{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Leber+Congenital+Amaurosis+%28LCA%29",
    "query": {
      "condition": "Leber Congenital Amaurosis (LCA)"
    },
    "page_size": 10
  },
  "pagination": {
    "page": 1,
    "page_size": 10,
    "total_count": 20,
    "total_pages": 2,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Leber+Congenital+Amaurosis+%28LCA%29&page=2&page_size=10",
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-07-23T16:30:05.650Z",
  "attribution": "Data derived from public ClinicalTrials.gov records. The official record at https://clinicaltrials.gov/ remains the source of truth for current availability, contacts, and full study details.",
  "notes": [
    "This endpoint exposes only public summary fields — no participant contact emails, phone numbers, or scraped investigator contact details.",
    "For contact information and full protocol detail, follow each trial's `official_url` to the ClinicalTrials.gov record.",
    "This API is a navigational aid. It does not provide medical advice, eligibility determinations, or compensation guarantees."
  ],
  "trials": [
    {
      "nct_id": "NCT03913143",
      "title": "A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2",
        "PHASE3"
      ],
      "conditions": [
        "Leber Congenital Amaurosis 10",
        "Blindness",
        "Leber Congenital Amaurosis",
        "Vision Disorders",
        "Sensation Disorders",
        "Neurologic Manifestations",
        "Eye Diseases",
        "Eye Diseases, Hereditary",
        "Eye Disorders Congenital",
        "Retinal Disease"
      ],
      "interventions": [
        {
          "name": "sepofarsen",
          "type": "DRUG"
        },
        {
          "name": "Sham",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "DRUG",
        "OTHER"
      ],
      "sponsor": "ProQR Therapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "8 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "8 Years and older"
      },
      "enrollment_count": 36,
      "start_date": "2019-04-04",
      "completion_date": "2023-03",
      "has_results": false,
      "last_update_posted_date": "2022-03-17",
      "last_synced_at": "2026-07-23T16:30:05.650Z",
      "location_count": 1,
      "location_summary": "Iowa City, Iowa",
      "locations": [
        {
          "city": "Iowa City",
          "state": "Iowa"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03913143"
    },
    {
      "nct_id": "NCT03140969",
      "title": "Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Leber's Congenital Amaurosis"
      ],
      "interventions": [
        {
          "name": "QR-110",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Laboratoires Thea",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "6 Years and older"
      },
      "enrollment_count": 11,
      "start_date": "2017-10-16",
      "completion_date": "2019-10-02",
      "has_results": true,
      "last_update_posted_date": "2024-10-15",
      "last_synced_at": "2026-07-23T16:30:05.650Z",
      "location_count": 2,
      "location_summary": "Iowa City, Iowa • Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "Iowa City",
          "state": "Iowa"
        },
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03140969"
    },
    {
      "nct_id": "NCT02575430",
      "title": "Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Leber Congenital Amaurosis (LCA)",
        "Retinitis Pigmentosa (RP)"
      ],
      "interventions": [
        {
          "name": "No treatment: retrospective chart review",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "QLT Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "8 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "8 Years and older"
      },
      "enrollment_count": 59,
      "start_date": "2015-12",
      "completion_date": "2016-03",
      "has_results": false,
      "last_update_posted_date": "2016-04-29",
      "last_synced_at": "2026-07-23T16:30:05.650Z",
      "location_count": 2,
      "location_summary": "Baltimore, Maryland • Portland, Oregon",
      "locations": [
        {
          "city": "Baltimore",
          "state": "Maryland"
        },
        {
          "city": "Portland",
          "state": "Oregon"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02575430"
    },
    {
      "nct_id": "NCT02781480",
      "title": "Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Leber Congenital Amaurosis"
      ],
      "interventions": [
        {
          "name": "AAV RPE65",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "MeiraGTx UK II Ltd",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "3 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "3 Years and older"
      },
      "enrollment_count": 15,
      "start_date": "2016-04",
      "completion_date": "2018-12",
      "has_results": true,
      "last_update_posted_date": "2021-07-12",
      "last_synced_at": "2026-07-23T16:30:05.650Z",
      "location_count": 1,
      "location_summary": "Ann Arbor, Michigan",
      "locations": [
        {
          "city": "Ann Arbor",
          "state": "Michigan"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02781480"
    },
    {
      "nct_id": "NCT01208389",
      "title": "Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Leber Congenital Amaurosis"
      ],
      "interventions": [
        {
          "name": "voretigene neparvovec-rzyl",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Spark Therapeutics, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "8 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "8 Years and older"
      },
      "enrollment_count": 12,
      "start_date": "2010-11",
      "completion_date": "2027-10",
      "has_results": false,
      "last_update_posted_date": "2025-04-29",
