{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=44",
    "query": {
      "condition": "Inborn Errors of Metabolism",
      "page": 44
    },
    "page_size": 10
  },
  "pagination": {
    "page": 44,
    "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=45&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=43&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-18T13:34:40.962Z",
  "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": "NCT01327807",
      "title": "Cure Cystinosis International Registry",
      "overall_status": "UNKNOWN",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Cystinosis",
        "Nephropathic Cystinosis",
        "Renal Fanconi Syndrome"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Cystinosis Research Foundation",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 750,
      "start_date": "2010-08",
      "completion_date": "2022-12",
      "has_results": false,
      "last_update_posted_date": "2014-03-12",
      "last_synced_at": "2026-10-18T13:34:40.962Z",
      "location_count": 1,
      "location_summary": "San Diego, California",
      "locations": [
        {
          "city": "San Diego",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01327807"
    },
    {
      "nct_id": "NCT07332091",
      "title": "Efficacy and Safety of Vamifeport in Adult Participants With Homeostatic Iron Regulator Gene (HFE)-Related Hereditary Hemochromatosis",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis"
      ],
      "interventions": [
        {
          "name": "Vamifeport",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "CSL Behring",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 84,
      "start_date": "2026-01-22",
      "completion_date": "2028-05-15",
      "has_results": false,
      "last_update_posted_date": "2026-09-16",
      "last_synced_at": "2026-10-18T13:34:40.962Z",
      "location_count": 20,
      "location_summary": "Gilbert, Arizona • San Diego, California • Jacksonville, Florida + 16 more",
      "locations": [
        {
          "city": "Gilbert",
          "state": "Arizona"
        },
        {
          "city": "San Diego",
          "state": "California"
        },
        {
          "city": "San Diego",
          "state": "California"
        },
        {
          "city": "Jacksonville",
          "state": "Florida"
        },
        {
          "city": "Plantation",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07332091"
    },
    {
      "nct_id": "NCT05705674",
      "title": "The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3)",
      "overall_status": "UNKNOWN",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Sanfilippo Syndrome",
        "MPS3"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Lysosomal and Rare Disorders Research and Treatment Center, Inc.",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "5 Years",
        "maximum_age": "99 Years",
        "sex": "ALL",
        "summary": "5 Years to 99 Years"
      },
      "enrollment_count": 6,
      "start_date": "2023-05-01",
      "completion_date": "2024-12",
      "has_results": false,
      "last_update_posted_date": "2024-03-12",
      "last_synced_at": "2026-10-18T13:34:40.962Z",
      "location_count": 1,
      "location_summary": "Fairfax, Virginia",
      "locations": [
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05705674"
    },
    {
      "nct_id": "NCT00081497",
      "title": "A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE4"
      ],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [
        {
          "name": "agalsidase beta",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Genzyme, a Sanofi Company",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "16 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "16 Years and older"
      },
      "enrollment_count": 67,
      "start_date": "2004-01",
      "completion_date": "2005-09",
      "has_results": true,
      "last_update_posted_date": "2015-04-02",
      "last_synced_at": "2026-10-18T13:34:40.962Z",
      "location_count": 18,
      "location_summary": "Birmingham, Alabama • Los Angeles, California • San Francisco, California + 15 more",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "San Francisco",
