{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Lipid+Metabolism%2C+Inborn+Errors",
    "query": {
      "condition": "Lipid Metabolism, Inborn Errors"
    },
    "page_size": 10
  },
  "pagination": {
    "page": 1,
    "page_size": 10,
    "total_count": 497,
    "total_pages": 50,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Lipid+Metabolism%2C+Inborn+Errors&page=2&page_size=10",
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-05-22T08:14:07.563Z",
  "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": "NCT00001972",
      "title": "PET Scan of Brain Metabolism in Relation to Age and Disease",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Alzheimer's Disease",
        "Brain Neoplasm",
        "Niemann Pick Disease"
      ],
      "interventions": [
        {
          "name": "15 O Water",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "National Institute of Neurological Disorders and Stroke (NINDS)",
      "sponsor_class": "NIH",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 123,
      "start_date": "1994-09",
      "completion_date": "2002-08",
      "has_results": false,
      "last_update_posted_date": "2008-03-04",
      "last_synced_at": "2026-05-22T08:14:07.563Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00001972"
    },
    {
      "nct_id": "NCT00343577",
      "title": "Antiproteinuric Agents and Fabry Disease",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Fabry Disease",
        "Proteinuria"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "University of Alabama at Birmingham",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "14 Years",
        "maximum_age": "95 Years",
        "sex": "ALL",
        "summary": "14 Years to 95 Years"
      },
      "enrollment_count": 12,
      "start_date": "2001-01",
      "completion_date": "2006-12",
      "has_results": false,
      "last_update_posted_date": "2013-11-19",
      "last_synced_at": "2026-05-22T08:14:07.563Z",
      "location_count": 1,
      "location_summary": "Birmingham, Alabama",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00343577"
    },
    {
      "nct_id": "NCT04221451",
      "title": "A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Tay-Sachs Disease",
        "Sandhoff Disease"
      ],
      "interventions": [
        {
          "name": "venglustat GZ402671",
          "type": "DRUG"
        },
        {
          "name": "placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Genzyme, a Sanofi Company",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "2 Years and older"
      },
      "enrollment_count": 75,
      "start_date": "2020-06-29",
      "completion_date": "2024-12-26",
      "has_results": true,
      "last_update_posted_date": "2026-01-28",
      "last_synced_at": "2026-05-22T08:14:07.563Z",
      "location_count": 5,
      "location_summary": "Los Angeles, California • Atlanta, Georgia • Bethesda, Maryland + 2 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Bethesda",
          "state": "Maryland"
        },
        {
          "city": "Boston",
          "state": "Massachusetts"
        },
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04221451"
    },
    {
      "nct_id": "NCT04721366",
      "title": "A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Gaucher Disease"
      ],
      "interventions": [
        {
          "name": "Standard of Care",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "Takeda",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "5 Years",
        "sex": "ALL",
        "summary": "Up to 5 Years"
      },
      "enrollment_count": 11,
      "start_date": "2021-01-08",
      "completion_date": "2023-04-17",
      "has_results": false,
      "last_update_posted_date": "2024-03-29",
      "last_synced_at": "2026-05-22T08:14:07.563Z",
      "location_count": 1,
      "location_summary": "Fairfax, Virginia",
      "locations": [
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04721366"
    },
    {
      "nct_id": "NCT02574286",
      "title": "Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE4"
      ],
      "conditions": [
        "Gaucher Disease"
      ],
      "interventions": [
        {
          "name": "Velaglucerase alfa",
          "type": "DRUG"
        },
        {
          "name": "Vitamin D",
          "type": "DIETARY_SUPPLEMENT"
        }
      ],
      "intervention_types": [
        "DRUG",
        "DIETARY_SUPPLEMENT"
      ],
      "sponsor": "Shire",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "70 Years",
        "sex": "ALL",
        "summary": "18 Years to 70 Years"
      },
      "enrollment_count": 21,
      "start_date": "2016-06-29",
      "completion_date": "2020-11-30",
      "has_results": true,
      "last_update_posted_date": "2022-02-01",
      "last_synced_at": "2026-05-22T08:14:07.563Z",
      "location_count": 8,
      "location_summary": "Beverly Hills, California • Los Angeles, California • Atlanta, Georgia + 5 more",
      "locations": [
        {
          "city": "Beverly Hills",
          "state": "California"
        },
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02574286"
    },
    {
      "nct_id": "NCT06539169",
      "title": "FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Alpha-Thalassemia",
        "Beta-Thalassemia",
        "Amyloidosis",
        "Amyotrophic Lateral Sclerosis",
        "Creutzfeld-Jakob Disease",
        "Cystic Fibrosis",
        "Duchenne Muscular Dystrophy",
