{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=51",
    "query": {
      "condition": "Inborn Errors of Metabolism Disorders",
      "page": 51
    },
    "page_size": 10
  },
  "pagination": {
    "page": 51,
    "page_size": 10,
    "total_count": 1513,
    "total_pages": 152,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=52&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=50&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-11-15T14:46:03.686Z",
  "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": "NCT01869270",
      "title": "Gene Therapy for Tay-Sachs Disease",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Tay Sachs Disease",
        "Sandhoff Disease",
        "Late Onset Tay Sachs Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "University of Minnesota",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 4,
      "start_date": "2010-12",
      "completion_date": "2014-08",
      "has_results": false,
      "last_update_posted_date": "2014-12-04",
      "last_synced_at": "2026-11-15T14:46:03.686Z",
      "location_count": 2,
      "location_summary": "Tampa, Florida • Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Tampa",
          "state": "Florida"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01869270"
    },
    {
      "nct_id": "NCT06614569",
      "title": "Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "GM2 Gangliosidosis",
        "Tay Sachs Disease",
        "Sandhoff Disease"
      ],
      "interventions": [
        {
          "name": "AXO-AAV-GM2",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Terence Flotte",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 7,
      "start_date": "2024-09-17",
      "completion_date": "2028-12",
      "has_results": false,
      "last_update_posted_date": "2025-10-16",
      "last_synced_at": "2026-11-15T14:46:03.686Z",
      "location_count": 1,
      "location_summary": "Worcester, Massachusetts",
      "locations": [
        {
          "city": "Worcester",
          "state": "Massachusetts"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06614569"
    },
    {
      "nct_id": "NCT01735617",
      "title": "Pilot Study to Characterize and Examine the Pharmacokinetics and Efficacy of Chronocort® in Adults With CAH",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Endocrine Disease",
        "Adrenal Insufficiency",
        "Congenital Adrenal Hyperplasia"
      ],
      "interventions": [
        {
          "name": "Hydrocortisone Modified Release Capsules",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Neurocrine UK Limited",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 16,
      "start_date": "2012-12",
      "completion_date": "2013-12",
      "has_results": true,
      "last_update_posted_date": "2026-10-02",
      "last_synced_at": "2026-11-15T14:46:03.686Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01735617"
    },
    {
      "nct_id": "NCT04020055",
      "title": "A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "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": 14,
      "start_date": "2022-10-31",
      "completion_date": "2026-12-31",
      "has_results": false,
      "last_update_posted_date": "2026-07-06",
      "last_synced_at": "2026-11-15T14:46:03.686Z",
      "location_count": 3,
      "location_summary": "Atlanta, Georgia • Cleveland, Ohio • Fairfax, Virginia",
      "locations": [
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Cleveland",
          "state": "Ohio"
        },
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04020055"
    },
    {
      "nct_id": "NCT05843552",
      "title": "Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Gaucher Disease"
      ],
      "interventions": [
        {
          "name": "no intervention",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "University of Minnesota",
      "sponsor_class": "OTHER",
      "healthy_volunteers": null,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "80 Years",
        "sex": "ALL",
        "summary": "18 Years to 80 Years"
      },
      "enrollment_count": 30,
      "start_date": "2023-08-14",
      "completion_date": "2026-12-31",
      "has_results": false,
      "last_update_posted_date": "2026-04-06",
      "last_synced_at": "2026-11-15T14:46:03.686Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05843552"
    },
    {
      "nct_id": "NCT01129648",
      "title": "Study to Evaluate sUA-Lowering Activity, Safety & PK Interaction of Oral BCX4208 & Allopurinol Admin. in Subjects w/Gout",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Gout"
      ],
      "interventions": [
        {
          "name": "Placebo",
          "type": "DRUG"
        },
        {
          "name": "Allopurinol",
          "type": "DRUG"
        },
        {
          "name": "BCX4208",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "BioCryst Pharmaceuticals",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "69 Years",
