{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Leigh+Syndrome",
    "query": {
      "condition": "Leigh Syndrome"
    },
    "page_size": 10
  },
  "pagination": {
    "page": 1,
    "page_size": 10,
    "total_count": 14,
    "total_pages": 2,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Leigh+Syndrome&page=2&page_size=10",
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-05-22T04:21:05.560Z",
  "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": "NCT02023866",
      "title": "Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Inherited Mitochondrial Disease, Including Leigh Syndrome"
      ],
      "interventions": [
        {
          "name": "Cysteamine Bitartrate",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Amgen",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Years",
        "maximum_age": "17 Years",
        "sex": "ALL",
        "summary": "6 Years to 17 Years"
      },
      "enrollment_count": 36,
      "start_date": "2014-05",
      "completion_date": "2016-10",
      "has_results": true,
      "last_update_posted_date": "2024-12-27",
      "last_synced_at": "2026-05-22T04:21:05.560Z",
      "location_count": 5,
      "location_summary": "San Diego, California • Stanford, California • Akron, Ohio + 2 more",
      "locations": [
        {
          "city": "San Diego",
          "state": "California"
        },
        {
          "city": "Stanford",
          "state": "California"
        },
        {
          "city": "Akron",
          "state": "Ohio"
        },
        {
          "city": "Houston",
          "state": "Texas"
        },
        {
          "city": "Salt Lake City",
          "state": "Utah"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02023866"
    },
    {
      "nct_id": "NCT01803906",
      "title": "Tissue Sample Study for Mitochondrial Disorders",
      "overall_status": "ENROLLING_BY_INVITATION",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Mitochondrial Disorders",
        "Mitochondrial Disease",
        "Melas",
        "Kearns Sayer",
        "NARP",
        "MNGIE",
        "LHON",
        "Mitochondrial Depletion Syndrome",
        "Leigh's Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Columbia University",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 6900,
      "start_date": "2012-02",
      "completion_date": "2026-12",
      "has_results": false,
      "last_update_posted_date": "2026-01-07",
      "last_synced_at": "2026-05-22T04:21:05.560Z",
      "location_count": 1,
      "location_summary": "New York, New York",
      "locations": [
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01803906"
    },
    {
      "nct_id": "NCT01694940",
      "title": "North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Mitochondrial Disorders",
        "Mitochondrial Genetic Disorders",
        "Mitochondrial Diseases",
        "Disorder of Mitochondrial Respiratory Chain Complexes",
        "Deletion and Duplication of Mitochondrial DNA"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Columbia University",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 1000,
      "start_date": "2011-01-31",
      "completion_date": "2026-12-31",
      "has_results": false,
      "last_update_posted_date": "2026-02-04",
      "last_synced_at": "2026-05-22T04:21:05.560Z",
      "location_count": 16,
      "location_summary": "San Diego, California • Stanford, California • Aurora, Colorado + 12 more",
      "locations": [
        {
          "city": "San Diego",
          "state": "California"
        },
        {
          "city": "Stanford",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Washington D.C.",
          "state": "District of Columbia"
        },
        {
          "city": "Gainsville",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01694940"
    },
    {
      "nct_id": "NCT03137355",
      "title": "The International Registry for Leigh Syndrome",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Leigh Syndrome",
        "Leigh Disease",
        "Leigh's Necrotizing Encephalopathy",
        "Subacute Necrotizing Encephalomyopathy",
        "Subacute Necrotizing Encephalomyelopathy"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "The University of Texas Health Science Center, Houston",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "0 Days",
        "maximum_age": "100 Years",
        "sex": "ALL",
        "summary": "0 Days to 100 Years"
      },
      "enrollment_count": 200,
      "start_date": "2015-06-17",
      "completion_date": "2030-06-17",
      "has_results": false,
      "last_update_posted_date": "2023-12-01",
      "last_synced_at": "2026-05-22T04:21:05.560Z",
      "location_count": 1,
      "location_summary": "Houston, Texas",
      "locations": [
        {
          "city": "Houston",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03137355"
    },
    {
      "nct_id": "NCT01370447",
      "title": "EPI-743 for Mitochondrial Respiratory Chain Diseases",
      "overall_status": "NO_LONGER_AVAILABLE",
      "study_type": "EXPANDED_ACCESS",
      "phases": [],
      "conditions": [
        "Mitochondrial Diseases"
      ],
      "interventions": [
        {
          "name": "EPI-743",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "PTC Therapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Year",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "1 Year and older"
      },
      "enrollment_count": null,
      "start_date": null,
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2024-01-23",
      "last_synced_at": "2026-05-22T04:21:05.560Z",
      "location_count": 16,
      "location_summary": "Los Angeles, California • Orange, California • Palo Alto, California + 12 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "Palo Alto",
          "state": "California"
        },
        {
          "city": "San Diego",
          "state": "California"
        },
        {
          "city": "Stanford",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01370447"
    },
    {
      "nct_id": "NCT06843811",
