[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100539323":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":22,"centralContacts":23,"locations":29,"responsibleParty":144,"collaborators":22,"id":146,"slug":147,"hasResults":148,"nctId":149,"briefTitle":150,"officialTitle":151,"acronym":152,"eligibilityCriteria":153,"healthyVolunteers":148,"sex":154,"minAge":22,"maxAge":155,"enrollmentInfo":156,"targetDuration":22,"studyType":159,"phases":160,"briefSummary":162,"conditions":163,"keywords":22,"overallStatus":32,"whyStopped":22,"lastUpdateSubmitDate":165,"lastUpdatePostDateStruct":166,"startDateStruct":169,"completionDateStruct":171,"leadSponsor":173,"locationsCount":174},{"fullName":5,"class":6},"PTC Therapeutics","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Sepiapterin","EXPERIMENTAL","Participants will receive age- and weight-adjusted doses of sepiapterin orally once daily for up to 6 years.",[13],"Drug: Sepiapterin",[15],{"type":16,"name":9,"description":17,"armGroupLabels":18,"otherNames":19},"DRUG","Sepiapterin powder for oral use will be mixed in water or apple juice prior to administration.",[9],[20,21],"PTC923","Sephience",null,[24],{"name":25,"role":26,"phone":27,"phoneExt":22,"email":28},"Patient Advocacy","CONTACT","1-866-562-4620","medinfo@ptcbio.com",[30,50,64,76,90,104,118,130],{"facility":31,"status":32,"city":33,"state":34,"zip":35,"country":36,"countryCode":37,"cosmosGeoPoint":38,"geoPoint":43,"contacts":44},"Indiana University School of Medicine","RECRUITING","Indianapolis","Indiana","46202","United States","US",{"type":39,"coordinates":40},"Point",[41,42],-86.15804,39.76838,{"lat":42,"lon":41},[45,47],{"name":46,"role":26,"phone":22,"phoneExt":22,"email":22},"Susan Romie",{"name":48,"role":49,"phone":22,"phoneExt":22,"email":22},"Melissa Lah","PRINCIPAL_INVESTIGATOR",{"facility":51,"status":32,"city":52,"state":22,"zip":53,"country":54,"countryCode":55,"cosmosGeoPoint":56,"geoPoint":60,"contacts":61},"Women and Children Hospital","North Adelaide","SA 5006","Australia","AU",{"type":39,"coordinates":57},[58,59],138.59141,-34.90733,{"lat":59,"lon":58},[62],{"name":63,"role":49,"phone":22,"phoneExt":22,"email":22},"Drago Bratkovic",{"facility":65,"status":32,"city":66,"state":22,"zip":67,"country":54,"countryCode":55,"cosmosGeoPoint":68,"geoPoint":72,"contacts":73},"The Royal Children's Hospital","Parkville","VIC 3052",{"type":39,"coordinates":69},[70,71],144.95,-37.78333,{"lat":71,"lon":70},[74],{"name":75,"role":49,"phone":22,"phoneExt":22,"email":22},"Heidi Peters",{"facility":77,"status":32,"city":78,"state":22,"zip":79,"country":80,"countryCode":81,"cosmosGeoPoint":82,"geoPoint":86,"contacts":87},"Centre Hospitalier Régional Universitaire (CHRU) de Tours - Hôpital Clocheville","Tours","37044","France","FR",{"type":39,"coordinates":83},[84,85],0.70398,47.39484,{"lat":85,"lon":84},[88],{"name":89,"role":49,"phone":22,"phoneExt":22,"email":22},"François Labarthe, Dr.",{"facility":91,"status":32,"city":92,"state":22,"zip":93,"country":94,"countryCode":95,"cosmosGeoPoint":96,"geoPoint":100,"contacts":101},"Children's Health Ireland (CHI)","Dublin","D01 YC67","Ireland","IE",{"type":39,"coordinates":97},[98,99],-6.24889,53.33306,{"lat":99,"lon":98},[102],{"name":103,"role":49,"phone":22,"phoneExt":22,"email":22},"Joanne Hughes, Dr.",{"facility":105,"status":32,"city":106,"state":22,"zip":107,"country":108,"countryCode":109,"cosmosGeoPoint":110,"geoPoint":114,"contacts":115},"Pomorski Uniwersytet Medyczny w Szczecinie","Szczecin","70-204","Poland","PL",{"type":39,"coordinates":111},[112,113],14.55302,53.42894,{"lat":113,"lon":112},[116],{"name":117,"role":49,"phone":22,"phoneExt":22,"email":22},"Maria Gizewska",{"facility":119,"status":32,"city":120,"state":22,"zip":121,"country":108,"countryCode":109,"cosmosGeoPoint":122,"geoPoint":126,"contacts":127},"Instytut Matki i Dziecka","Warsaw","01-211",{"type":39,"coordinates":123},[124,125],21.01178,52.22977,{"lat":125,"lon":124},[128],{"name":129,"role":49,"phone":22,"phoneExt":22,"email":22},"Joanna Taybert, Dr.",{"facility":131,"status":32,"city":132,"state":22,"zip":133,"country":134,"countryCode":135,"cosmosGeoPoint":136,"geoPoint":140,"contacts":141},"Birmingham Women's and Children's NHS Foundation Trust","Birmingham","B4 6NH","United Kingdom","UK",{"type":39,"coordinates":137},[138,139],-1.89983,52.48142,{"lat":139,"lon":138},[142],{"name":143,"role":49,"phone":22,"phoneExt":22,"email":22},"Suresh Vijay",{"type":145,"investigatorFullName":22,"investigatorTitle":22,"investigatorAffiliation":22,"oldNameTitle":22,"oldOrganization":22},"SPONSOR","100539323","phase-3-a-study-of-sepiapterin-in-participants-with-phenylketonuria-pku-100539323",false,"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.","ALL","9 Years",{"count":157,"type":158},56,"ESTIMATED","INTERVENTIONAL",[161],"PHASE3","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.",[164],"Phenylketonuria","2026-06-15",{"date":167,"type":168},"2026-06-17","ACTUAL",{"date":170,"type":168},"2024-03-04",{"date":172,"type":158},"2031-02-28",{"name":5,"class":6},8]