{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Metabolism%2C+Inborn+Errors",
    "query": {
      "condition": "Metabolism, Inborn Errors"
    },
    "page_size": 10
  },
  "pagination": {
    "page": 1,
    "page_size": 10,
    "total_count": 1594,
    "total_pages": 160,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Metabolism%2C+Inborn+Errors&page=2&page_size=10",
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-07-22T14:18:56.193Z",
  "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": "NCT01597596",
      "title": "A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE4"
      ],
      "conditions": [
        "Pompe Disease (Infantile-Onset)",
        "Glycogen Storage Disease Type II (GSD II)",
        "Glycogenosis 2",
        "Acid Maltase Deficiency"
      ],
      "interventions": [
        {
          "name": "alglucosidase alfa",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Genzyme, a Sanofi Company",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "12 Months",
        "sex": "ALL",
        "summary": "Up to 12 Months"
      },
      "enrollment_count": 4,
      "start_date": "2012-08",
      "completion_date": "2014-12",
      "has_results": true,
      "last_update_posted_date": "2016-01-18",
      "last_synced_at": "2026-07-22T14:18:56.193Z",
      "location_count": 14,
      "location_summary": "Little Rock, Arkansas • Oakland, California • Gainsville, Florida + 11 more",
      "locations": [
        {
          "city": "Little Rock",
          "state": "Arkansas"
        },
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Gainsville",
          "state": "Florida"
        },
        {
          "city": "Decatur",
          "state": "Georgia"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01597596"
    },
    {
      "nct_id": "NCT05972629",
      "title": "A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Phenylketonuria"
      ],
      "interventions": [
        {
          "name": "SAR444836",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Sanofi",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "65 Years",
        "sex": "ALL",
        "summary": "18 Years to 65 Years"
      },
      "enrollment_count": 32,
      "start_date": "2023-08-07",
      "completion_date": "2030-04-02",
      "has_results": false,
      "last_update_posted_date": "2025-09-23",
      "last_synced_at": "2026-07-22T14:18:56.193Z",
      "location_count": 7,
      "location_summary": "San Francisco, California • Aurora, Colorado • Gainesville, Florida + 4 more",
      "locations": [
        {
          "city": "San Francisco",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Gainesville",
          "state": "Florida"
        },
        {
          "city": "Cleveland",
          "state": "Ohio"
        },
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05972629"
    },
    {
      "nct_id": "NCT07280156",
      "title": "A Study to Investigate the Clinical Effect and the Safety of PRX-115 Infused Intravenously at Different Dosing Regimens, With and Without Methotrexate, Versus Placebo in Adults Gout Patients (RELEASE)",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Gout"
      ],
      "interventions": [
        {
          "name": "PRX-115",
          "type": "BIOLOGICAL"
        },
        {
          "name": "Methotrexate (MTX)",
          "type": "DRUG"
        },
        {
          "name": "PRX-115 placebo",
          "type": "OTHER"
        },
        {
          "name": "Placebo-Methotrexate",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL",
        "DRUG",
        "OTHER"
      ],
      "sponsor": "Protalix",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 150,
      "start_date": "2025-12-22",
      "completion_date": "2028-06",
      "has_results": false,
      "last_update_posted_date": "2026-06-11",
      "last_synced_at": "2026-07-22T14:18:56.193Z",
      "location_count": 18,
      "location_summary": "Little Rock, Arkansas • El Cajon, California • Clearwater, Florida + 12 more",
      "locations": [
        {
          "city": "Little Rock",
          "state": "Arkansas"
        },
        {
          "city": "El Cajon",
          "state": "California"
        },
        {
          "city": "Clearwater",
          "state": "Florida"
        },
        {
          "city": "DeLand",
          "state": "Florida"
        },
        {
          "city": "Doral",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07280156"
    },
    {
      "nct_id": "NCT02356653",
      "title": "Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "EARLY_PHASE1"
      ],
      "conditions": [
        "Leukemia",
        "Inborn Errors of Metabolism",
        "Bone Marrow Failure Syndromes",
        "Immunodeficiencies",
        "Immunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome"
      ],
      "interventions": [
        {
          "name": "Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs)",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Children's Hospital of Philadelphia",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "30 Years",
        "sex": "ALL",
        "summary": "Up to 30 Years"
      },
      "enrollment_count": 100,
      "start_date": "2013-12",
      "completion_date": "2030-01",
      "has_results": false,
      "last_update_posted_date": "2026-02-20",
      "last_synced_at": "2026-07-22T14:18:56.193Z",
      "location_count": 1,
      "location_summary": "Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02356653"
    },
    {
      "nct_id": "NCT00446550",
      "title": "A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Gaucher Disease",
        "Type 1 Gaucher Disease",
        "Gaucher Disease, Type 1"
      ],
      "interventions": [
        {
          "name": "afegostat tartrate",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Amicus Therapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "74 Years",
        "sex": "ALL",
        "summary": "18 Years to 74 Years"
      },
      "enrollment_count": 19,
      "start_date": "2008-06-11",
      "completion_date": "2009-08-20",
      "has_results": true,
      "last_update_posted_date": "2018-08-15",
