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    "canonicalTrial": {
      "canonicalId": "NCT06891443",
      "title": "Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)",
      "scientificTitle": "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",
      "status": "recruiting",
      "studyType": "interventional",
      "phase": [
        "PHASE3"
      ],
      "conditions": [
        "Leber Congenital Amaurosis 10",
        "Blindness",
        "Leber Congenital Amaurosis",
        "Sensation Disorders",
        "Vision Disorder",
        "Neurological Manifestations",
        "Eye Diseases, Hereditary",
        "Eye Diseases",
        "Eye Disorders Congenital",
        "Retinal Disease"
      ],
      "interventions": [
        {
          "name": "sepofarsen",
          "type": "DRUG",
          "description": "RNA antisense oligonucleotide for intravitreal injection"
        },
        {
          "name": "Placebo IVT",
          "type": "OTHER",
          "description": "Placebo with identical appearance to sepofarsen"
        }
      ],
      "sponsor": {
        "name": "Laboratoires Thea",
        "type": "INDUSTRY"
      },
      "locations": [
        {
          "city": "San Francisco",
          "country": "United States",
          "facility": "UCSF Wayne and Gladys Valley Center for Vision"
        },
        {
          "city": "Miami",
          "country": "United States",
          "facility": "University of Miami - Bascom Palmer Eye Institute"
        },
        {
          "city": "Iowa City",
          "country": "United States",
          "facility": "University of Iowa"
        },
        {
          "city": "Minneapolis",
          "country": "United States",
          "facility": "University of Minnesota Medical School"
        },
        {
          "city": "Philadelphia",
          "country": "United States",
          "facility": "University of Pennsylvania - Center for Advanced Retinal & Ocular Therapeutics"
        },
        {
          "city": "Sydney",
          "country": "Australia",
          "facility": "Sydney Eye Hospital"
        },
        {
          "city": "Ghent",
          "country": "Belgium",
          "facility": "Universitair Ziekenhuis Gent (UZ)"
        },
        {
          "city": "Belo Horizonte",
          "country": "Brazil",
          "facility": "INRET Clínica/ Santa Casa de Misericórdia de Belo Horizonte"
        },
        {
          "city": "São Paulo",
          "country": "Brazil",
          "facility": "Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP)"
        },
        {
          "city": "Edmonton",
          "country": "Canada",
          "facility": "University of Alberta"
        },
        {
          "city": "Toronto",
          "country": "Canada",
          "facility": "The Hospital for Sick Children - SickKids"
        },
        {
          "city": "Paris",
          "country": "France",
          "facility": "Centre de maladies rares CHNO des Quinze Vingt"
        },
        {
          "city": "Giessen",
          "country": "Germany",
          "facility": "Justus-Liebig Universität - Department of Ophthalmology"
        },
        {
          "city": "München",
          "country": "Germany",
          "facility": "Klinikum der Ludwig-Maximilian Universität München"
        },
        {
          "city": "Tübingen",
          "country": "Germany",
          "facility": "University of Tuebingen - Inst. for Ophthalmic Research"
        },
        {
          "city": "Nijmegen",
          "country": "Netherlands",
          "facility": "Radboud Universitair Medisch Centrum"
        },
        {
          "city": "Barcelona",
          "country": "Spain",
          "facility": "Hospital Sant Joan de Déu (SJD Barcelona Children's Hospital)"
        },
        {
          "city": "London",
          "country": "United Kingdom",
          "facility": "Moorfields Eye Hospital NHS Foundation Trust"
        }
      ],
      "outcomes": {
        "other": [],
        "primary": [
          "Change from baseline in Best-Corrected Visual Acuity (BCVA)"
        ],
        "secondary": [
          "Eye-specific Patient Global Impression of Change (PGI-C) between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs)",
          "Change from baseline in Low Luminance Visual Acuity (LLVA) based on FrACT between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs)",
          "Change from baseline in retinal sensitivity as measured by dark-adapted Full-Field Stimulus Test (FST) between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs)",
          "Change from baseline in Contrast Sensitivity (CS) between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) based on qCSF",
          "Change from baseline in retinal sensitivity as measured by light-adapted Full-Field Stimulus Test (FST) between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs)",
          "Response in BCVA (FrACT), defined as an improvement of at least 0.2 logMAR from baseline at 12 months and compared between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs), evaluated by the percentage of eyes that achieve this improvement.",
          "Response in BCVA (FrACT), defined as an improvement of at least 0.3 logMAR from baseline at 12 months and compared between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs), evaluated by the percentage of eyes that achieve this improvement.",
          "Response in Low Luminance Visual Acuity (LLVA) defined as clinically relevant improvement between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) at 12 months, evaluated by the percentage of eyes that achieve this improvement.",
          "Response in Full-Field Stimulus Test (FST) defined as clinically relevant improvement between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) at 12 months, evaluated by the percentage of eyes that achieve this improvement.",
          "Response in Patient Global Impression of Change (PGI-C) defined as clinically relevant improvement between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) at 12 months, evaluated by the percentage of eyes that achieve this improvement.",
          "Response in Contrast Sensitivity (CS) defined as clinically relevant improvement between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) at 12 months, evaluated by the percentage of eyes that achieve this improvement.",
          "Response defined as a clinically relevant improvement according to a multimodal assessment in TEs compared to the corresponding response in PCEs at 12 months",
          "Change from baseline in the Michigan Retinal Degeneration Questionnaire (MRDQ) score for subjects ≥ 13 years of age",
          "Change from baseline in the Michigan Vision-Related Anxiety Questionnaire (MVAQ) score for subjects ≥ 13 years of age",
          "Change from baseline in the Pediatric Eye Questionnaire (PedEyeQ) score for subjects < 13 years of age",
          "Change from baseline in eye-specific Patient Global Impression of Severity (PGI-S) between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs)",
          "Change from baseline in Best-Corrected Visual Acuity (BCVA) based on the ETDRS and/or the BRVT between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs)",
          "Change from baseline in Low Luminance Visual Acuity (LLVA) based on the ETDRS between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs)"
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        "criteria": "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 / Minors: 6 to \\<18 years.\n3. BCVA (FrACT) equal to or worse than logMAR +0.4 (approximate Snellen equivalent 20/50) 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/cataracts 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.",
        "minimumAge": "6 Years",
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        "count": 32
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      "startDate": "2025-06-04",
      "completionDate": "2028-10-01",
      "lastUpdated": "2026-10-21T00:00:00.000Z",
      "firstPosted": "2025-03-24T00:00:00.000Z",
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