        "sex": "ALL",
        "summary": "18 Years to 69 Years"
      },
      "enrollment_count": 87,
      "start_date": "2010-05",
      "completion_date": "2010-11",
      "has_results": false,
      "last_update_posted_date": "2012-01-23",
      "last_synced_at": "2026-11-15T14:46:03.686Z",
      "location_count": 18,
      "location_summary": "Scottsdale, Arizona • Irvine, California • South Miami, Florida + 15 more",
      "locations": [
        {
          "city": "Scottsdale",
          "state": "Arizona"
        },
        {
          "city": "Irvine",
          "state": "California"
        },
        {
          "city": "South Miami",
          "state": "Florida"
        },
        {
          "city": "Honolulu",
          "state": "Hawaii"
        },
        {
          "city": "Boise",
          "state": "Idaho"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01129648"
    },
    {
      "nct_id": "NCT00782548",
      "title": "Comparative Study to Evaluate KADIAN and Avinza in Healthy Adults",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE4"
      ],
      "conditions": [
        "Healthy"
      ],
      "interventions": [
        {
          "name": "KADIAN (morphine sulfate sustained-release) capsules",
          "type": "DRUG"
        },
        {
          "name": "Avinza (morphine sulfate sustained-release) capsules",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Actavis Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "19 Years",
        "maximum_age": "40 Years",
        "sex": "ALL",
        "summary": "19 Years to 40 Years"
      },
      "enrollment_count": 40,
      "start_date": "2004-05",
      "completion_date": "2004-06",
      "has_results": false,
      "last_update_posted_date": "2020-07-16",
      "last_synced_at": "2026-11-15T14:46:03.686Z",
      "location_count": 1,
      "location_summary": "Lincoln, Nebraska",
      "locations": [
        {
          "city": "Lincoln",
          "state": "Nebraska"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00782548"
    },
    {
      "nct_id": "NCT05579548",
      "title": "A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Phenylketonuria, Maternal"
      ],
      "interventions": [
        {
          "name": "Pegvaliase",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "BioMarin Pharmaceutical",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": null,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "FEMALE",
        "summary": "Female only"
      },
      "enrollment_count": 50,
      "start_date": "2022-11-22",
      "completion_date": "2032-10-04",
      "has_results": false,
      "last_update_posted_date": "2026-03-24",
      "last_synced_at": "2026-11-15T14:46:03.686Z",
      "location_count": 1,
      "location_summary": "Wilmington, North Carolina",
      "locations": [
        {
          "city": "Wilmington",
          "state": "North Carolina"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05579548"
    },
    {
      "nct_id": "NCT02120235",
      "title": "Investigating Lysosomal Storage Diseases in Minority Groups",
      "overall_status": "UNKNOWN",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Lysosomal Storage Disorders",
        "Gaucher Disease",
        "Fabry Disease",
        "Pompe Disease",
        "Niemann-Pick Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "O & O Alpan LLC",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "1 Day",
        "maximum_age": "100 Years",
        "sex": "ALL",
        "summary": "1 Day to 100 Years"
      },
      "enrollment_count": 20000,
      "start_date": "2014-02",
      "completion_date": "2018-12",
      "has_results": false,
      "last_update_posted_date": "2017-04-04",
      "last_synced_at": "2026-11-15T14:46:03.686Z",
      "location_count": 1,
      "location_summary": "Fairfax, Virginia",
      "locations": [
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02120235"
    },
    {
      "nct_id": "NCT07667387",
      "title": "Study of LNP.UCD.ABE in Patients With Urea Cycle Disorders",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Urea Cycle Disorders",
        "Carbamoyl-Phosphate Synthase I Deficiency"
      ],
      "interventions": [
        {
          "name": "LNP.UCD.ABE",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Rebecca Ahrens-Nicklas",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "24 Hours",
        "maximum_age": "5 Years",
        "sex": "ALL",
        "summary": "24 Hours to 5 Years"
      },
      "enrollment_count": 7,
      "start_date": "2026-08-07",
      "completion_date": "2028-09",
      "has_results": false,
      "last_update_posted_date": "2026-08-26",
      "last_synced_at": "2026-11-15T14:46:03.686Z",
      "location_count": 1,
      "location_summary": "Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07667387"
    }
  ]
}