{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=12",
    "query": {
      "condition": "Inborn Errors of Metabolism Disorders",
      "page": 12
    },
    "page_size": 10
  },
  "pagination": {
    "page": 12,
    "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=13&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=11&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-21T20:03:28.740Z",
  "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": "NCT02904772",
      "title": "Alipogene Tiparvovec for the Treatment of LPLD Patients",
      "overall_status": "WITHDRAWN",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "LPL Deficiency"
      ],
      "interventions": [
        {
          "name": "alipogene tiparvovec",
          "type": "DRUG"
        },
        {
          "name": "Prednisolone",
          "type": "DRUG"
        },
        {
          "name": "Cyclosporins",
          "type": "DRUG"
        },
        {
          "name": "Mycophenolate mofetil",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "UniQure Biopharma B.V.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 0,
      "start_date": "2016-10",
      "completion_date": "2020-09",
      "has_results": false,
      "last_update_posted_date": "2017-08-17",
      "last_synced_at": "2026-10-21T20:03:28.740Z",
      "location_count": 1,
      "location_summary": "Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02904772"
    },
    {
      "nct_id": "NCT04476862",
      "title": "Cerliponase Alfa Observational Study in the US",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2"
      ],
      "interventions": [
        {
          "name": "Cerliponase Alfa",
          "type": "DRUG"
        },
        {
          "name": "Administration Kit",
          "type": "DEVICE"
        }
      ],
      "intervention_types": [
        "DRUG",
        "DEVICE"
      ],
      "sponsor": "BioMarin Pharmaceutical",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 35,
      "start_date": "2020-08-19",
      "completion_date": "2030-08-31",
      "has_results": false,
      "last_update_posted_date": "2026-06-30",
      "last_synced_at": "2026-10-21T20:03:28.740Z",
      "location_count": 16,
      "location_summary": "Little Rock, Arkansas • Orange, California • Aurora, Colorado + 12 more",
      "locations": [
        {
          "city": "Little Rock",
          "state": "Arkansas"
        },
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Washington D.C.",
          "state": "District of Columbia"
        },
        {
          "city": "Orlando",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04476862"
    },
    {
      "nct_id": "NCT01339494",
      "title": "Nitric Oxide Production in MELAS Syndrome",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "EARLY_PHASE1"
      ],
      "conditions": [
        "MELAS Syndrome"
      ],
      "interventions": [
        {
          "name": "Arginine and citrulline supplementations",
          "type": "DIETARY_SUPPLEMENT"
        }
      ],
      "intervention_types": [
        "DIETARY_SUPPLEMENT"
      ],
      "sponsor": "Baylor College of Medicine",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "3 Years",
        "maximum_age": "65 Years",
        "sex": "ALL",
        "summary": "3 Years to 65 Years"
      },
      "enrollment_count": 30,
      "start_date": "2009-07",
      "completion_date": "2016-04",
      "has_results": false,
      "last_update_posted_date": "2016-04-05",
      "last_synced_at": "2026-10-21T20:03:28.740Z",
      "location_count": 1,
      "location_summary": "Houston, Texas",
      "locations": [
        {
          "city": "Houston",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01339494"
    },
    {
      "nct_id": "NCT00000894",
      "title": "Comparison of Two Drugs, Cidofovir and Ganciclovir, in Treating Patients With AIDS Who Have CMV Retinitis",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE4"
      ],
      "conditions": [
        "Cytomegalovirus Retinitis",
        "HIV Infections"
      ],
      "interventions": [
        {
          "name": "Cidofovir",
          "type": "DRUG"
        },
        {
          "name": "Probenecid",
          "type": "DRUG"
        },
        {
          "name": "Ganciclovir",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "National Institute of Allergy and Infectious Diseases (NIAID)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "13 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "13 Years and older"
      },
      "enrollment_count": 300,
      "start_date": null,
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2011-02-23",
      "last_synced_at": "2026-10-21T20:03:28.740Z",
      "location_count": 12,
      "location_summary": "Los Angeles, California • Miami, Florida • Atlanta, Georgia + 7 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Miami",
          "state": "Florida"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00000894"
    },
    {
      "nct_id": "NCT00215527",
      "title": "Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Mucopolysaccharidosis I",
        "Lysosomal Storage Diseases",
        "Spinal Cord Compression"
      ],
      "interventions": [
        {
          "name": "laronidase",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Patricia I. Dickson, M.D.",
      "sponsor_class": "INDIV",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "8 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "8 Years and older"
      },
