{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=17",
    "query": {
      "condition": "Inborn Errors of Metabolism Disorders",
      "page": 17
    },
    "page_size": 10
  },
  "pagination": {
    "page": 17,
    "page_size": 10,
    "total_count": 1510,
    "total_pages": 151,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=18&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=16&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-23T14:50:07.951Z",
  "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": "NCT03766386",
      "title": "The Natural History of Danon Disease",
      "overall_status": "UNKNOWN",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Danon Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "University of California, San Diego",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "0 Years",
        "maximum_age": "100 Years",
        "sex": "ALL",
        "summary": "0 Years to 100 Years"
      },
      "enrollment_count": 200,
      "start_date": "2018-11-30",
      "completion_date": "2024-11",
      "has_results": false,
      "last_update_posted_date": "2022-05-12",
      "last_synced_at": "2026-10-23T14:50:07.951Z",
      "location_count": 1,
      "location_summary": "San Diego, California",
      "locations": [
        {
          "city": "San Diego",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03766386"
    },
    {
      "nct_id": "NCT01184651",
      "title": "Tweens to Teens Project at Penn State",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Congenital Adrenal Hyperplasia"
      ],
      "interventions": [
        {
          "name": "Questionnaires/Interviews",
          "type": "BEHAVIORAL"
        }
      ],
      "intervention_types": [
        "BEHAVIORAL"
      ],
      "sponsor": "Penn State University",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "10 Years",
        "maximum_age": "13 Years",
        "sex": "FEMALE",
        "summary": "10 Years to 13 Years · Female only"
      },
      "enrollment_count": 188,
      "start_date": "2009-08",
      "completion_date": "2013-07",
      "has_results": false,
      "last_update_posted_date": "2018-01-08",
      "last_synced_at": "2026-10-23T14:50:07.951Z",
      "location_count": 1,
      "location_summary": "University Park, Pennsylvania",
      "locations": [
        {
          "city": "University Park",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01184651"
    },
    {
      "nct_id": "NCT03876522",
      "title": "Natural History and Functional Status Study of Patients With Lafora Disease",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Lafora Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Ionis Pharmaceuticals, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "5 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "5 Years and older"
      },
      "enrollment_count": 33,
      "start_date": "2019-01-09",
      "completion_date": "2022-04-01",
      "has_results": false,
      "last_update_posted_date": "2022-09-28",
      "last_synced_at": "2026-10-23T14:50:07.951Z",
      "location_count": 2,
      "location_summary": "Los Angeles, California • Dallas, Texas",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Dallas",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03876522"
    },
    {
      "nct_id": "NCT00005111",
      "title": "PET Scans in Normal Volunteers and Patients With Fabry Disease",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Fabry Disease",
        "Healthy"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "National Institute of Neurological Disorders and Stroke (NINDS)",
      "sponsor_class": "NIH",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "MALE",
        "summary": "Male only"
      },
      "enrollment_count": 24,
      "start_date": "2000-04",
      "completion_date": "2001-03",
      "has_results": false,
      "last_update_posted_date": "2008-03-04",
      "last_synced_at": "2026-10-23T14:50:07.951Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00005111"
    },
    {
      "nct_id": "NCT02991144",
      "title": "Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Ornithine Transcarbamylase (OTC) Deficiency"
      ],
      "interventions": [
        {
          "name": "scAAV8OTC",
          "type": "GENETIC"
        },
        {
          "name": "Reactive Corticosteroid Taper Regimen",
          "type": "DRUG"
        },
        {
          "name": "Prophylactic Corticosteroid Taper Regimen",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "GENETIC",
        "DRUG"
      ],
      "sponsor": "Ultragenyx Pharmaceutical Inc",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 16,
      "start_date": "2017-07-31",
      "completion_date": "2021-12-16",
      "has_results": true,
      "last_update_posted_date": "2023-01-26",
      "last_synced_at": "2026-10-23T14:50:07.951Z",
      "location_count": 4,
      "location_summary": "Aurora, Colorado • Boston, Massachusetts • New York, New York + 1 more",
      "locations": [
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Boston",
          "state": "Massachusetts"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Cleveland",
          "state": "Ohio"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02991144"
    },
    {
      "nct_id": "NCT00355264",
      "title": "Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Tetrahydrobiopterin Deficiencies",
        "Hyperphenylalaninemia, Non-Phenylketonuric"
      ],
      "interventions": [
        {
          "name": "Phenoptin",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "BioMarin Pharmaceutical",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 12,
      "start_date": "2006-08",
      "completion_date": "2009-06",
      "has_results": true,
      "last_update_posted_date": "2020-09-25",
      "last_synced_at": "2026-10-23T14:50:07.951Z",
      "location_count": 9,
      "location_summary": "Los Angeles, California • Chicago, Illinois • Minneapolis, Minnesota + 6 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Chapel Hill",
          "state": "North Carolina"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00355264"
    },
    {
      "nct_id": "NCT02939547",
      "title": "Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Niemann-Pick Disease, Type C1"
      ],
      "interventions": [
        {
          "name": "Hydroxypropyl-beta-cyclodextrin",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Cyclo Therapeutics, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 13,
      "start_date": "2017-10-11",
      "completion_date": "2020-02-10",
      "has_results": false,
      "last_update_posted_date": "2021-02-21",
      "last_synced_at": "2026-10-23T14:50:07.951Z",
      "location_count": 2,
      "location_summary": "Oakland, California • Morristown, New Jersey",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Morristown",
          "state": "New Jersey"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02939547"
    },
    {
      "nct_id": "NCT00001455",
      "title": "Iron Overload in African Americans",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Hemochromatosis",
        "Iron Overload"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 500,
      "start_date": "1995-06",
      "completion_date": "2000-06",
      "has_results": false,
      "last_update_posted_date": "2008-03-04",
      "last_synced_at": "2026-10-23T14:50:07.951Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00001455"
    },
    {
      "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": "2026-10-23T14:50:07.951Z",
      "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": "NCT04195763",
      "title": "Patient Reported Outcomes in Adults With Pediatric-onset Hypophosphatasia Treated With Strensiq® (Asfotase Alfa)",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Hypophosphatasia"
      ],
      "interventions": [
        {
          "name": "asfotase alfa",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Alexion Pharmaceuticals, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "99 Years",
        "sex": "ALL",
        "summary": "18 Years to 99 Years"
      },
      "enrollment_count": 50,
      "start_date": "2019-11-06",
      "completion_date": "2024-04-12",
      "has_results": false,
      "last_update_posted_date": "2024-07-10",
      "last_synced_at": "2026-10-23T14:50:07.951Z",
      "location_count": 1,
      "location_summary": "Palm Harbor, Florida",
      "locations": [
        {
          "city": "Palm Harbor",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04195763"
    }
  ]
}