[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"PTC Therapeutics\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":66},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,41],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":26,"conditions":27,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":31,"startDateStruct":34,"completionDateStruct":36,"leadSponsor":38,"locationsCount":4},"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":21,"type":22},120,"ESTIMATED","INTERVENTIONAL",[25],"PHASE3","The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.",[28],"Friedreich's Ataxia","NOT_YET_RECRUITING","2026-06-26",{"date":32,"type":33},"2026-07-02","ACTUAL",{"date":35,"type":22},"2026-08-15",{"date":37,"type":22},"2029-03-15",{"name":39,"class":40},"PTC Therapeutics","INDUSTRY",{"id":42,"slug":43,"hasResults":11,"nctId":44,"briefTitle":45,"officialTitle":46,"acronym":47,"eligibilityCriteria":48,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":49,"enrollmentInfo":50,"targetDuration":4,"studyType":23,"phases":52,"briefSummary":53,"conditions":54,"keywords":4,"overallStatus":56,"whyStopped":4,"lastUpdateSubmitDate":57,"lastUpdatePostDateStruct":58,"startDateStruct":60,"completionDateStruct":62,"leadSponsor":64,"locationsCount":65},"100539323","phase-3-a-study-of-sepiapterin-in-participants-with-phenylketonuria-pku-100539323","NCT06302348","A Study of Sepiapterin in Participants With Phenylketonuria (PKU)","A Phase 3b Open-Label Study of Long-Term Neurocognitive Outcomes in Children With Phenylketonuria Treated With Sepiapterin","EPIPHENY","Key Inclusion Criteria:\n\nFor all participants:\n\n* Women of childbearing potential must have a negative pregnancy test at Screening and agree to abstinence or the use of at least one highly effective form of contraception for the duration of the study, and for at least 90 days after the last dose of the study drug.\n* Willing to maintain prescribed daily protein\u002FPhe during Screening and Part 1.\n\nFor participants ≥1 month of age at Screening:\n\n* Established diagnosis of PKU with hyperphenylalaninemia (HPA) evidenced by at least 2 blood Phe measurement ≥600 micromoles (μmol)\u002Fliter (L) as documented in the medical history.\n* A minimum of 1 documented blood Phe measurement \\\u003C480 μmol\u002FL within 1 month prior to Screening.\n* Two screening blood Phe concentration values must be in the range ≥120 to ≤480 μmol\u002FL.\n\nFor participants \\\u003C1 month of age at the time of informed consent\u002Fassent only:\n\n* Blood Phe at newborn screening ≥600 μmol\u002FL.\n\nFor participants ≥30 months to \\\u003C10 years of age:\n\n* Baseline FSIQ score ≥80.\n\nKey Exclusion Criteria:\n\n* History of allergies or adverse reactions to any of the ingredients or excipients of synthetic tetrahydrobiopterin (BH4) or sepiapterin.\n* Serious neuropsychiatric illness (for example, major depression) not currently under medical control or other concurrent disease or condition that, in the opinion of the investigator or sponsor, would interfere with the participant's ability to participate in the study or increase the risk of participation for that participant.\n* Treatment with BH4 supplementation (sapropterin, KUVAN®) within 3 months prior to Screening.\n* Current participation in another investigational drug study or use of any investigational agent within 30 days prior to Screening.\n* Confirmed diagnosis of a primary BH4 deficiency as evidenced by biallelic pathogenic mutations in 6-pyruvoyltetrahydropterin synthase, recessive Guanosine-5'-triphosphate (GTP) cyclohydrolase I, sepiapterin reductase, quinoid dihydropteridine reductase, or pterin 4-alphacarbinolamine dehydratase genes.\n* Any clinically significant laboratory abnormality as determined by the investigator.\n* Any past medical history of an abnormal physical examination and\u002For laboratory findings indicative of signs or symptoms of renal disease, including calculated (Bedside Schwartz Equation) glomerular filtration rate (GFR) \\\u003C60 milliliters (mL)\u002Fminute (min)\u002F1.73 square meter (m\\^2).\n* Major surgery within 90 days prior to Screening visit.\n* Previous treatment for \\>6 weeks with sepiapterin (that is, Sephience).\n\nNote: Other protocol-defined inclusion and exclusion criteria may apply.","9 Years",{"count":51,"type":22},56,[25],"The main purpose of this trial is to evaluate the long-term efficacy of sepiapterin on preserving neurocognitive functioning in children with PKU when treatment is initiated in early childhood.",[55],"Phenylketonuria","RECRUITING","2026-06-15",{"date":59,"type":33},"2026-06-17",{"date":61,"type":33},"2024-03-04",{"date":63,"type":22},"2031-02-28",{"name":39,"class":40},8,""]