{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=11",
    "query": {
      "condition": "Inborn Errors of Metabolism Disorders",
      "page": 11
    },
    "page_size": 10
  },
  "pagination": {
    "page": 11,
    "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=12&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=10&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-21T19:18:35.029Z",
  "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": "NCT03580083",
      "title": "RGX-111 Gene Therapy in Patients With MPS I",
      "overall_status": "SUSPENDED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Mucopolysaccharidosis Type I (MPS I)",
        "Hurler Syndrome",
        "Hurler-Scheie Syndrome"
      ],
      "interventions": [
        {
          "name": "RGX-111",
          "type": "GENETIC"
        }
      ],
      "intervention_types": [
        "GENETIC"
      ],
      "sponsor": "REGENXBIO Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "4 Months",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "4 Months and older"
      },
      "enrollment_count": 21,
      "start_date": "2019-04-03",
      "completion_date": "2029-03-06",
      "has_results": false,
      "last_update_posted_date": "2026-03-12",
      "last_synced_at": "2026-10-21T19:18:35.029Z",
      "location_count": 1,
      "location_summary": "Orange, California",
      "locations": [
        {
          "city": "Orange",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03580083"
    },
    {
      "nct_id": "NCT01675674",
      "title": "Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics",
      "overall_status": "TERMINATED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Mucopolysaccharidoses",
        "Mucopolysaccharidosis I",
        "Mucopolysaccharidosis II",
        "Mucopolysaccharidosis IV",
        "Mucopolysaccharidosis VI"
      ],
      "interventions": [
        {
          "name": "Dried blood spot test for MPS",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "National MPS Society",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Months",
        "maximum_age": "18 Years",
        "sex": "ALL",
        "summary": "6 Months to 18 Years"
      },
      "enrollment_count": 3000,
      "start_date": "2011-09",
      "completion_date": "2014-03",
      "has_results": false,
      "last_update_posted_date": "2013-05-24",
      "last_synced_at": "2026-10-21T19:18:35.029Z",
      "location_count": 2,
      "location_summary": "New Brunswick, New Jersey • New York, New York",
      "locations": [
        {
          "city": "New Brunswick",
          "state": "New Jersey"
        },
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01675674"
    },
    {
      "nct_id": "NCT00795197",
      "title": "A Screening Study Evaluating Disease Status of Gaucher Type I Patients",
      "overall_status": "WITHDRAWN",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Gaucher Disease, Type 1"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Genzyme, a Sanofi Company",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "16 Years",
        "maximum_age": "65 Years",
        "sex": "ALL",
        "summary": "16 Years to 65 Years"
      },
      "enrollment_count": 0,
      "start_date": "2008-12",
      "completion_date": "2009-12",
      "has_results": false,
      "last_update_posted_date": "2015-05-08",
      "last_synced_at": "2026-10-21T19:18:35.029Z",
      "location_count": 1,
      "location_summary": "New York, New York",
      "locations": [
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00795197"
    },
    {
      "nct_id": "NCT06149403",
      "title": "A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "MPS-IH (Hurler Syndrome)"
      ],
      "interventions": [
        {
          "name": "Experimental: OTL-203",
          "type": "GENETIC"
        },
        {
          "name": "Active Comparator: Allo-HSCT",
          "type": "GENETIC"
        }
      ],
      "intervention_types": [
        "GENETIC"
      ],
      "sponsor": "Orchard Therapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "28 Days",
        "maximum_age": "30 Months",
        "sex": "ALL",
        "summary": "28 Days to 30 Months"
      },
      "enrollment_count": 41,
      "start_date": "2023-12-11",
      "completion_date": "2031-03",
      "has_results": false,
      "last_update_posted_date": "2025-12-22",
      "last_synced_at": "2026-10-21T19:18:35.029Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06149403"
    },
    {
      "nct_id": "NCT01971957",
      "title": "Sjogren-Larsson Syndrome: Natural History, Clinical Variation and Evaluation of Biochemical Markers",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Sjogren-Larsson Syndrome (SLS)"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "University of Nebraska",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 20,
      "start_date": "2013-04-01",
      "completion_date": "2023-09-30",
      "has_results": false,
      "last_update_posted_date": "2024-10-08",
      "last_synced_at": "2026-10-21T19:18:35.029Z",
      "location_count": 2,
      "location_summary": "Omaha, Nebraska • Pittsburgh, Pennsylvania",
