{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=40",
    "query": {
      "condition": "Inborn Errors of Metabolism",
      "page": 40
    },
    "page_size": 10
  },
  "pagination": {
    "page": 40,
    "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=41&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=39&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-18T07:31:10.603Z",
  "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": "NCT02791230",
      "title": "Long-term Safety of Tafamidis in Subjects With Transthyretin Cardiomyopathy",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Transthyretin (TTR) Amyloid Cardiomyopathy"
      ],
      "interventions": [
        {
          "name": "Tafamidis",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Pfizer",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 1733,
      "start_date": "2016-06-13",
      "completion_date": "2023-11-02",
      "has_results": true,
      "last_update_posted_date": "2025-02-03",
      "last_synced_at": "2026-10-18T07:31:10.603Z",
      "location_count": 41,
      "location_summary": "Birmingham, Alabama • Beverly Hills, California • La Jolla, California + 20 more",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Beverly Hills",
          "state": "California"
        },
        {
          "city": "Beverly Hills",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02791230"
    },
    {
      "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": "2026-10-18T07:31:10.603Z",
      "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": "NCT03156621",
      "title": "Study in Participants With Homozygous Familial Hypercholesterolemia (HoFH)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Homozygous Familial Hypercholesterolemia"
      ],
      "interventions": [
        {
          "name": "Alirocumab",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Regeneron Pharmaceuticals",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 69,
      "start_date": "2017-10-03",
      "completion_date": "2020-02-13",
      "has_results": true,
      "last_update_posted_date": "2021-06-29",
      "last_synced_at": "2026-10-18T07:31:10.603Z",
      "location_count": 4,
      "location_summary": "Boca Raton, Florida • New York, New York • Cincinnati, Ohio + 1 more",
      "locations": [
        {
          "city": "Boca Raton",
          "state": "Florida"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Cincinnati",
          "state": "Ohio"
        },
        {
          "city": "Dallas",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03156621"
    },
    {
      "nct_id": "NCT04941599",
      "title": "2-Hydroxybenzylamine (2-HOBA) to Reduce HDL Modification and Improve HDL Function in Familial Hypercholesterolemia (FH)",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Familial Hypercholesterolemia"
      ],
      "interventions": [
        {
          "name": "2-Hydroxybenzylamine",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "DRUG",
        "OTHER"
      ],
      "sponsor": "Vanderbilt University Medical Center",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "69 Years",
        "sex": "ALL",
        "summary": "18 Years to 69 Years"
      },
      "enrollment_count": 72,
      "start_date": "2024-02-14",
      "completion_date": "2027-12-31",
      "has_results": false,
      "last_update_posted_date": "2026-08-07",
      "last_synced_at": "2026-10-18T07:31:10.603Z",
      "location_count": 1,
      "location_summary": "Nashville, Tennessee",
      "locations": [
        {
          "city": "Nashville",
          "state": "Tennessee"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04941599"
    },
    {
      "nct_id": "NCT03718234",
      "title": "Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Congenital Adrenal Hyperplasia",
        "Hyperplasia",
        "Adrenal Hyperplasia",
        "Congenital Disorders",
        "Adrenocortical Hyperfunction",
        "Disorders of Sex Development",
        "Urogenital Abnormalities",
        "Genetic Diseases, Inborn",
        "Steroid Metabolic Diseases, Inborn",
        "Adrenal Gland Disease",
        "Hydrocortisone"
      ],
      "interventions": [
        {
          "name": "Subcutaneous hydrocortisone",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "University of Minnesota",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "4 Years",
        "maximum_age": "18 Years",
        "sex": "ALL",
        "summary": "4 Years to 18 Years"
      },
      "enrollment_count": 11,
      "start_date": "2019-01-01",
      "completion_date": "2023-02-23",
      "has_results": true,
      "last_update_posted_date": "2025-06-25",
      "last_synced_at": "2026-10-18T07:31:10.603Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03718234"
    },
    {
      "nct_id": "NCT06150716",
      "title": "Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Pelizaeus-Merzbacher Disease"
      ],
      "interventions": [
        {
          "name": "ION356",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Ionis Pharmaceuticals, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": "17 Years",
        "sex": "MALE",
        "summary": "2 Years to 17 Years · Male only"
