[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100645351":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":21,"centralContacts":22,"locations":28,"responsibleParty":157,"collaborators":21,"id":159,"slug":160,"hasResults":161,"nctId":162,"briefTitle":163,"officialTitle":164,"acronym":165,"eligibilityCriteria":166,"healthyVolunteers":161,"sex":167,"minAge":168,"maxAge":169,"enrollmentInfo":170,"targetDuration":21,"studyType":173,"phases":174,"briefSummary":176,"conditions":177,"keywords":21,"overallStatus":179,"whyStopped":21,"lastUpdateSubmitDate":180,"lastUpdatePostDateStruct":181,"startDateStruct":184,"completionDateStruct":186,"leadSponsor":188,"locationsCount":189},{"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",[29,46,59,72,85,99,114,129,143],{"facility":30,"status":21,"city":31,"state":32,"zip":33,"country":34,"countryCode":35,"cosmosGeoPoint":36,"geoPoint":41,"contacts":42},"University of California Los Angeles","Los Angeles","California","90095","United States","US",{"type":37,"coordinates":38},"Point",[39,40],-118.24368,34.05223,{"lat":40,"lon":39},[43],{"name":44,"role":45,"phone":21,"phoneExt":21,"email":21},"Susan 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França",{"facility":115,"status":21,"city":116,"state":117,"zip":118,"country":119,"countryCode":120,"cosmosGeoPoint":121,"geoPoint":125,"contacts":126},"Centre Hospitalier de l'Universite de Montreal CHUM","Montreal","Quebec","H2X 0A9","Canada","CA",{"type":37,"coordinates":122},[123,124],-73.58781,45.50884,{"lat":124,"lon":123},[127],{"name":128,"role":45,"phone":21,"phoneExt":21,"email":21},"Antoine Duquette",{"facility":130,"status":21,"city":131,"state":21,"zip":132,"country":133,"countryCode":134,"cosmosGeoPoint":135,"geoPoint":139,"contacts":140},"Institut du Cerveau et de la Moelle Epinière","Paris","75013","France","FR",{"type":37,"coordinates":136},[137,138],2.3488,48.85341,{"lat":138,"lon":137},[141],{"name":142,"role":45,"phone":21,"phoneExt":21,"email":21},"Alexandra Durr",{"facility":144,"status":21,"city":145,"state":21,"zip":146,"country":147,"countryCode":148,"cosmosGeoPoint":149,"geoPoint":153,"contacts":154},"Hospital Sant Joan de Deu","Barcelona","08950","Spain","ES",{"type":37,"coordinates":150},[151,152],2.15899,41.38879,{"lat":152,"lon":151},[155],{"name":156,"role":45,"phone":21,"phoneExt":21,"email":21},"Alejandra Darling",{"type":158,"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":171,"type":172},120,"ESTIMATED","INTERVENTIONAL",[175],"PHASE3","The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.",[178],"Friedreich's Ataxia","NOT_YET_RECRUITING","2026-08-12",{"date":182,"type":183},"2026-08-14","ACTUAL",{"date":185,"type":172},"2026-08-15",{"date":187,"type":172},"2029-03-15",{"name":5,"class":6},9]