACTUAL",{"date":56,"type":43},"2026-08-15",{"date":58,"type":43},"2029-03-15",{"name":5,"class":6}]