{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=37",
    "query": {
      "condition": "Inborn Errors of Metabolism",
      "page": 37
    },
    "page_size": 10
  },
  "pagination": {
    "page": 37,
    "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=38&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=36&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-17T13:31:04.989Z",
  "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": "NCT04453085",
      "title": "An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Mucopolysaccharidosis I"
      ],
      "interventions": [
        {
          "name": "JR-171",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "JCR Pharmaceuticals Co., Ltd.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 14,
      "start_date": "2021-10-28",
      "completion_date": "2025-05-02",
      "has_results": false,
      "last_update_posted_date": "2025-08-28",
      "last_synced_at": "2026-10-17T13:31:04.989Z",
      "location_count": 1,
      "location_summary": "Oakland, California",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04453085"
    },
    {
      "nct_id": "NCT01414985",
      "title": "AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Late Infantile Neuronal Ceroid Lipofuscinosis",
        "Batten Disease"
      ],
      "interventions": [
        {
          "name": "AAVrh.10CUCLN2",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Weill Medical College of Cornell University",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "3 Years",
        "maximum_age": "18 Years",
        "sex": "ALL",
        "summary": "3 Years to 18 Years"
      },
      "enrollment_count": 8,
      "start_date": "2010-04-15",
      "completion_date": "2017-02-08",
      "has_results": false,
      "last_update_posted_date": "2020-08-31",
      "last_synced_at": "2026-10-17T13:31:04.989Z",
      "location_count": 1,
      "location_summary": "New York, New York",
      "locations": [
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01414985"
    },
    {
      "nct_id": "NCT07236606",
      "title": "RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "MPS II",
        "Hunter Syndrome (MPS II)"
      ],
      "interventions": [
        {
          "name": "RGX-121-3102",
          "type": "GENETIC"
        }
      ],
      "intervention_types": [
        "GENETIC"
      ],
      "sponsor": "REGENXBIO Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "4 Months",
        "maximum_age": "5 Years",
        "sex": "MALE",
        "summary": "4 Months to 5 Years · Male only"
      },
      "enrollment_count": 2,
      "start_date": "2025-11-25",
      "completion_date": "2031-05-30",
      "has_results": false,
      "last_update_posted_date": "2026-06-04",
      "last_synced_at": "2026-10-17T13:31:04.989Z",
      "location_count": 1,
      "location_summary": "New Brunswick, New Jersey",
      "locations": [
        {
          "city": "New Brunswick",
          "state": "New Jersey"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07236606"
    },
    {
      "nct_id": "NCT00048906",
      "title": "Alpha-Galactosidase A Replacement Therapy for Fabry Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [
        {
          "name": "DRX005B",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "National Institute of Neurological Disorders and Stroke (NINDS)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 3,
      "start_date": "2002-11",
      "completion_date": "2003-11",
      "has_results": false,
      "last_update_posted_date": "2008-03-04",
      "last_synced_at": "2026-10-17T13:31:04.989Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00048906"
    },
    {
      "nct_id": "NCT05494593",
      "title": "A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)",
      "overall_status": "WITHDRAWN",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE4"
      ],
      "conditions": [
        "Mucopolysaccharidosis (MPS)",
        "Hunter Syndrome"
      ],
      "interventions": [
        {
          "name": "ELAPRASE",
          "type": "DRUG"
        },
        {
          "name": "Rituximab",
          "type": "DRUG"
        },
        {
          "name": "Methotrexate",
          "type": "DRUG"
        },
        {
          "name": "Intravenous Immunoglobulin (IVIG)",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Takeda",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "6 Years",
        "sex": "MALE",
        "summary": "Up to 6 Years · Male only"
      },
      "enrollment_count": 0,
      "start_date": "2023-02-28",
      "completion_date": "2025-08-29",
      "has_results": false,
      "last_update_posted_date": "2025-12-09",
      "last_synced_at": "2026-10-17T13:31:04.989Z",
      "location_count": 8,
      "location_summary": "Phoenix, Arizona • Oakland, California • San Diego, California + 5 more",
      "locations": [
        {
          "city": "Phoenix",
          "state": "Arizona"
        },
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "San Diego",
          "state": "California"
        },
        {
          "city": "Torrance",
          "state": "California"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05494593"
    },
    {
      "nct_id": "NCT06214507",
      "title": "Danon Disease Natural History Study",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Danon Disease"
      ],
      "interventions": [
        {
          "name": "No intervention",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "Rocket Pharmaceuticals Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "8 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "8 Years and older"