      },
      "enrollment_count": 24,
      "start_date": "2024-04-10",
      "completion_date": "2028-06",
      "has_results": false,
      "last_update_posted_date": "2025-12-12",
      "last_synced_at": "2026-10-18T07:31:10.603Z",
      "location_count": 3,
      "location_summary": "Atlanta, Georgia • Philadelphia, Pennsylvania • Salt Lake City, Utah",
      "locations": [
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        },
        {
          "city": "Salt Lake City",
          "state": "Utah"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06150716"
    },
    {
      "nct_id": "NCT04828863",
      "title": "Neurocognitive Outcomes and Quality of Life in Adults With Maple Syrup Urine Disease (MSUD)",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Maple Syrup Urine Disease"
      ],
      "interventions": [
        {
          "name": "Neurocognitive testing",
          "type": "BEHAVIORAL"
        }
      ],
      "intervention_types": [
        "BEHAVIORAL"
      ],
      "sponsor": "Children's Hospital of Philadelphia",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "21 Years",
        "maximum_age": "80 Years",
        "sex": "ALL",
        "summary": "21 Years to 80 Years"
      },
      "enrollment_count": 44,
      "start_date": "2021-06-12",
      "completion_date": "2023-11-30",
      "has_results": false,
      "last_update_posted_date": "2024-06-04",
      "last_synced_at": "2026-10-18T07:31:10.603Z",
      "location_count": 1,
      "location_summary": "Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04828863"
    },
    {
      "nct_id": "NCT06019728",
      "title": "A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE4"
      ],
      "conditions": [
        "Fabry's Disease"
      ],
      "interventions": [
        {
          "name": "AGALSIDASE BETA (GZ419828)",
          "type": "DRUG"
        },
        {
          "name": "Acetaminophen",
          "type": "DRUG"
        },
        {
          "name": "Diphenhydramine",
          "type": "DRUG"
        },
        {
          "name": "Dexamethasone",
          "type": "DRUG"
        },
        {
          "name": "Montelukast",
          "type": "DRUG"
        },
        {
          "name": "Loratadine",
          "type": "DRUG"
        },
        {
          "name": "Cetirizine",
          "type": "DRUG"
        },
        {
          "name": "Fexofenadine",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Sanofi",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": "65 Years",
        "sex": "ALL",
        "summary": "2 Years to 65 Years"
      },
      "enrollment_count": 8,
      "start_date": "2023-11-10",
      "completion_date": "2024-10-25",
      "has_results": true,
      "last_update_posted_date": "2025-10-21",
      "last_synced_at": "2026-10-18T07:31:10.603Z",
      "location_count": 5,
      "location_summary": "Santa Monica, California • Atlanta, Georgia • Grand Rapids, Michigan + 2 more",
      "locations": [
        {
          "city": "Santa Monica",
          "state": "California"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Grand Rapids",
          "state": "Michigan"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06019728"
    },
    {
      "nct_id": "NCT04457336",
      "title": "A Ph2b to Evaluate Clinical Efficacy and Safety of Tildacerfont in Adult CAH",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Congenital Adrenal Hyperplasia"
      ],
      "interventions": [
        {
          "name": "Tildacerfont/Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Spruce Biosciences",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 96,
      "start_date": "2020-08-26",
      "completion_date": "2024-05-23",
      "has_results": true,
      "last_update_posted_date": "2025-07-08",
      "last_synced_at": "2026-10-18T07:31:10.603Z",
      "location_count": 28,
      "location_summary": "Birmingham, Alabama • Los Angeles, California • Orange, California + 24 more",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "Sacramento",
          "state": "California"
        },
        {
          "city": "Englewood",
          "state": "Colorado"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04457336"
    },
    {
      "nct_id": "NCT00425607",
      "title": "Phase II Trial of Lonafarnib (a Farnesyltransferase Inhibitor) for Progeria",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Progeria",
        "Hutchinson-Gilford Syndrome"
      ],
      "interventions": [
        {
          "name": "Lonafarnib",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Monica E. Kleinman",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Year",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "1 Year and older"
      },
      "enrollment_count": 29,
      "start_date": "2007-05",
      "completion_date": "2009-10",
      "has_results": true,
      "last_update_posted_date": "2019-06-25",
      "last_synced_at": "2026-10-18T07:31:10.603Z",
      "location_count": 1,
      "location_summary": "Boston, Massachusetts",
      "locations": [
        {
          "city": "Boston",
          "state": "Massachusetts"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00425607"
    }
  ]
}