{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?intervention=EPI-743",
    "query": {
      "intervention": "EPI-743"
    },
    "page_size": 10
  },
  "pagination": {
    "page": 1,
    "page_size": 10,
    "total_count": 17,
    "total_pages": 2,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?intervention=EPI-743&page=2&page_size=10",
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-09-03T13:34:25.829Z",
  "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": "NCT01642056",
      "title": "EPI-743 for Metabolism or Mitochondrial Disorders",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Mitochondrial Disease",
        "Neurology",
        "Myopathy"
      ],
      "interventions": [
        {
          "name": "EPI-743",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "National Human Genome Research Institute (NHGRI)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": "11 Years",
        "sex": "ALL",
        "summary": "2 Years to 11 Years"
      },
      "enrollment_count": 20,
      "start_date": "2012-09-01",
      "completion_date": "2019-09-24",
      "has_results": true,
      "last_update_posted_date": "2021-04-14",
      "last_synced_at": "2026-09-03T13:34:25.829Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01642056"
    },
    {
      "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": "2026-09-03T13:34:25.829Z",
      "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": "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": "2026-09-03T13:34:25.829Z",
      "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"
    },
    {
      "nct_id": "NCT01923584",
      "title": "A Phase 2A Trial of EPI-743 for Patients With Parkinson's Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Parkinson's Disease"
      ],
      "interventions": [
        {
          "name": "EPI-743 400mg",
          "type": "DRUG"
        },
        {
          "name": "EPI-743 200mg",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "University of South Florida",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "40 Years",
        "maximum_age": "75 Years",
        "sex": "ALL",
        "summary": "40 Years to 75 Years"
      },
      "enrollment_count": 15,
      "start_date": "2013-09",
      "completion_date": "2016-06",
      "has_results": false,
      "last_update_posted_date": "2016-09-19",
      "last_synced_at": "2026-09-03T13:34:25.829Z",
      "location_count": 1,
      "location_summary": "Tampa, Florida",
      "locations": [
        {
          "city": "Tampa",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01923584"
    },
    {
      "nct_id": "NCT01728064",
      "title": "Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Friedreich's Ataxia"
      ],
      "interventions": [
        {
          "name": "Placebo",
          "type": "DRUG"
        },
        {
          "name": "EPI-743 400 mg",
          "type": "DRUG"
        },
        {
          "name": "EPI-743 200 mg",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "PTC Therapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "45 Years",
        "sex": "ALL",
        "summary": "18 Years to 45 Years"
      },
      "enrollment_count": 60,
      "start_date": "2012-12-31",
      "completion_date": "2016-02-29",
      "has_results": false,
      "last_update_posted_date": "2020-08-31",
      "last_synced_at": "2026-09-03T13:34:25.829Z",
      "location_count": 3,
      "location_summary": "Los Angeles, California • Tampa, Florida • Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Tampa",
          "state": "Florida"
        },
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01728064"
    },
    {
      "nct_id": "NCT02104336",
      "title": "Phase 2 Study of EPI-743 in Children With Pearson Syndrome",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Pearson Syndrome"
      ],
      "interventions": [
        {
          "name": "EPI-743",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Edison Pharmaceuticals Inc",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "18 Years",
        "sex": "ALL",
        "summary": "Up to 18 Years"
      },
      "enrollment_count": 2,
      "start_date": "2014-08-31",
      "completion_date": "2016-02-29",
      "has_results": false,
      "last_update_posted_date": "2020-11-18",
      "last_synced_at": "2026-09-03T13:34:25.829Z",
      "location_count": 4,
      "location_summary": "Orange, California • Cleveland, Ohio • Pittsburgh, Pennsylvania + 1 more",
      "locations": [
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "Cleveland",
          "state": "Ohio"
        },
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        },
        {
          "city": "Houston",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02104336"
    },
    {
      "nct_id": "NCT01721733",
      "title": "Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Leigh Syndrome"
      ],
      "interventions": [
        {
          "name": "Placebo",
          "type": "DRUG"
        },
        {
          "name": "EPI-743 15 mg/kg",
          "type": "DRUG"
        },
        {
          "name": "EPI-743 5 mg/kg",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "PTC Therapeutics",
      "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": 35,
      "start_date": "2012-10-31",
      "completion_date": "2015-05-31",
      "has_results": false,
      "last_update_posted_date": "2020-08-31",
      "last_synced_at": "2026-09-03T13:34:25.829Z",
      "location_count": 4,
      "location_summary": "Palo Alto, California • Akron, Ohio • Houston, Texas + 1 more",
      "locations": [
        {
          "city": "Palo Alto",
          "state": "California"
        },
        {
          "city": "Akron",
          "state": "Ohio"
        },
        {
          "city": "Houston",
          "state": "Texas"
        },
        {
          "city": "Seattle",
          "state": "Washington"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01721733"
    },
    {
      "nct_id": "NCT01962363",
      "title": "EPI-743 in Friedreich's Ataxia Point Mutations",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Friedreich's Ataxia"
      ],
      "interventions": [
        {
          "name": "EPI-743",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "University of South Florida",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "65 Years",
        "sex": "ALL",
        "summary": "18 Years to 65 Years"
      },
      "enrollment_count": 4,
      "start_date": "2013-10",
      "completion_date": "2016-06",
      "has_results": false,
      "last_update_posted_date": "2016-06-29",
      "last_synced_at": "2026-09-03T13:34:25.829Z",
      "location_count": 1,
      "location_summary": "Tampa, Florida",
      "locations": [
        {
          "city": "Tampa",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01962363"
    },
    {
      "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": "2026-09-03T13:34:25.829Z",
      "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": "NCT02300753",
      "title": "Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]",
      "overall_status": "NO_LONGER_AVAILABLE",
      "study_type": "EXPANDED_ACCESS",
      "phases": [],
      "conditions": [
        "Leber's Hereditary Optic Neuropathy"
      ],
      "interventions": [
        {
          "name": "EPI-743",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Edison Pharmaceuticals Inc",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": null,
      "eligibility": {
        "minimum_age": "8 Years",
        "maximum_age": "65 Years",
        "sex": "ALL",
        "summary": "8 Years to 65 Years"
      },
      "enrollment_count": null,
      "start_date": null,
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2014-11-25",
      "last_synced_at": "2026-09-03T13:34:25.829Z",
      "location_count": 1,
      "location_summary": "Los Angeles, California",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02300753"
    }
  ]
}