{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inherited+Metabolic+Disorder",
    "query": {
      "condition": "Inherited Metabolic Disorder"
    },
    "page_size": 10
  },
  "pagination": {
    "page": 1,
    "page_size": 10,
    "total_count": 2382,
    "total_pages": 239,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inherited+Metabolic+Disorder&page=2&page_size=10",
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-07-22T18:49:34.332Z",
  "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": "2026-07-22T18:49:34.332Z",
      "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": "2026-07-22T18:49:34.332Z",
      "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": "NCT00056979",
      "title": "Monoclonal Antibody Conditioning for Allogeneic Stem Cell Transplantation of Patients With Inherited Metabolic Storage Diseases",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Inherited Metabolic Storage Diseases"
      ],
      "interventions": [
        {
          "name": "CAMPATH-1H",
          "type": "DRUG"
        },
        {
          "name": "Anti-CD45",
          "type": "DRUG"
        },
        {
          "name": "FK506",
          "type": "DRUG"
        },
        {
          "name": "Fludarabine",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Baylor College of Medicine",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 2,
      "start_date": "2002-06",
      "completion_date": "2003-06",
      "has_results": false,
      "last_update_posted_date": "2015-09-23",
      "last_synced_at": "2026-07-22T18:49:34.332Z",
      "location_count": 2,
      "location_summary": "Houston, Texas",
      "locations": [
        {
          "city": "Houston",
          "state": "Texas"
        },
        {
          "city": "Houston",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00056979"
    },
    {
      "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": "2026-07-22T18:49:34.332Z",
      "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": "NCT00139451",
      "title": "Nutrients and Hormones: Effects in Boys With Disordered Growth",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Constitutional Growth Delay",
        "Nutrition Disorders",
        "Growth Disorders"
      ],
      "interventions": [
        {
          "name": "Pediasure",
          "type": "DRUG"
        },
        {
          "name": "GH",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Nemours Children's Clinic",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "7 Years",
        "maximum_age": "10 Years",
        "sex": "MALE",
        "summary": "7 Years to 10 Years · Male only"
      },
      "enrollment_count": 20,
      "start_date": "2004-05",
      "completion_date": "2010-09",
      "has_results": false,
      "last_update_posted_date": "2011-10-12",
      "last_synced_at": "2026-07-22T18:49:34.332Z",
      "location_count": 1,
      "location_summary": "Jacksonville, Florida",
      "locations": [
        {
          "city": "Jacksonville",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00139451"
    },
    {
      "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": "2026-07-22T18:49:34.332Z",
      "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": "2026-07-22T18:49:34.332Z",
      "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": "2026-07-22T18:49:34.332Z",
      "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": "NCT00424242",
      "title": "Pemetrexed Disodium in the Cerebrospinal Fluid of Patients With Leptomeningeal Metastases",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "EARLY_PHASE1"
      ],
      "conditions": [
        "Brain and Central Nervous System Tumors",
        "Chronic Myeloproliferative Disorders",
        "Leukemia",
        "Lymphoma",
        "Lymphoproliferative Disorder",
        "Metastatic Cancer",
        "Multiple Myeloma and Plasma Cell Neoplasm",
        "Myelodysplastic Syndromes",
        "Precancerous Condition",
        "Secondary Myelofibrosis",
        "Unspecified Adult Solid Tumor, Protocol Specific"
      ],
      "interventions": [
        {
          "name": "Pemetrexed",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Northwestern University",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "120 Years",
        "sex": "ALL",
        "summary": "18 Years to 120 Years"
      },
      "enrollment_count": 15,
      "start_date": "2007-01",
      "completion_date": "2011-09",
      "has_results": false,
      "last_update_posted_date": "2021-09-08",
      "last_synced_at": "2026-07-22T18:49:34.332Z",
      "location_count": 1,
      "location_summary": "Chicago, Illinois",
      "locations": [
        {
          "city": "Chicago",
          "state": "Illinois"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00424242"
    },
    {
      "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": "2026-07-22T18:49:34.332Z",
      "location_count": 1,
      "location_summary": "Irvine, California",
      "locations": [
        {
          "city": "Irvine",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02654886"
    }
  ]
}