      },
      "enrollment_count": 60,
      "start_date": "2023-12-20",
      "completion_date": "2030-03",
      "has_results": false,
      "last_update_posted_date": "2026-07-28",
      "last_synced_at": "2026-10-17T13:31:04.989Z",
      "location_count": 9,
      "location_summary": "Phoenix, Arizona • La Jolla, California • Aurora, Colorado + 6 more",
      "locations": [
        {
          "city": "Phoenix",
          "state": "Arizona"
        },
        {
          "city": "La Jolla",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Gainesville",
          "state": "Florida"
        },
        {
          "city": "Ann Arbor",
          "state": "Michigan"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06214507"
    },
    {
      "nct_id": "NCT07643844",
      "title": "AAVrh10-PCCA Gene Therapy for Propionic Acidemia",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Propionic Acidemia"
      ],
      "interventions": [
        {
          "name": "AAVrh10-PCCA low dose",
          "type": "DRUG"
        },
        {
          "name": "AAVrh10-PCCA middle dose",
          "type": "DRUG"
        },
        {
          "name": "AAVrh10-PCCA high dose",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Mayo Clinic",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Months",
        "maximum_age": "2 Years",
        "sex": "ALL",
        "summary": "6 Months to 2 Years"
      },
      "enrollment_count": 9,
      "start_date": "2026-07-20",
      "completion_date": "2033-12",
      "has_results": false,
      "last_update_posted_date": "2026-08-21",
      "last_synced_at": "2026-10-17T13:31:04.989Z",
      "location_count": 1,
      "location_summary": "Rochester, Minnesota",
      "locations": [
        {
          "city": "Rochester",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07643844"
    },
    {
      "nct_id": "NCT05682144",
      "title": "ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Mucopolysaccharidosis IH/S",
        "Mucopolysaccharidosis IS"
      ],
      "interventions": [
        {
          "name": "Autologous Plasmablasts (B cells)",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Immusoft of CA, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "10 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "10 Years and older"
      },
      "enrollment_count": 11,
      "start_date": "2023-04-12",
      "completion_date": "2044-06-01",
      "has_results": false,
      "last_update_posted_date": "2026-07-17",
      "last_synced_at": "2026-10-17T13:31:04.989Z",
      "location_count": 2,
      "location_summary": "Oakland, California • Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05682144"
    },
    {
      "nct_id": "NCT03639844",
      "title": "BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study",
      "overall_status": "NO_LONGER_AVAILABLE",
      "study_type": "EXPANDED_ACCESS",
      "phases": [],
      "conditions": [
        "Hurler Syndrome",
        "Inherited Metabolic Disorder",
        "Lysosomal Storage Disorder",
        "Metachromatic Leukodystrophy",
        "Inborn Errors of Metabolism"
      ],
      "interventions": [
        {
          "name": "rivogenlecleucel",
          "type": "BIOLOGICAL"
        },
        {
          "name": "rimiducid",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL",
        "DRUG"
      ],
      "sponsor": "Bellicum Pharmaceuticals",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": null,
      "eligibility": {
        "minimum_age": "3 Months",
        "maximum_age": "21 Years",
        "sex": "ALL",
        "summary": "3 Months to 21 Years"
      },
      "enrollment_count": null,
      "start_date": null,
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2020-10-05",
      "last_synced_at": "2026-10-17T13:31:04.989Z",
      "location_count": 2,
      "location_summary": "Los Angeles, California • Palo Alto, California",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Palo Alto",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03639844"
    },
    {
      "nct_id": "NCT02648269",
      "title": "Safety and Pharmacodynamics of SEL-212 (Pegsiticase + SEL-110) in Subjects With Elevated Blood Uric Acid Levels",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Gout"
      ],
      "interventions": [
        {
          "name": "SEL-110",
          "type": "DRUG"
        },
        {
          "name": "SEL-212",
          "type": "BIOLOGICAL"
        },
        {
          "name": "SEL-037",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "DRUG",
        "BIOLOGICAL"
      ],
      "sponsor": "Selecta Biosciences, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "21 Years",
        "maximum_age": "70 Years",
        "sex": "ALL",
        "summary": "21 Years to 70 Years"
      },
      "enrollment_count": 63,
      "start_date": "2015-12",
      "completion_date": "2016-12",
      "has_results": false,
      "last_update_posted_date": "2017-01-30",
      "last_synced_at": "2026-10-17T13:31:04.989Z",
      "location_count": 6,
      "location_summary": "Little Rock, Arkansas • Miami, Florida • Orlando, Florida + 3 more",
      "locations": [
        {
          "city": "Little Rock",
          "state": "Arkansas"
        },
        {
          "city": "Miami",
          "state": "Florida"
        },
        {
          "city": "Orlando",
          "state": "Florida"
        },
        {
          "city": "Baltimore",
          "state": "Maryland"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02648269"
    }
  ]
}