      "locations": [
        {
          "city": "Omaha",
          "state": "Nebraska"
        },
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01971957"
    },
    {
      "nct_id": "NCT04404530",
      "title": "Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Phenylketonurias"
      ],
      "interventions": [
        {
          "name": "Palynziq",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Emory University",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "16 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "16 Years and older"
      },
      "enrollment_count": 45,
      "start_date": "2019-10-08",
      "completion_date": "2028-12",
      "has_results": false,
      "last_update_posted_date": "2026-01-14",
      "last_synced_at": "2026-10-21T19:18:35.029Z",
      "location_count": 2,
      "location_summary": "Atlanta, Georgia",
      "locations": [
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04404530"
    },
    {
      "nct_id": "NCT06805695",
      "title": "Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Ornithine Transcarbamylase Deficiency",
        "Ornithine Transcarbamylase Deficiency Disease",
        "Urea Cycle Disorders, Inborn"
      ],
      "interventions": [
        {
          "name": "No Intervention",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "iECURE, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "7 Months",
        "maximum_age": "15 Months",
        "sex": "ALL",
        "summary": "7 Months to 15 Months"
      },
      "enrollment_count": 13,
      "start_date": "2024-12-23",
      "completion_date": "2041-07",
      "has_results": false,
      "last_update_posted_date": "2026-08-31",
      "last_synced_at": "2026-10-21T19:18:35.029Z",
      "location_count": 2,
      "location_summary": "Aurora, Colorado • New York, New York",
      "locations": [
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06805695"
    },
    {
      "nct_id": "NCT01078675",
      "title": "An Study to Evaluate Rosuvastatin in Children and Adolescents With Familial Hypercholesterolaemia",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Familial Hypercholesterolaemia"
      ],
      "interventions": [
        {
          "name": "rosuvastatin calcium",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "AstraZeneca",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Years",
        "maximum_age": "17 Years",
        "sex": "ALL",
        "summary": "6 Years to 17 Years"
      },
      "enrollment_count": 315,
      "start_date": "2010-02",
      "completion_date": "2013-02",
      "has_results": true,
      "last_update_posted_date": "2015-04-07",
      "last_synced_at": "2026-10-21T19:18:35.029Z",
      "location_count": 1,
      "location_summary": "Cincinnati, Ohio",
      "locations": [
        {
          "city": "Cincinnati",
          "state": "Ohio"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01078675"
    },
    {
      "nct_id": "NCT03770572",
      "title": "Gene Therapy for Children With CLN3 Batten Disease",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "CLN3",
        "Batten Disease"
      ],
      "interventions": [
        {
          "name": "Low dose CLN-301",
          "type": "GENETIC"
        },
        {
          "name": "High dose CLN-301",
          "type": "GENETIC"
        }
      ],
      "intervention_types": [
        "GENETIC"
      ],
      "sponsor": "Neela Therapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "3 Years",
        "maximum_age": "10 Years",
        "sex": "ALL",
        "summary": "3 Years to 10 Years"
      },
      "enrollment_count": 7,
      "start_date": "2018-11-13",
      "completion_date": "2029-09-30",
      "has_results": false,
      "last_update_posted_date": "2026-06-10",
      "last_synced_at": "2026-10-21T19:18:35.029Z",
      "location_count": 1,
      "location_summary": "Columbus, Ohio",
      "locations": [
        {
          "city": "Columbus",
          "state": "Ohio"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03770572"
    },
    {
      "nct_id": "NCT01572922",
      "title": "Massive Iron Deposit Assessment",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "NA"
      ],
      "conditions": [
        "Iron Overload",
        "Excessive Body Iron Burden"
      ],
      "interventions": [
        {
          "name": "R2*-UTE",
          "type": "DEVICE"
        },
        {
          "name": "R2*-GRE",
          "type": "DEVICE"
        },
        {
          "name": "Liver biopsy",
          "type": "PROCEDURE"
        }
      ],
      "intervention_types": [
        "DEVICE",
        "PROCEDURE"
      ],
      "sponsor": "St. Jude Children's Research Hospital",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 142,
      "start_date": "2012-06-11",
      "completion_date": "2018-02-28",
      "has_results": true,
      "last_update_posted_date": "2019-06-11",
      "last_synced_at": "2026-10-21T19:18:35.029Z",
      "location_count": 1,
      "location_summary": "Memphis, Tennessee",
      "locations": [
        {
          "city": "Memphis",
          "state": "Tennessee"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01572922"
    }
  ]
}