[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100584599":3},{"organization":4,"armGroups":7,"interventions":37,"overallOfficials":50,"centralContacts":51,"locations":59,"responsibleParty":241,"collaborators":243,"id":246,"slug":247,"hasResults":248,"nctId":249,"briefTitle":250,"officialTitle":251,"acronym":252,"eligibilityCriteria":253,"healthyVolunteers":248,"sex":254,"minAge":255,"maxAge":50,"enrollmentInfo":256,"targetDuration":50,"studyType":259,"phases":260,"briefSummary":262,"conditions":263,"keywords":274,"overallStatus":62,"whyStopped":50,"lastUpdateSubmitDate":282,"lastUpdatePostDateStruct":283,"startDateStruct":286,"completionDateStruct":288,"leadSponsor":290,"locationsCount":291},{"fullName":5,"class":6},"Laboratoires Thea","INDUSTRY",[8,14,19,23,26,30,33],{"label":9,"type":10,"description":11,"interventionNames":12},"Sepofarsen - Treatment Eye - up to Month 12","ACTIVE_COMPARATOR","Subjects to receive Sepofarsen in one eye (160µg first then 40µg) and Placebo in the fellow eye at baseline and at month 6.",[13],"Drug: sepofarsen",{"label":15,"type":16,"description":11,"interventionNames":17},"Placebo - 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Hospital São Paulo (UNIFESP-HSP)","São Paulo","04023-062",{"type":69,"coordinates":142},[143,144],-46.63611,-23.5475,{"lat":144,"lon":143},{"facility":147,"status":62,"city":148,"state":149,"zip":150,"country":151,"countryCode":152,"cosmosGeoPoint":153,"geoPoint":157,"contacts":50},"University of Alberta","Edmonton","Alberta","T6G 2C8","Canada","CA",{"type":69,"coordinates":154},[155,156],-113.46871,53.55014,{"lat":156,"lon":155},{"facility":159,"status":62,"city":160,"state":161,"zip":162,"country":151,"countryCode":152,"cosmosGeoPoint":163,"geoPoint":167,"contacts":50},"The Hospital for Sick Children - SickKids","Toronto","Ontario","M5G 2L3",{"type":69,"coordinates":164},[165,166],-79.39864,43.70643,{"lat":166,"lon":165},{"facility":169,"status":62,"city":170,"state":50,"zip":171,"country":172,"countryCode":173,"cosmosGeoPoint":174,"geoPoint":178,"contacts":50},"Centre de maladies rares CHNO des Quinze Vingt","Paris","75012","France","FR",{"type":69,"coordinates":175},[176,177],2.3488,48.85341,{"lat":177,"lon":176},{"facility":180,"status":62,"city":181,"state":50,"zip":182,"country":183,"countryCode":184,"cosmosGeoPoint":185,"geoPoint":189,"contacts":50},"Justus-Liebig Universität - Department of Ophthalmology","Giessen","35392","Germany","DE",{"type":69,"coordinates":186},[187,188],8.67554,50.58727,{"lat":188,"lon":187},{"facility":191,"status":62,"city":192,"state":50,"zip":193,"country":183,"countryCode":184,"cosmosGeoPoint":194,"geoPoint":198,"contacts":50},"Klinikum der Ludwig-Maximilian Universität München","München","81377",{"type":69,"coordinates":195},[196,197],13.31243,51.60698,{"lat":197,"lon":196},{"facility":200,"status":62,"city":201,"state":50,"zip":202,"country":183,"countryCode":184,"cosmosGeoPoint":203,"geoPoint":207,"contacts":50},"University of Tuebingen - Inst. for Ophthalmic Research","Tübingen","72076",{"type":69,"coordinates":204},[205,206],9.05222,48.52266,{"lat":206,"lon":205},{"facility":209,"status":62,"city":210,"state":50,"zip":211,"country":212,"countryCode":213,"cosmosGeoPoint":214,"geoPoint":218,"contacts":50},"Radboud Universitair Medisch Centrum","Nijmegen","6525 