{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=25",
    "query": {
      "condition": "Inborn Errors of Metabolism Disorders",
      "page": 25
    },
    "page_size": 10
  },
  "pagination": {
    "page": 25,
    "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=26&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=24&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-25T15:33:15.530Z",
  "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": "NCT00004454",
      "title": "Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Mucopolysaccharidosis II"
      ],
      "interventions": [
        {
          "name": "lymphocyte gene therapy",
          "type": "GENETIC"
        }
      ],
      "intervention_types": [
        "GENETIC"
      ],
      "sponsor": "Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 2,
      "start_date": "1996-10",
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2005-06-24",
      "last_synced_at": "2026-10-25T15:33:15.530Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00004454"
    },
    {
      "nct_id": "NCT02112994",
      "title": "Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Lysosomal Acid Lipase Deficiency"
      ],
      "interventions": [
        {
          "name": "Sebelipase Alfa",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Alexion Pharmaceuticals, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "8 Months",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "8 Months and older"
      },
      "enrollment_count": 31,
      "start_date": "2014-06-24",
      "completion_date": "2017-12-28",
      "has_results": true,
      "last_update_posted_date": "2019-12-04",
      "last_synced_at": "2026-10-25T15:33:15.530Z",
      "location_count": 3,
      "location_summary": "Chicago, Illinois • Shreveport, Louisiana • Cincinnati, Ohio",
      "locations": [
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "Shreveport",
          "state": "Louisiana"
        },
        {
          "city": "Cincinnati",
          "state": "Ohio"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02112994"
    },
    {
      "nct_id": "NCT05418829",
      "title": "AT-007 in Adult Subjects With Classic Galactosemia (CG)",
      "overall_status": "UNKNOWN",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Classic Galactosemia"
      ],
      "interventions": [
        {
          "name": "AT-007",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Applied Therapeutics, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "65 Years",
        "sex": "ALL",
        "summary": "18 Years to 65 Years"
      },
      "enrollment_count": 7,
      "start_date": "2021-10-01",
      "completion_date": "2022-12",
      "has_results": false,
      "last_update_posted_date": "2022-06-14",
      "last_synced_at": "2026-10-25T15:33:15.530Z",
      "location_count": 1,
      "location_summary": "Berlin, New Jersey",
      "locations": [
        {
          "city": "Berlin",
          "state": "New Jersey"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05418829"
    },
    {
      "nct_id": "NCT04849741",
      "title": "A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Alexander Disease"
      ],
      "interventions": [
        {
          "name": "zilganersen",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Ionis Pharmaceuticals, Inc.",
      "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": 54,
      "start_date": "2021-06-01",
      "completion_date": "2034-07",
      "has_results": false,
      "last_update_posted_date": "2026-08-21",
      "last_synced_at": "2026-10-25T15:33:15.530Z",
      "location_count": 4,
      "location_summary": "Palo Alto, California • Atlanta, Georgia • Boston, Massachusetts + 1 more",
      "locations": [
        {
          "city": "Palo Alto",
          "state": "California"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Boston",
          "state": "Massachusetts"
        },
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04849741"
    },
    {
      "nct_id": "NCT04783181",
      "title": "A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Congenital Adrenal Hyperplasia"
      ],
      "interventions": [
        {
          "name": "AAV BBP-631",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Adrenas 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": 8,
      "start_date": "2021-07-01",
      "completion_date": "2029-02",
      "has_results": false,
      "last_update_posted_date": "2025-12-23",
      "last_synced_at": "2026-10-25T15:33:15.530Z",
      "location_count": 5,
      "location_summary": "Chicago, Illinois • Bethesda, Maryland • Minneapolis, Minnesota + 2 more",
      "locations": [
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "Bethesda",
          "state": "Maryland"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        },
        {
          "city": "Morehead City",
          "state": "North Carolina"
        },
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04783181"
    },
    {
      "nct_id": "NCT04628871",
