{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Lipid+Metabolism%2C+Inborn+Errors&page=12",
    "query": {
      "condition": "Lipid Metabolism, Inborn Errors",
      "page": 12
    },
    "page_size": 10
  },
  "pagination": {
    "page": 12,
    "page_size": 10,
    "total_count": 501,
    "total_pages": 51,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Lipid+Metabolism%2C+Inborn+Errors&page=13&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Lipid+Metabolism%2C+Inborn+Errors&page=11&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-14T10:49:56.933Z",
  "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": "NCT01003912",
      "title": "Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases",
      "overall_status": "WITHDRAWN",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Lysosomal Storage Diseases",
        "Inborn Errors of Metabolism"
      ],
      "interventions": [
        {
          "name": "ALD-601",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Joanne Kurtzberg, MD",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "FEMALE",
        "summary": "18 Years and older · Female only"
      },
      "enrollment_count": 0,
      "start_date": "2009-10",
      "completion_date": "2017-03",
      "has_results": false,
      "last_update_posted_date": "2017-06-29",
      "last_synced_at": "2026-10-14T10:49:56.933Z",
      "location_count": 2,
      "location_summary": "Durham, North Carolina",
      "locations": [
        {
          "city": "Durham",
          "state": "North Carolina"
        },
        {
          "city": "Durham",
          "state": "North Carolina"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01003912"
    },
    {
      "nct_id": "NCT03333200",
      "title": "Longitudinal Study of Neurodegenerative Disorders",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "MLD",
        "Krabbe Disease",
        "ALD",
        "MPS I",
        "MPS II",
        "MPS III",
        "Vanishing White Matter Disease",
        "GM3 Gangliosidosis",
        "PKAN",
        "Tay-Sachs Disease",
        "NP Deficiency",
        "Osteopetrosis",
        "Alpha-Mannosidosis",
        "Sandhoff Disease",
        "Niemann-Pick Diseases",
        "MPS IV",
        "Gaucher Disease",
        "GAN",
        "GM1 Gangliosidoses",
        "Morquio Disease",
        "S-Adenosylhomocysteine Hydrolase Deficiency",
        "Batten Disease",
        "Pelizaeus-Merzbacher Disease",
        "Leukodystrophy",
        "Lysosomal Storage Diseases",
        "Purine Nucleoside Phosphorylase Deficiency",
        "Multiple Sulfatase Deficiency Disease"
      ],
      "interventions": [
        {
          "name": "Palliative Care",
          "type": "OTHER"
        },
        {
          "name": "Hematopoetic Stem Cell Transplantation",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "OTHER",
        "BIOLOGICAL"
      ],
      "sponsor": "University of Pittsburgh",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 1500,
      "start_date": "2012-01-11",
      "completion_date": "2035-01",
      "has_results": false,
      "last_update_posted_date": "2026-02-09",
      "last_synced_at": "2026-10-14T10:49:56.933Z",
      "location_count": 1,
      "location_summary": "Pittsburgh, Pennsylvania",
      "locations": [
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03333200"
    },
    {
      "nct_id": "NCT01019629",
      "title": "Fabry Screening Study",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Baylor Research Institute",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 2724,
      "start_date": "2009-01-29",
      "completion_date": "2017-12-07",
      "has_results": false,
      "last_update_posted_date": "2018-02-13",
      "last_synced_at": "2026-10-14T10:49:56.933Z",
      "location_count": 1,
      "location_summary": "Dallas, Texas",
      "locations": [
        {
          "city": "Dallas",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01019629"
    },
    {
      "nct_id": "NCT00679744",
      "title": "A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis",
      "overall_status": "WITHDRAWN",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "G(M2) Ganglioside",
        "Tay-Sachs Disease Ganglioside",
        "Sandhoff Disease Ganglioside"
      ],
      "interventions": [
        {
          "name": "Pyrimethamine",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Exsar Corporation",
      "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": "2008-05",
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2013-02-25",
      "last_synced_at": "2026-10-14T10:49:56.933Z",
      "location_count": 2,
      "location_summary": "New York, New York • Cleveland, Ohio",
      "locations": [
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Cleveland",
          "state": "Ohio"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00679744"
    },
    {
      "nct_id": "NCT01434745",
      "title": "SLOS: The Effect of Simvastatin in Patients Receiving Cholesterol Supplementation",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "NA"
      ],
      "conditions": [
        "Smith-Lemli-Opitz Syndrome"
      ],
      "interventions": [
        {
          "name": "Simvastatin",
          "type": "DRUG"
        },
        {
          "name": "Lactose",
          "type": "DIETARY_SUPPLEMENT"
        }
      ],
      "intervention_types": [
        "DRUG",
        "DIETARY_SUPPLEMENT"
      ],
      "sponsor": "Oregon Health and Science University",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Year",