      "enrollment_count": 4,
      "start_date": "2005-11",
      "completion_date": "2011-10",
      "has_results": false,
      "last_update_posted_date": "2013-02-21",
      "last_synced_at": "2026-10-21T20:03:28.740Z",
      "location_count": 1,
      "location_summary": "Torrance, California",
      "locations": [
        {
          "city": "Torrance",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00215527"
    },
    {
      "nct_id": "NCT00084305",
      "title": "Analysis of Specimens From Individuals With Pulmonary Fibrosis",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Pulmonary Fibrosis",
        "Healthy Volunteers",
        "Hermansky-Pudlak Syndrome (HPS)"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "National Human Genome Research Institute (NHGRI)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "115 Years",
        "sex": "ALL",
        "summary": "18 Years to 115 Years"
      },
      "enrollment_count": 315,
      "start_date": "2004-06-09",
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2026-10-21",
      "last_synced_at": "2026-10-21T20:03:28.740Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00084305"
    },
    {
      "nct_id": "NCT03440905",
      "title": "Proxy-Reported Symptoms and Quality of Life Survey in Zellweger Spectrum Disorders",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Zellweger Spectrum"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "University of South Florida",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 92,
      "start_date": "2018-01-29",
      "completion_date": "2018-11-30",
      "has_results": false,
      "last_update_posted_date": "2019-10-16",
      "last_synced_at": "2026-10-21T20:03:28.740Z",
      "location_count": 1,
      "location_summary": "Tampa, Florida",
      "locations": [
        {
          "city": "Tampa",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03440905"
    },
    {
      "nct_id": "NCT00477594",
      "title": "Open Label Extension of ISIS 301012 (Mipomersen) to Treat Familial Hypercholesterolemia",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Lipid Metabolism, Inborn Errors",
        "Hypercholesterolemia, Autosomal Dominant",
        "Hyperlipidemias",
        "Metabolic Diseases",
        "Hyperlipoproteinemia Type II",
        "Metabolism, Inborn Errors",
        "Genetic Diseases, Inborn",
        "Infant, Newborn, Diseases",
        "Metabolic Disorder",
        "Congenital Abnormalities",
        "Hypercholesterolemia",
        "Hyperlipoproteinemias",
        "Dyslipidemias",
        "Lipid Metabolism Disorders"
      ],
      "interventions": [
        {
          "name": "mipomersen sodium",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Kastle Therapeutics, LLC",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "12 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "12 Years and older"
      },
      "enrollment_count": 21,
      "start_date": "2007-05",
      "completion_date": "2011-07",
      "has_results": true,
      "last_update_posted_date": "2016-09-09",
      "last_synced_at": "2026-10-21T20:03:28.740Z",
      "location_count": 5,
      "location_summary": "Chicago, Illinois • Auburn, Maine • Biddeford, Maine + 2 more",
      "locations": [
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "Auburn",
          "state": "Maine"
        },
        {
          "city": "Biddeford",
          "state": "Maine"
        },
        {
          "city": "Scarborough",
          "state": "Maine"
        },
        {
          "city": "Cincinnati",
          "state": "Ohio"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00477594"
    },
    {
      "nct_id": "NCT06065605",
      "title": "Assess Urine Biomarkers to Predict Nephropathy in Fabry Disease",
      "overall_status": "UNKNOWN",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Lysosomal and Rare Disorders Research and Treatment Center, Inc.",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "80 Years",
        "sex": "ALL",
        "summary": "18 Years to 80 Years"
      },
      "enrollment_count": 40,
      "start_date": "2023-09-14",
      "completion_date": "2025-09-12",
      "has_results": false,
      "last_update_posted_date": "2023-10-04",
      "last_synced_at": "2026-10-21T20:03:28.740Z",
      "location_count": 2,
      "location_summary": "Rockville, Maryland • Fairfax, Virginia",
      "locations": [
        {
          "city": "Rockville",
          "state": "Maryland"
        },
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06065605"
    },
    {
      "nct_id": "NCT02194985",
      "title": "Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [
        {
          "name": "migalastat HCl 150 mg",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Amicus Therapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 84,
      "start_date": "2015-03-14",
      "completion_date": "2019-10-23",
      "has_results": true,
      "last_update_posted_date": "2020-12-21",
      "last_synced_at": "2026-10-21T20:03:28.740Z",
      "location_count": 7,
      "location_summary": "Atlanta, Georgia • Kansas City, Kansas • Grand Rapids, Michigan + 4 more",
      "locations": [
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Kansas City",
          "state": "Kansas"
        },
        {
          "city": "Grand Rapids",
          "state": "Michigan"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Portland",
          "state": "Oregon"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02194985"
    }
  ]
}