{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=39",
    "query": {
      "condition": "Inborn Errors of Metabolism",
      "page": 39
    },
    "page_size": 10
  },
  "pagination": {
    "page": 39,
    "page_size": 10,
    "total_count": 1605,
    "total_pages": 161,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=40&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=38&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-17T21:03:16.235Z",
  "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": "NCT00004493",
      "title": "Phase II Pilot Randomized Study of Sodium Dichloroacetate in Patients With Congenital Lactic Acidemia",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Lactic Acidosis"
      ],
      "interventions": [
        {
          "name": "sodium dichloroacetate",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "University of California, San Diego",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "3 Months",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "3 Months and older"
      },
      "enrollment_count": 75,
      "start_date": "1998-09",
      "completion_date": "2003-09",
      "has_results": false,
      "last_update_posted_date": "2015-03-25",
      "last_synced_at": "2026-10-17T21:03:16.235Z",
      "location_count": 1,
      "location_summary": "La Jolla, California",
      "locations": [
        {
          "city": "La Jolla",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00004493"
    },
    {
      "nct_id": "NCT05139316",
      "title": "A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Glycogen Storage Disease Type IA"
      ],
      "interventions": [
        {
          "name": "DTX401",
          "type": "GENETIC"
        },
        {
          "name": "Placebo",
          "type": "OTHER"
        },
        {
          "name": "Oral prednisolone",
          "type": "DRUG"
        },
        {
          "name": "Placebo for oral prednisolone",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "GENETIC",
        "OTHER",
        "DRUG"
      ],
      "sponsor": "Ultragenyx Pharmaceutical Inc",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "8 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "8 Years and older"
      },
      "enrollment_count": 52,
      "start_date": "2021-11-08",
      "completion_date": "2026-02-20",
      "has_results": true,
      "last_update_posted_date": "2026-10-14",
      "last_synced_at": "2026-10-17T21:03:16.235Z",
      "location_count": 9,
      "location_summary": "Orange, California • Aurora, Colorado • Farmington, Connecticut + 6 more",
      "locations": [
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Farmington",
          "state": "Connecticut"
        },
        {
          "city": "The Bronx",
          "state": "New York"
        },
        {
          "city": "Durham",
          "state": "North Carolina"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05139316"
    },
    {
      "nct_id": "NCT01001338",
      "title": "Allopurinol Combination Study",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Gout"
      ],
      "interventions": [
        {
          "name": "RDEA594",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        },
        {
          "name": "Allopurinol",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Ardea Biosciences, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "80 Years",
        "sex": "ALL",
        "summary": "18 Years to 80 Years"
      },
      "enrollment_count": 227,
      "start_date": "2009-10",
      "completion_date": "2016-08",
      "has_results": false,
      "last_update_posted_date": "2017-01-24",
      "last_synced_at": "2026-10-17T21:03:16.235Z",
      "location_count": 25,
      "location_summary": "Phoenix, Arizona • La Jolla, California • Los Angeles, California + 22 more",
      "locations": [
        {
          "city": "Phoenix",
          "state": "Arizona"
        },
        {
          "city": "La Jolla",
          "state": "California"
        },
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Stanford",
          "state": "California"
        },
        {
          "city": "Boca Raton",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01001338"
    },
    {
      "nct_id": "NCT07054515",
      "title": "A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Niemann-Pick Type C Disease",
        "GM1 Gangliosidosis",
        "GM2 Gangliosidosis"
      ],
      "interventions": [
        {
          "name": "AZ-3102",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Azafaros B.V.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "4 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "4 Years and older"
      },
      "enrollment_count": 147,
      "start_date": "2025-06-30",
      "completion_date": "2028-06-14",
      "has_results": false,
      "last_update_posted_date": "2026-09-24",
      "last_synced_at": "2026-10-17T21:03:16.235Z",
      "location_count": 5,
      "location_summary": "Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        },
        {
          "city": "Rochester",
          "state": "Minnesota"
        },
        {
          "city": "Dallas",
          "state": "Texas"
        },
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07054515"
    },
    {
      "nct_id": "NCT01633489",
      "title": "Lysosomal Acid Lipase (LAL) Deficiency Registry",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Lysosomal Acid Lipase Deficiency",
        "Cholesterol Ester Storage Disease",
        "Wolman Disease",
        "Acid Cholesteryl Ester Hydrolase Deficiency, Type 2",
        "Acid Lipase Deficiency",
        "LIPA Deficiency",
        "LAL-Deficiency"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Alexion Pharmaceuticals, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 300,