GA","Netherlands","NL",{"type":69,"coordinates":215},[216,217],5.85278,51.8425,{"lat":217,"lon":216},{"facility":220,"status":62,"city":221,"state":50,"zip":222,"country":223,"countryCode":224,"cosmosGeoPoint":225,"geoPoint":229,"contacts":50},"Hospital Sant Joan de Déu (SJD Barcelona Children's Hospital)","Barcelona","08950","Spain","ES",{"type":69,"coordinates":226},[227,228],2.15899,41.38879,{"lat":228,"lon":227},{"facility":231,"status":62,"city":232,"state":50,"zip":233,"country":234,"countryCode":235,"cosmosGeoPoint":236,"geoPoint":240,"contacts":50},"Moorfields Eye Hospital NHS Foundation Trust","London","EC1V 2PD","United Kingdom","UK",{"type":69,"coordinates":237},[238,239],-0.12574,51.50853,{"lat":239,"lon":238},{"type":242,"investigatorFullName":50,"investigatorTitle":50,"investigatorAffiliation":50,"oldNameTitle":50,"oldOrganization":50},"SPONSOR",[244],{"name":245,"class":6},"Sepul Bio","100584599","phase-3-study-to-evaluate-sepofarsen-in-subjects-with-leber-congenital-amaurosis-lca-type-10-hyperion-100584599",false,"NCT06891443","Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)","A Double-Masked, Randomized, Placebo-Controlled, Paired-Eye Study to Evaluate the Efficacy, Safety and Tolerability of Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Due to the c.2991+1655A>G (p.Cys998X) Mutation in the CEP290 Gene","HYPERION","Inclusion Criteria:\n\n1. Confirmed clinical diagnosis of LCA10 and a molecular diagnosis of homozygosity or compound heterozygosity for the c.2991+1655A\\>G mutation in CEP290.\n2. Adults: \\>=18 years \u002F Minors: 6 to \\\u003C18 years.\n3. BCVA (FrACT) equal to or worse than logMAR +0.4 (approximate Snellen equivalent 20\u002F50) to +2.9 logMAR based on quantifiable, reliable FrACT. LP subjects with documented evidence of prior better vision eligible.\n4. Symmetrical disease between the two eyes as defined by a BCVA (FrACT) within 0.2 logMAR at baseline.\n5. Detectable ONL in the macular area as determined by the CRC at Screening.\n\nExclusion Criteria:\n\n1. Mutations in genes other than the CEP290 gene associated with other IRD diseases or syndromes.\n2. Presence of any ocular pathology in either eye that may make comparison of the eyes not feasible.\n3. Presence of unstable concurrent CME, or subject started on (or changed dose of) topical or systemic carbonic anhydrase inhibitor treatment in the 3 months prior to enrollment. CME is allowed if stable for 3 months (with or without treatment).\n4. Presence of any clinically significant lens opacities\u002Fcataracts based on the AREDS lens grading scale.\n5. Any prior receipt of genetic (RNA or DNA therapy) or stem-cell therapy for ocular or non-ocular disease, including sepofarsen.","ALL","6 Years",{"count":257,"type":258},32,"ESTIMATED","INTERVENTIONAL",[261],"PHASE3","The purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subjects with Leber Congenital Amaurosis (LCA) due to the c.2991+1655A\\>G (p.Cys998X) mutation in the CEP290.",[264,265,266,267,268,269,270,271,272,273],"Leber Congenital Amaurosis 10","Blindness","Leber Congenital Amaurosis","Sensation Disorders","Vision Disorder","Neurological Manifestations","Eye Diseases, Hereditary","Eye Diseases","Eye Disorders Congenital","Retinal Disease",[275,276,277,278,44,40,279,280,281],"LCA10","p.Cys998X","Antisense oligonucleotides","RNA therapy","CEP290","Leber's Congenital Amaurosis","c.2991+1655A>G","2026-06-24",{"date":284,"type":285},"2026-06-25","ACTUAL",{"date":287,"type":285},"2025-06-04",{"date":289,"type":258},"2028-10",{"name":5,"class":6},17]