      "last_synced_at": "2026-07-23T16:30:05.650Z",
      "location_count": 1,
      "location_summary": "Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01208389"
    },
    {
      "nct_id": "NCT06891443",
      "title": "Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "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"
        },
        {
          "name": "Placebo IVT",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "DRUG",
        "OTHER"
      ],
      "sponsor": "Laboratoires Thea",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "6 Years and older"
      },
      "enrollment_count": 32,
      "start_date": "2025-06-04",
      "completion_date": "2028-10",
      "has_results": false,
      "last_update_posted_date": "2026-06-25",
      "last_synced_at": "2026-07-23T16:30:05.650Z",
      "location_count": 5,
      "location_summary": "San Francisco, California • Miami, Florida • Iowa City, Iowa + 2 more",
      "locations": [
        {
          "city": "San Francisco",
          "state": "California"
        },
        {
          "city": "Miami",
          "state": "Florida"
        },
        {
          "city": "Iowa City",
          "state": "Iowa"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        },
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06891443"
    },
    {
      "nct_id": "NCT03396042",
      "title": "Natural History Study of CEP290-Related Retinal Degeneration",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Blindness",
        "Leber Congenital Amaurosis 10",
        "Vision Disorders",
        "Eye Diseases",
        "Eye Diseases, Hereditary",
        "Eye Disorders Congenital",
        "Retinal Disease",
        "Retinal Degeneration"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Editas Medicine, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "3 Years",
        "maximum_age": "99 Years",
        "sex": "ALL",
        "summary": "3 Years to 99 Years"
      },
      "enrollment_count": 26,
      "start_date": "2017-12-17",
      "completion_date": "2022-05-06",
      "has_results": false,
      "last_update_posted_date": "2022-05-19",
      "last_synced_at": "2026-07-23T16:30:05.650Z",
      "location_count": 4,
      "location_summary": "Miami, Florida • Boston, Massachusetts • Ann Arbor, Michigan + 1 more",
      "locations": [
        {
          "city": "Miami",
          "state": "Florida"
        },
        {
          "city": "Boston",
          "state": "Massachusetts"
        },
        {
          "city": "Ann Arbor",
          "state": "Michigan"
        },
        {
          "city": "Portland",
          "state": "Oregon"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03396042"
    },
    {
      "nct_id": "NCT02946879",
      "title": "Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Leber Congenital Amaurosis (LCA)",
        "Eye Diseases",
        "Eye Diseases, Hereditary",
        "Retinal Diseases"
      ],
      "interventions": [
        {
          "name": "AAV OPTIRPE65",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "MeiraGTx UK II Ltd",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "3 Years",
        "maximum_age": "100 Years",
        "sex": "ALL",
        "summary": "3 Years to 100 Years"
      },
      "enrollment_count": 15,
      "start_date": "2016-11",
      "completion_date": "2023-07",
      "has_results": false,
      "last_update_posted_date": "2024-04-11",
      "last_synced_at": "2026-07-23T16:30:05.650Z",
      "location_count": 1,
      "location_summary": "Ann Arbor, Michigan",
      "locations": [
        {
          "city": "Ann Arbor",
          "state": "Michigan"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02946879"
    },
    {
      "nct_id": "NCT05906953",
      "title": "Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Leber Congenital Amaurosis",
        "Inherited Retinal Diseases Caused by RPE65 Mutations"
      ],
      "interventions": [
        {
          "name": "HG004",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "HuidaGene Therapeutics Co., Ltd.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Years",
        "maximum_age": "50 Years",
        "sex": "ALL",
        "summary": "6 Years to 50 Years"
      },
      "enrollment_count": 20,
      "start_date": "2023-10-31",
      "completion_date": "2025-12",
      "has_results": false,
      "last_update_posted_date": "2024-09-19",
      "last_synced_at": "2026-07-23T16:30:05.650Z",
      "location_count": 2,
      "location_summary": "Sacramento, California • Houston, Texas",
      "locations": [
        {
          "city": "Sacramento",
          "state": "California"
        },
        {
          "city": "Houston",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05906953"
    },
    {
      "nct_id": "NCT00481546",
      "title": "Phase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Amaurosis of Leber",
        "Retinal Diseases"
      ],
      "interventions": [
        {
          "name": "rAAV2-CBSB-hRPE65",
          "type": "GENETIC"
        }
      ],
      "intervention_types": [
        "GENETIC"
      ],
      "sponsor": "University of Pennsylvania",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "8 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "8 Years and older"
      },
      "enrollment_count": 15,
      "start_date": "2007-07",
      "completion_date": "2016-12",
      "has_results": false,
      "last_update_posted_date": "2026-07-17",
      "last_synced_at": "2026-07-23T16:30:05.650Z",
      "location_count": 2,
      "location_summary": "Gainesville, Florida • Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "Gainesville",
          "state": "Florida"
        },
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00481546"
    }
  ]
}