      "last_synced_at": "2026-07-22T14:18:56.193Z",
      "location_count": 7,
      "location_summary": "Beverly Hills, California • Coral Springs, Florida • Decatur, Georgia + 4 more",
      "locations": [
        {
          "city": "Beverly Hills",
          "state": "California"
        },
        {
          "city": "Coral Springs",
          "state": "Florida"
        },
        {
          "city": "Decatur",
          "state": "Georgia"
        },
        {
          "city": "Iowa City",
          "state": "Iowa"
        },
        {
          "city": "Kansas City",
          "state": "Kansas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00446550"
    },
    {
      "nct_id": "NCT00027274",
      "title": "Cancer in Inherited Bone Marrow Failure Syndromes",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Diamond Blackfan Anemia",
        "Dyskeratosis Congenita",
        "Fanconi Anemia",
        "Shwachman Diamond Syndrome",
        "Inherited Bone Marrow Failure Syndrome, Aplastic Anemia"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "National Cancer Institute (NCI)",
      "sponsor_class": "NIH",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "1 Day",
        "maximum_age": "100 Years",
        "sex": "ALL",
        "summary": "1 Day to 100 Years"
      },
      "enrollment_count": 4000,
      "start_date": "2001-11-28",
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2026-07-08",
      "last_synced_at": "2026-07-22T14:18:56.193Z",
      "location_count": 2,
      "location_summary": "Bethesda, Maryland • Rockville, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        },
        {
          "city": "Rockville",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00027274"
    },
    {
      "nct_id": "NCT02654886",
      "title": "Safety and Effectiveness of Resistance Exercise Training in Patients With Pompe Disease.",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "NA"
      ],
      "conditions": [
        "Pompe Disease",
        "Muscle Weakness"
      ],
      "interventions": [
        {
          "name": "Resistance Exercise Training",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "University of California, Irvine",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "16 Years",
        "maximum_age": "75 Years",
        "sex": "ALL",
        "summary": "16 Years to 75 Years"
      },
      "enrollment_count": 10,
      "start_date": "2015-10",
      "completion_date": "2019-09-24",
      "has_results": false,
      "last_update_posted_date": "2019-09-26",
      "last_synced_at": "2026-07-22T14:18:56.193Z",
      "location_count": 1,
      "location_summary": "Irvine, California",
      "locations": [
        {
          "city": "Irvine",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02654886"
    },
    {
      "nct_id": "NCT02393560",
      "title": "DECT Study in Allopurinol-Treated Gout Patients",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Gout"
      ],
      "interventions": [
        {
          "name": "DECT scan",
          "type": "PROCEDURE"
        }
      ],
      "intervention_types": [
        "PROCEDURE"
      ],
      "sponsor": "Ardea Biosciences, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "85 Years",
        "sex": "ALL",
        "summary": "18 Years to 85 Years"
      },
      "enrollment_count": 223,
      "start_date": "2015-04",
      "completion_date": "2016-10",
      "has_results": false,
      "last_update_posted_date": "2017-01-25",
      "last_synced_at": "2026-07-22T14:18:56.193Z",
      "location_count": 9,
      "location_summary": "Irvine, California • Englewood, Colorado • Wheat Ridge, Colorado + 6 more",
      "locations": [
        {
          "city": "Irvine",
          "state": "California"
        },
        {
          "city": "Englewood",
          "state": "Colorado"
        },
        {
          "city": "Wheat Ridge",
          "state": "Colorado"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Cleveland",
          "state": "Ohio"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02393560"
    },
    {
      "nct_id": "NCT04848779",
      "title": "A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Glycogen Storage Disease Type II"
      ],
      "interventions": [
        {
          "name": "Alglucosidase alfa GZ419829",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Sanofi",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "0 Days",
        "maximum_age": "6 Months",
        "sex": "ALL",
        "summary": "0 Days to 6 Months"
      },
      "enrollment_count": 16,
      "start_date": "2021-06-10",
      "completion_date": "2026-10-28",
      "has_results": false,
      "last_update_posted_date": "2026-06-24",
      "last_synced_at": "2026-07-22T14:18:56.193Z",
      "location_count": 5,
      "location_summary": "Hawthorne, New York • Durham, North Carolina • Cincinnati, Ohio + 2 more",
      "locations": [
        {
          "city": "Hawthorne",
          "state": "New York"
        },
        {
          "city": "Durham",
          "state": "North Carolina"
        },
        {
          "city": "Cincinnati",
          "state": "Ohio"
        },
        {
          "city": "Memphis",
          "state": "Tennessee"
        },
        {
          "city": "Seattle",
          "state": "Washington"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04848779"
    },
    {
      "nct_id": "NCT06488261",
      "title": "Long Term Follow-Up for Safety of AVR-RD-02",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Gaucher Disease"
      ],
      "interventions": [
        {
          "name": "AVR-RD-02",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "John Bernat",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 2,
      "start_date": "2024-02-21",
      "completion_date": "2037-10",
      "has_results": false,
      "last_update_posted_date": "2025-07-22",
      "last_synced_at": "2026-07-22T14:18:56.193Z",
      "location_count": 1,
      "location_summary": "Iowa City, Iowa",
      "locations": [
        {
          "city": "Iowa City",
          "state": "Iowa"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06488261"
    }
  ]
}