      "start_date": "2013-05-30",
      "completion_date": "2029-08-30",
      "has_results": false,
      "last_update_posted_date": "2026-03-06",
      "last_synced_at": "2026-10-17T21:03:16.235Z",
      "location_count": 19,
      "location_summary": "Phoenix, Arizona • Stanford, California • Miramar, Florida + 14 more",
      "locations": [
        {
          "city": "Phoenix",
          "state": "Arizona"
        },
        {
          "city": "Stanford",
          "state": "California"
        },
        {
          "city": "Miramar",
          "state": "Florida"
        },
        {
          "city": "Orlando",
          "state": "Florida"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01633489"
    },
    {
      "nct_id": "NCT01994889",
      "title": "Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Transthyretin (TTR) Amyloid Cardiomyopathy"
      ],
      "interventions": [
        {
          "name": "Tafamidis",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Pfizer",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "90 Years",
        "sex": "ALL",
        "summary": "18 Years to 90 Years"
      },
      "enrollment_count": 441,
      "start_date": "2013-12-09",
      "completion_date": "2018-02-07",
      "has_results": true,
      "last_update_posted_date": "2019-04-24",
      "last_synced_at": "2026-10-17T21:03:16.235Z",
      "location_count": 50,
      "location_summary": "Birmingham, Alabama • Phoenix, Arizona • Scottsdale, Arizona + 24 more",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Phoenix",
          "state": "Arizona"
        },
        {
          "city": "Phoenix",
          "state": "Arizona"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01994889"
    },
    {
      "nct_id": "NCT00074516",
      "title": "Kidney Transplantation in Patients With Cystinosis",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Cystinosis"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "7 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "7 Years and older"
      },
      "enrollment_count": 11,
      "start_date": "2003-12-10",
      "completion_date": "2014-06-11",
      "has_results": false,
      "last_update_posted_date": "2017-10-06",
      "last_synced_at": "2026-10-17T21:03:16.235Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00074516"
    },
    {
      "nct_id": "NCT05368038",
      "title": "ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program",
      "overall_status": "ENROLLING_BY_INVITATION",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Acid Sphingomyelinase Deficiency",
        "Ceroid Lipofuscinosis, Neuronal, 2",
        "Cerebrotendinous Xanthomatosis",
        "Fabry Disease",
        "GM1 Gangliosidosis",
        "Gaucher Disease",
        "Lysosomal Acid Lipase Deficiency",
        "Metachromatic Leukodystrophy",
        "Mucopolysaccharidosis II",
        "Mucopolysaccharidosis III-B",
        "Mucopolysaccharidosis IV A",
        "Mucopolysaccharidosis VI",
        "Mucopolysaccharidosis VII",
        "Niemann-Pick Disease, Type C"
      ],
      "interventions": [
        {
          "name": "Confirmatory Testing",
          "type": "DIAGNOSTIC_TEST"
        }
      ],
      "intervention_types": [
        "DIAGNOSTIC_TEST"
      ],
      "sponsor": "Albert Einstein College of Medicine",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "4 Weeks",
        "sex": "ALL",
        "summary": "Up to 4 Weeks"
      },
      "enrollment_count": 100000,
      "start_date": "2021-05-10",
      "completion_date": "2029-08-31",
      "has_results": false,
      "last_update_posted_date": "2026-07-28",
      "last_synced_at": "2026-10-17T21:03:16.235Z",
      "location_count": 9,
      "location_summary": "Brooklyn, New York • Manhasset, New York • New York, New York + 2 more",
      "locations": [
        {
          "city": "Brooklyn",
          "state": "New York"
        },
        {
          "city": "Brooklyn",
          "state": "New York"
        },
        {
          "city": "Manhasset",
          "state": "New York"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05368038"
    },
    {
      "nct_id": "NCT02843035",
      "title": "Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Gaucher Disease Type 1",
        "Gaucher Disease Type 3"
      ],
      "interventions": [
        {
          "name": "venglustat (GZ402671)",
          "type": "DRUG"
        },
        {
          "name": "imiglucerase",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Genzyme, a Sanofi Company",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 12,
      "start_date": "2017-01-04",
      "completion_date": "2026-10-30",
      "has_results": false,
      "last_update_posted_date": "2026-07-10",
      "last_synced_at": "2026-10-17T21:03:16.235Z",
      "location_count": 3,
      "location_summary": "New Haven, Connecticut • Dallas, Texas • Fairfax, Virginia",
      "locations": [
        {
          "city": "New Haven",
          "state": "Connecticut"
        },
        {
          "city": "Dallas",
          "state": "Texas"
        },
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02843035"
    },
    {
      "nct_id": "NCT00728676",
      "title": "Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive Behaviors",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Phenylketonuria"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Shoji Yano",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 10,
      "start_date": "2008-08",
      "completion_date": "2010-05",
      "has_results": false,
      "last_update_posted_date": "2013-01-18",
      "last_synced_at": "2026-10-17T21:03:16.235Z",
      "location_count": 1,
      "location_summary": "Los Angeles, California",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00728676"
    }
  ]
}