      "title": "Sirolimus for Leigh Syndrome",
      "overall_status": "ENROLLING_BY_INVITATION",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Leigh Syndrome"
      ],
      "interventions": [
        {
          "name": "Sirolimus",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Matthew Demczko",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Months",
        "maximum_age": "55 Years",
        "sex": "ALL",
        "summary": "6 Months to 55 Years"
      },
      "enrollment_count": 15,
      "start_date": "2025-02-27",
      "completion_date": "2027-12",
      "has_results": false,
      "last_update_posted_date": "2026-03-31",
      "last_synced_at": "2026-05-22T04:21:05.560Z",
      "location_count": 1,
      "location_summary": "Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06843811"
    },
    {
      "nct_id": "NCT04378075",
      "title": "A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2",
        "PHASE3"
      ],
      "conditions": [
        "Mitochondrial Diseases",
        "Drug Resistant Epilepsy",
        "Leigh Disease",
        "Leigh Syndrome",
        "Mitochondrial Encephalopathy (MELAS)",
        "Pontocerebellar Hypoplasia Type 6 (PCH6)",
        "Alpers Disease",
        "Alpers Syndrome"
      ],
      "interventions": [
        {
          "name": "Vatiquinone",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "DRUG",
        "OTHER"
      ],
      "sponsor": "PTC Therapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "20 Years",
        "sex": "ALL",
        "summary": "Up to 20 Years"
      },
      "enrollment_count": 68,
      "start_date": "2020-09-28",
      "completion_date": "2023-12-27",
      "has_results": true,
      "last_update_posted_date": "2026-03-31",
      "last_synced_at": "2026-05-22T04:21:05.560Z",
      "location_count": 12,
      "location_summary": "San Diego, California • Stanford, California • New Haven, Connecticut + 7 more",
      "locations": [
        {
          "city": "San Diego",
          "state": "California"
        },
        {
          "city": "Stanford",
          "state": "California"
        },
        {
          "city": "New Haven",
          "state": "Connecticut"
        },
        {
          "city": "Washington D.C.",
          "state": "District of Columbia"
        },
        {
          "city": "Baltimore",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04378075"
    },
    {
      "nct_id": "NCT05218655",
      "title": "A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Inherited Mitochondrial Disease"
      ],
      "interventions": [
        {
          "name": "Vatiquinone",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "PTC Therapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 101,
      "start_date": "2022-06-22",
      "completion_date": "2025-04-15",
      "has_results": true,
      "last_update_posted_date": "2025-12-22",
      "last_synced_at": "2026-05-22T04:21:05.560Z",
      "location_count": 14,
      "location_summary": "La Jolla, California • Stanford, California • New Haven, Connecticut + 10 more",
      "locations": [
        {
          "city": "La Jolla",
          "state": "California"
        },
        {
          "city": "Stanford",
          "state": "California"
        },
        {
          "city": "New Haven",
          "state": "Connecticut"
        },
        {
          "city": "Washington D.C.",
          "state": "District of Columbia"
        },
        {
          "city": "Gulf Breeze",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05218655"
    },
    {
      "nct_id": "NCT02473445",
      "title": "A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Mitochondrial Diseases"
      ],
      "interventions": [
        {
          "name": "Cysteamine Bitartrate",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Amgen",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Years",
        "maximum_age": "17 Years",
        "sex": "ALL",
        "summary": "6 Years to 17 Years"
      },
      "enrollment_count": 22,
      "start_date": "2015-05-19",
      "completion_date": "2017-03-06",
      "has_results": true,
      "last_update_posted_date": "2024-12-27",
      "last_synced_at": "2026-05-22T04:21:05.560Z",
      "location_count": 5,
      "location_summary": "San Diego, California • Stanford, California • Akron, Ohio + 2 more",
      "locations": [
        {
          "city": "San Diego",
          "state": "California"
        },
        {
          "city": "Stanford",
          "state": "California"
        },
        {
          "city": "Akron",
          "state": "Ohio"
        },
        {
          "city": "Houston",
          "state": "Texas"
        },
        {
          "city": "Salt Lake City",
          "state": "Utah"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02473445"
    },
    {
      "nct_id": "NCT02352896",
      "title": "Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Leigh Syndrome"
      ],
      "interventions": [
        {
          "name": "EPI-743",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "PTC Therapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Year",
        "maximum_age": "18 Years",
        "sex": "ALL",
        "summary": "1 Year to 18 Years"
      },
      "enrollment_count": 30,
      "start_date": "2014-01-31",
      "completion_date": "2023-10-30",
      "has_results": false,
      "last_update_posted_date": "2024-10-23",
      "last_synced_at": "2026-05-22T04:21:05.560Z",
      "location_count": 4,
      "location_summary": "Stanford University, California • Akron, Ohio • Houston, Texas + 1 more",
      "locations": [
        {
          "city": "Stanford University",
          "state": "California"
        },
        {
          "city": "Akron",
          "state": "Ohio"
        },
        {
          "city": "Houston",
          "state": "Texas"
        },
        {
          "city": "Seattle",
          "state": "Washington"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02352896"
    }
  ]
}