[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"eye-diseases-hereditary\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:eye-diseases-hereditary":33},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,60],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":37,"overallStatus":47,"whyStopped":4,"lastUpdateSubmitDate":48,"lastUpdatePostDateStruct":49,"startDateStruct":52,"completionDateStruct":54,"leadSponsor":56,"locationsCount":59},"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":20,"type":21},32,"ESTIMATED","INTERVENTIONAL",[24],"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.",[27,28,29,30,31,32,33,34,35,36],"Leber Congenital Amaurosis 10","Blindness","Leber Congenital Amaurosis","Sensation Disorders","Vision Disorder","Neurological Manifestations","Eye Diseases, Hereditary","Eye Diseases","Eye Disorders Congenital","Retinal Disease",[38,39,40,41,42,43,44,45,46],"LCA10","p.Cys998X","Antisense oligonucleotides","RNA therapy","QR-110","sepofarsen","CEP290","Leber's Congenital Amaurosis","c.2991+1655A>G","RECRUITING","2026-06-24",{"date":50,"type":51},"2026-06-25","ACTUAL",{"date":53,"type":51},"2025-06-04",{"date":55,"type":21},"2028-10",{"name":57,"class":58},"Laboratoires Thea","INDUSTRY",17,{"id":61,"slug":62,"hasResults":11,"nctId":63,"briefTitle":64,"officialTitle":65,"acronym":66,"eligibilityCriteria":67,"healthyVolunteers":68,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":69,"targetDuration":71,"studyType":72,"phases":4,"briefSummary":73,"conditions":74,"keywords":101,"overallStatus":47,"whyStopped":4,"lastUpdateSubmitDate":130,"lastUpdatePostDateStruct":131,"startDateStruct":133,"completionDateStruct":135,"leadSponsor":137,"locationsCount":140},"100242565","inherited-retinal-degenerative-disease-registry-100242565","NCT02435940","Inherited Retinal Degenerative Disease Registry","Foundation Fighting Blindness My Retina Tracker Registry","MRTR","Inclusion Criteria:\n\n* Diagnosed with an inherited retinal degenerative disease OR\n\nExclusion Criteria:\n\n* Glaucoma only\n* Diabetic retinopathy only\n* Non-retinal disease\n* Not heritable retinal disease",true,{"count":70,"type":21},20000,"20 Years","OBSERVATIONAL","The My Retina Tracker® Registry is sponsored by the Foundation Fighting Blindness and is for people affected by one of the rare inherited retinal degenerative diseases studied by the Foundation. It is a patient-initiated registry accessible via a secure on-line portal at www.MyRetinaTracker.org. Affected individuals who register are guided to create a profile that captures their perspective on their retinal disease and its progress; family history; genetic testing results; preventive measures; general health and interest in participation in research studies. The participants may also choose to ask their clinician to add clinical measurements and results at each clinical visit. Participants are urged to update the information regularly to create longitudinal records of their disease, from their own perspective, and their clinical progress. The overall goals of the Registry are: to better understand the diversity within the inherited retinal degenerative diseases; to understand the prevalence of the different diseases and gene variants; to assist in the establishment of genotype-phenotype relationships; to help understand the natural history of the diseases; to help accelerate research and development of clinical trials for treatments; and to provide a tool to investigators that can assist with recruitment for research studies and clinical trials.",[75,36,76,77,78,79,80,81,82,83,84,85,86,87,88,89,90,91,29,92,93,94,95,96,97,98,99,100],"Eye Diseases Hereditary","Achromatopsia","Bardet-Biedl Syndrome","Bassen-Kornzweig Syndrome","Batten Disease","Best Disease","Choroidal Dystrophy","Choroideremia","Cone Dystrophy","Cone-Rod Dystrophy","Congenital Stationary Night Blindness","Enhanced S-Cone Syndrome","Fundus Albipunctatus","Goldmann-Favre Syndrome","Gyrate Atrophy","Juvenile Macular Degeneration","Kearns-Sayre Syndrome","Refsum Syndrome","Retinitis Pigmentosa","Retinitis Punctata Albescens","Retinoschisis","Rod-Cone Dystrophy","Rod Dystrophy","Rod Monochromacy","Stargardt Disease","Usher Syndrome",[102,103,104,105,106,107,108,109,110,111,112,113,114,115,116,117,118,119,120,121,122,123,124,125,126,127,128,129],"inherited retinal degenerative disease","retinitis pigmentosa","Usher","Leber","Bardet-Biedl","Batten","Best","cone dystrophy","cone-rod dystrophy","choroideremia","congenital night blindness","enhanced s-cone","cone monochromacy","Goldmann-Favre","Kearns-Sayre","Refsum","retinoschisis","rod-cone dystrophy","rod dystrophy","rod monochromacy","Sorsby pseudoinflammatory dystrophy","stargardt","achromatopsia","juvenile inherited macular degeneration","cone dichromacy","cone trichromacy","Charcot-Marie-Tooth","albipunctate dystrophy","2026-05-18",{"date":132,"type":51},"2026-05-19",{"date":134,"type":4},"2014-06",{"date":136,"type":21},"2037-06",{"name":138,"class":139},"Foundation Fighting Blindness","OTHER",1]