        "maximum_age": "89 Years",
        "sex": "ALL",
        "summary": "1 Year to 89 Years"
      },
      "enrollment_count": 1,
      "start_date": "2011-09",
      "completion_date": "2014-10",
      "has_results": true,
      "last_update_posted_date": "2019-10-01",
      "last_synced_at": "2026-10-14T10:49:56.933Z",
      "location_count": 1,
      "location_summary": "Portland, Oregon",
      "locations": [
        {
          "city": "Portland",
          "state": "Oregon"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01434745"
    },
    {
      "nct_id": "NCT00230607",
      "title": "Study of the Effects of Fabrazyme Treatment on Lactation and Infants",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE4"
      ],
      "conditions": [
        "Fabry Disease",
        "Alpha Galactosidase A Deficiency"
      ],
      "interventions": [
        {
          "name": "agalsidase beta",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Genzyme, a Sanofi Company",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 7,
      "start_date": "2006-05-28",
      "completion_date": "2024-02-09",
      "has_results": true,
      "last_update_posted_date": "2024-04-10",
      "last_synced_at": "2026-10-14T10:49:56.933Z",
      "location_count": 3,
      "location_summary": "Decatur, Georgia • Fairfax, Virginia • Milwaukee, Wisconsin",
      "locations": [
        {
          "city": "Decatur",
          "state": "Georgia"
        },
        {
          "city": "Fairfax",
          "state": "Virginia"
        },
        {
          "city": "Milwaukee",
          "state": "Wisconsin"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00230607"
    },
    {
      "nct_id": "NCT00694109",
      "title": "An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "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": 144,
      "start_date": "2008-04",
      "completion_date": "2014-09",
      "has_results": true,
      "last_update_posted_date": "2016-09-09",
      "last_synced_at": "2026-10-14T10:49:56.933Z",
      "location_count": 20,
      "location_summary": "Mission Viejo, California • Newport Beach, California • Bridgeport, Connecticut + 17 more",
      "locations": [
        {
          "city": "Mission Viejo",
          "state": "California"
        },
        {
          "city": "Newport Beach",
          "state": "California"
        },
        {
          "city": "Bridgeport",
          "state": "Connecticut"
        },
        {
          "city": "Melbourne",
          "state": "Florida"
        },
        {
          "city": "Winter Park",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00694109"
    },
    {
      "nct_id": "NCT00106912",
      "title": "Study to Collect Data on Fabry Disease Patients With Enhanceable Alpha-Galactosidase A Activity",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "National Institute of Neurological Disorders and Stroke (NINDS)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "MALE",
        "summary": "18 Years and older · Male only"
      },
      "enrollment_count": 35,
      "start_date": "2005-03-28",
      "completion_date": "2008-03-27",
      "has_results": false,
      "last_update_posted_date": "2017-07-02",
      "last_synced_at": "2026-10-14T10:49:56.933Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00106912"
    },
    {
      "nct_id": "NCT05682378",
      "title": "Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20, or ORION-19 Studies",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Heterozygous or Homozygous Familial Hypercholesterolemia"
      ],
      "interventions": [
        {
          "name": "Inclisiran",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Novartis Pharmaceuticals",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": "100 Years",
        "sex": "ALL",
        "summary": "2 Years to 100 Years"
      },
      "enrollment_count": 195,
      "start_date": "2023-02-10",
      "completion_date": "2032-03-28",
      "has_results": false,
      "last_update_posted_date": "2026-09-18",
      "last_synced_at": "2026-10-14T10:49:56.933Z",
      "location_count": 5,
      "location_summary": "Boca Raton, Florida • New York, New York • Cincinnati, Ohio + 2 more",
      "locations": [
        {
          "city": "Boca Raton",
          "state": "Florida"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Cincinnati",
          "state": "Ohio"
        },
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        },
        {
          "city": "Salt Lake City",
          "state": "Utah"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05682378"
    },
    {
      "nct_id": "NCT03737214",
      "title": "A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [
        {
          "name": "Lucerastat",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Idorsia Pharmaceuticals Ltd.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 107,
      "start_date": "2018-12-18",
      "completion_date": "2029-11",
      "has_results": false,
      "last_update_posted_date": "2026-08-31",
      "last_synced_at": "2026-10-14T10:49:56.933Z",
      "location_count": 14,
      "location_summary": "Birmingham, Alabama • Irvine, California • Oakland, California + 10 more",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Irvine",
          "state": "California"
        },
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Gainesville",
          "state": "Florida"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03737214"
    }
  ]
}