      "title": "Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX",
      "overall_status": "TERMINATED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Hemophilia B",
        "Mucopolysaccharidosis I",
        "Mucopolysaccharidosis II"
      ],
      "interventions": [
        {
          "name": "SB-318",
          "type": "BIOLOGICAL"
        },
        {
          "name": "SB-913",
          "type": "BIOLOGICAL"
        },
        {
          "name": "SB-FIX",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Sangamo Therapeutics",
      "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": "2020-11-03",
      "completion_date": "2026-06-17",
      "has_results": false,
      "last_update_posted_date": "2026-08-13",
      "last_synced_at": "2026-10-25T15:33:15.530Z",
      "location_count": 5,
      "location_summary": "Oakland, California • Chicago, Illinois • New York, New York + 2 more",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Chapel Hill",
          "state": "North Carolina"
        },
        {
          "city": "Cincinnati",
          "state": "Ohio"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04628871"
    },
    {
      "nct_id": "NCT00730236",
      "title": "A Safety and Efficacy Study of AEGR-733 to Treat Homozygous Familial Hypercholesterolemia (FH)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Homozygous Familial Hypercholesterolemia"
      ],
      "interventions": [
        {
          "name": "AEGR-733",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Aegerion Pharmaceuticals, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 29,
      "start_date": "2007-12",
      "completion_date": "2011-10",
      "has_results": true,
      "last_update_posted_date": "2018-03-20",
      "last_synced_at": "2026-10-25T15:33:15.530Z",
      "location_count": 2,
      "location_summary": "Los Angeles, California • Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00730236"
    },
    {
      "nct_id": "NCT02488044",
      "title": "A Phase 1/2 Study of AEB1102 in Patients With Arginase I Deficiency",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Arginase I Deficiency",
        "Hyperargininemia"
      ],
      "interventions": [
        {
          "name": "AEB1102",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Aeglea Biotherapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "2 Years and older"
      },
      "enrollment_count": 16,
      "start_date": "2016-06",
      "completion_date": "2019-02",
      "has_results": false,
      "last_update_posted_date": "2019-06-12",
      "last_synced_at": "2026-10-25T15:33:15.530Z",
      "location_count": 6,
      "location_summary": "Stanford, California • Gainesville, Florida • Detroit, Michigan + 3 more",
      "locations": [
        {
          "city": "Stanford",
          "state": "California"
        },
        {
          "city": "Gainesville",
          "state": "Florida"
        },
        {
          "city": "Detroit",
          "state": "Michigan"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Dallas",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02488044"
    },
    {
      "nct_id": "NCT00808106",
      "title": "Clinical, Cellular, and Molecular Investigation Into Oculocutaneous Albinism",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Oculocutaneous Albinism"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "National Human Genome Research Institute (NHGRI)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Year",
        "maximum_age": "80 Years",
        "sex": "ALL",
        "summary": "1 Year to 80 Years"
      },
      "enrollment_count": 206,
      "start_date": "2008-12-11",
      "completion_date": "2019-12-31",
      "has_results": false,
      "last_update_posted_date": "2020-01-06",
      "last_synced_at": "2026-10-25T15:33:15.530Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00808106"
    },
    {
      "nct_id": "NCT02798458",
      "title": "Evaluation of the Gastrointestinal Manifestation of Fabry's Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "NA"
      ],
      "conditions": [
        "Fabry's Disease"
      ],
      "interventions": [
        {
          "name": "Smartpill",
          "type": "DEVICE"
        },
        {
          "name": "Endoscopic Mucosal Resection",
          "type": "PROCEDURE"
        }
      ],
      "intervention_types": [
        "DEVICE",
        "PROCEDURE"
      ],
      "sponsor": "Massachusetts General Hospital",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "70 Years",
        "sex": "ALL",
        "summary": "18 Years to 70 Years"
      },
      "enrollment_count": 48,
      "start_date": "2016-05",
      "completion_date": "2022-11",
      "has_results": true,
      "last_update_posted_date": "2023-03-27",
      "last_synced_at": "2026-10-25T15:33:15.530Z",
      "location_count": 1,
      "location_summary": "Boston, Massachusetts",
      "locations": [
        {
          "city": "Boston",
          "state": "Massachusetts"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02798458"
    }
  ]
}