        "Early-Onset Alzheimer Disease",
        "Ehlers-Danlos Syndrome",
        "Huntington Disease",
        "Gaucher Disease",
        "GM1 Gangliosidosis",
        "Myasthenia Gravis",
        "Pompe Disease",
        "Sickle Cell Disease",
        "Transthyretin Amyloid Cardiomyopathy",
        "Rare Diseases"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "xCures",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 1000,
      "start_date": "2024-06-10",
      "completion_date": "2026-06-10",
      "has_results": false,
      "last_update_posted_date": "2024-11-14",
      "last_synced_at": "2026-05-22T08:14:07.563Z",
      "location_count": 1,
      "location_summary": "Los Altos, California",
      "locations": [
        {
          "city": "Los Altos",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06539169"
    },
    {
      "nct_id": "NCT04552691",
      "title": "Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients",
      "overall_status": "APPROVED_FOR_MARKETING",
      "study_type": "EXPANDED_ACCESS",
      "phases": [],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [
        {
          "name": "Pegunigalsidase Alfa",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Chiesi Farmaceutici S.p.A.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": null,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": null,
      "start_date": null,
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2024-07-31",
      "last_synced_at": "2026-05-22T08:14:07.563Z",
      "location_count": 11,
      "location_summary": "Birmingham, Alabama • Phoenix, Arizona • Orange, California + 8 more",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Phoenix",
          "state": "Arizona"
        },
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "Salinas",
          "state": "California"
        },
        {
          "city": "Jacksonville",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04552691"
    },
    {
      "nct_id": "NCT02325674",
      "title": "MEASuRE: Metreleptin Effectiveness And Safety Registry",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Generalised Lipodystrophy",
        "Partial Lipodystrophy"
      ],
      "interventions": [
        {
          "name": "Metreleptin",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Amryt Pharma",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 100,
      "start_date": "2016-10-11",
      "completion_date": "2031-10-31",
      "has_results": false,
      "last_update_posted_date": "2026-01-20",
      "last_synced_at": "2026-05-22T08:14:07.563Z",
      "location_count": 16,
      "location_summary": "Birmingham, Alabama • Duarte, California • Atlanta, Georgia + 11 more",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Duarte",
          "state": "California"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "Kansas City",
          "state": "Kansas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02325674"
    },
    {
      "nct_id": "NCT04118348",
      "title": "Evaluating the Efficacy of Pediatric Lipid Screening Alerts",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "NA"
      ],
      "conditions": [
        "Hypercholesterolemia",
        "Hypercholesterolemia, Familial",
        "Hypercholesteremia in Children",
        "Hyperlipidemia in Children"
      ],
      "interventions": [
        {
          "name": "Best Practice Alert",
          "type": "BEHAVIORAL"
        },
        {
          "name": "Health Maintenance Topic",
          "type": "BEHAVIORAL"
        }
      ],
      "intervention_types": [
        "BEHAVIORAL"
      ],
      "sponsor": "Geisinger Clinic",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "9 Years",
        "maximum_age": "11 Years",
        "sex": "ALL",
        "summary": "9 Years to 11 Years"
      },
      "enrollment_count": 13340,
      "start_date": "2019-10-10",
      "completion_date": "2020-10-11",
      "has_results": true,
      "last_update_posted_date": "2023-11-07",
      "last_synced_at": "2026-05-22T08:14:07.563Z",
      "location_count": 1,
      "location_summary": "Danville, Pennsylvania",
      "locations": [
        {
          "city": "Danville",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04118348"
    },
    {
      "nct_id": "NCT00730314",
      "title": "Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Sickle Cell Disease",
        "Thalassemia",
        "Anemia",
        "Granuloma",
        "Wiskott-Aldrich Syndrome",
        "Chediak Higashi Syndrome",
        "Osteopetrosis",
        "Neutropenia",
        "Thrombocytopenia",
        "Hurler Disease",
        "Niemann-Pick Disease",
        "Fucosidosis"
      ],
      "interventions": [
        {
          "name": "Hematopoietic stem cell transplantation",
          "type": "PROCEDURE"
        }
      ],
      "intervention_types": [
        "PROCEDURE"
      ],
      "sponsor": "Children's Hospital Los Angeles",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "21 Years",
        "sex": "ALL",
        "summary": "Up to 21 Years"
      },
      "enrollment_count": 25,
      "start_date": "2008-08",
      "completion_date": "2015-08",
      "has_results": false,
      "last_update_posted_date": "2016-06-23",
      "last_synced_at": "2026-05-22T08:14:07.563Z",
      "location_count": 1,
      "location_summary": "Los Angeles, California",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00730314"
    }
  ]
}