{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=9",
    "query": {
      "condition": "Inborn Errors of Metabolism",
      "page": 9
    },
    "page_size": 10
  },
  "pagination": {
    "page": 9,
    "page_size": 10,
    "total_count": 1605,
    "total_pages": 161,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=10&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=8&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-14T04:45:34.129Z",
  "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": "NCT00280995",
      "title": "Dose-escalating Safety Study of ISIS 301012 in Homozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Hypercholesterolemia, Familial"
      ],
      "interventions": [
        {
          "name": "ISIS 301012",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Kastle Therapeutics, LLC",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 12,
      "start_date": "2006-01",
      "completion_date": "2007-08",
      "has_results": false,
      "last_update_posted_date": "2016-08-03",
      "last_synced_at": "2026-10-14T04:45:34.129Z",
      "location_count": 5,
      "location_summary": "Chicago, Illinois • New York, New York • Winston-Salem, North Carolina + 2 more",
      "locations": [
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Winston-Salem",
          "state": "North Carolina"
        },
        {
          "city": "Cincinnati",
          "state": "Ohio"
        },
        {
          "city": "Houston",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00280995"
    },
    {
      "nct_id": "NCT05196165",
      "title": "Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storage Disease Type III",
      "overall_status": "TERMINATED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Glycogen Storage Disease Type III"
      ],
      "interventions": [
        {
          "name": "No Intervention",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "Ultragenyx Pharmaceutical Inc",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Year",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "1 Year and older"
      },
      "enrollment_count": 14,
      "start_date": "2022-05-20",
      "completion_date": "2023-03-02",
      "has_results": false,
      "last_update_posted_date": "2023-09-28",
      "last_synced_at": "2026-10-14T04:45:34.129Z",
      "location_count": 3,
      "location_summary": "Philadelphia, Pennsylvania • Houston, Texas",
      "locations": [
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        },
        {
          "city": "Houston",
          "state": "Texas"
        },
        {
          "city": "Houston",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05196165"
    },
    {
      "nct_id": "NCT01624311",
      "title": "Pilot Study For Hypothermia Treatment In Hyperammonemic Encephalopathy In Neonates And Very Young Infants",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Urea Cycle Disorders",
        "Organic Acidemias"
      ],
      "interventions": [
        {
          "name": "Therapeutic Hypothermia",
          "type": "OTHER"
        },
        {
          "name": "Standard of Care Therapy",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "Uta Lichter-Konecki",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Day",
        "maximum_age": "30 Days",
        "sex": "ALL",
        "summary": "1 Day to 30 Days"
      },
      "enrollment_count": 5,
      "start_date": "2007-08",
      "completion_date": "2015-05",
      "has_results": false,
      "last_update_posted_date": "2015-06-01",
      "last_synced_at": "2026-10-14T04:45:34.129Z",
      "location_count": 3,
      "location_summary": "Washington D.C., District of Columbia • New York, New York • Milwaukee, Wisconsin",
      "locations": [
        {
          "city": "Washington D.C.",
          "state": "District of Columbia"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Milwaukee",
          "state": "Wisconsin"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01624311"
    },
    {
      "nct_id": "NCT07667387",
      "title": "Study of LNP.UCD.ABE in Patients With Urea Cycle Disorders",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Urea Cycle Disorders",
        "Carbamoyl-Phosphate Synthase I Deficiency"
      ],
      "interventions": [
        {
          "name": "LNP.UCD.ABE",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Rebecca Ahrens-Nicklas",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "24 Hours",
        "maximum_age": "5 Years",
        "sex": "ALL",
        "summary": "24 Hours to 5 Years"
      },
      "enrollment_count": 7,
      "start_date": "2026-08-07",
      "completion_date": "2028-09",
      "has_results": false,
      "last_update_posted_date": "2026-08-26",
      "last_synced_at": "2026-10-14T04:45:34.129Z",
      "location_count": 1,
      "location_summary": "Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07667387"
    },
    {
      "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": "2026-10-14T04:45:34.129Z",
      "location_count": 1,
      "location_summary": "Houston, Texas",
      "locations": [
        {
          "city": "Houston",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03137355"
    },
    {
      "nct_id": "NCT00587535",
      "title": "Evaluation of a New MR Pulse Sequence to Quantify Liver Iron Concentration",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Hemochromatosis"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Mayo Clinic",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 18,
      "start_date": "2003-08",
      "completion_date": "2011-07",
      "has_results": false,
      "last_update_posted_date": "2016-01-22",
      "last_synced_at": "2026-10-14T04:45:34.129Z",
      "location_count": 1,
      "location_summary": "Rochester, Minnesota",
      "locations": [
        {
          "city": "Rochester",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00587535"
    },
    {
      "nct_id": "NCT01899950",
      "title": "Longitudinal Study of Cognition With Niemann-Pick Disease, Type C",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Niemann-Pick Disease, Type C"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Mayo Clinic",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": "99 Years",
        "sex": "ALL",
        "summary": "2 Years to 99 Years"
      },
      "enrollment_count": 70,
      "start_date": "2011-03",
      "completion_date": "2014-09",
      "has_results": false,
      "last_update_posted_date": "2016-01-06",
      "last_synced_at": "2026-10-14T04:45:34.129Z",
      "location_count": 1,
      "location_summary": "Rochester, Minnesota",
      "locations": [
        {
          "city": "Rochester",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01899950"
    },
    {
      "nct_id": "NCT01371825",
      "title": "Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2",
        "PHASE3"
      ],
      "conditions": [
        "Lysosomal Acid Lipase Deficiency",
        "Wolman Disease"
      ],
      "interventions": [
        {
          "name": "Sebelipase alfa (SBC-102)",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Alexion Pharmaceuticals, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "24 Months",
        "sex": "ALL",
        "summary": "Up to 24 Months"
      },
      "enrollment_count": 9,
      "start_date": "2011-05-04",
      "completion_date": "2018-01-03",
      "has_results": true,
      "last_update_posted_date": "2019-01-30",
      "last_synced_at": "2026-10-14T04:45:34.129Z",
      "location_count": 1,
      "location_summary": "Irvine, California",
      "locations": [
        {
          "city": "Irvine",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01371825"
    },
    {
      "nct_id": "NCT03720990",
      "title": "Smith-Lemli-Opitz Syndrome and Cholic Acid",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Smith-Lemli-Opitz Syndrome"
      ],
      "interventions": [
        {
          "name": "Cholic Acid",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "University of Nebraska",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": "25 Years",
        "sex": "ALL",
        "summary": "2 Years to 25 Years"
      },
      "enrollment_count": 12,
      "start_date": "2021-03-27",
      "completion_date": "2023-09-30",
      "has_results": true,
      "last_update_posted_date": "2024-04-30",
      "last_synced_at": "2026-10-14T04:45:34.129Z",
      "location_count": 2,
      "location_summary": "Aurora, Colorado • Omaha, Nebraska",
      "locations": [
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Omaha",
          "state": "Nebraska"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03720990"
    },
    {
      "nct_id": "NCT01109368",
      "title": "The Rogosin Institute Homozygous Familial Hypercholesterolemia Repository",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Homozygous Familial Hypercholesterolemia"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "The Rogosin Institute",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 60,
      "start_date": "2010-06",
      "completion_date": "2030-05",
      "has_results": false,
      "last_update_posted_date": "2026-04-06",
      "last_synced_at": "2026-10-14T04:45:34.129Z",
      "location_count": 1,
      "location_summary": "New York, New York",
      "locations": [
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01109368"
    }
  ]
}