          "state": "California"
        },
        {
          "city": "West Hartford",
          "state": "Connecticut"
        },
        {
          "city": "Coral Springs",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00081497"
    },
    {
      "nct_id": "NCT01529827",
      "title": "Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Accelerated Phase Chronic Myelogenous Leukemia",
        "Adult Acute Lymphoblastic Leukemia in Remission",
        "Adult Acute Myeloid Leukemia in Remission",
        "Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities",
        "Adult Acute Myeloid Leukemia With Del(5q)",
        "Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)",
        "Adult Acute Myeloid Leukemia With t(15;17)(q22;q12)",
        "Adult Acute Myeloid Leukemia With t(16;16)(p13;q22)",
        "Adult Acute Myeloid Leukemia With t(8;21)(q22;q22)",
        "Adult Grade III Lymphomatoid Granulomatosis",
        "Adult Nasal Type Extranodal NK/T-cell Lymphoma",
        "Anaplastic Large Cell Lymphoma",
        "Angioimmunoblastic T-cell Lymphoma",
        "Aplastic Anemia",
        "Burkitt Lymphoma",
        "Childhood Acute Lymphoblastic Leukemia in Remission",
        "Childhood Acute Myeloid Leukemia in Remission",
        "Childhood Chronic Myelogenous Leukemia",
        "Childhood Diffuse Large Cell Lymphoma",
        "Childhood Grade III Lymphomatoid Granulomatosis",
        "Childhood Immunoblastic Large Cell Lymphoma",
        "Childhood Myelodysplastic Syndromes",
        "Childhood Nasal Type Extranodal NK/T-cell Lymphoma",
        "Chronic Myelomonocytic Leukemia",
        "Chronic Phase Chronic Myelogenous Leukemia",
        "Congenital Amegakaryocytic Thrombocytopenia",
        "Diamond-Blackfan Anemia",
        "Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue",
        "Hepatosplenic T-cell Lymphoma",
        "Juvenile Myelomonocytic Leukemia",
        "Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable",
        "Nodal Marginal Zone B-cell Lymphoma",
        "Paroxysmal Nocturnal Hemoglobinuria",
        "Peripheral T-cell Lymphoma",
        "Polycythemia Vera",
        "Post-transplant Lymphoproliferative Disorder",
        "Previously Treated Myelodysplastic Syndromes",
        "Primary Myelofibrosis",
        "Recurrent Adult Acute Lymphoblastic Leukemia",
        "Recurrent Adult Acute Myeloid Leukemia",
        "Recurrent Adult Burkitt Lymphoma",
        "Recurrent Adult Diffuse Large Cell Lymphoma",
        "Recurrent Adult Diffuse Mixed Cell Lymphoma",
        "Recurrent Adult Diffuse Small Cleaved Cell Lymphoma",
        "Recurrent Adult Grade III Lymphomatoid Granulomatosis",
        "Recurrent Adult Hodgkin Lymphoma",
        "Recurrent Adult Immunoblastic Large Cell Lymphoma",
        "Recurrent Adult Lymphoblastic Lymphoma",
        "Recurrent Adult T-cell Leukemia/Lymphoma",
        "Recurrent Childhood Acute Lymphoblastic Leukemia",
        "Recurrent Childhood Acute Myeloid Leukemia",
        "Recurrent Childhood Anaplastic Large Cell Lymphoma",
        "Recurrent Childhood Grade III Lymphomatoid Granulomatosis",
        "Recurrent Childhood Large Cell Lymphoma",
        "Recurrent Childhood Lymphoblastic Lymphoma",
        "Recurrent Childhood Small Noncleaved Cell Lymphoma",
        "Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma",
        "Recurrent Grade 1 Follicular Lymphoma",
        "Recurrent Grade 2 Follicular Lymphoma",
        "Recurrent Grade 3 Follicular Lymphoma",
        "Recurrent Mantle Cell Lymphoma",
        "Recurrent Marginal Zone Lymphoma",
        "Recurrent Mycosis Fungoides/Sezary Syndrome",
        "Recurrent Small Lymphocytic Lymphoma",
        "Recurrent/Refractory Childhood Hodgkin Lymphoma",
        "Refractory Chronic Lymphocytic Leukemia",
        "Refractory Hairy Cell Leukemia",
        "Refractory Multiple Myeloma",
        "Secondary Acute Myeloid Leukemia",
        "Secondary Myelodysplastic Syndromes",
        "Secondary Myelofibrosis",
        "Severe Combined Immunodeficiency",
        "Severe Congenital Neutropenia",
        "Shwachman-Diamond Syndrome",
        "Splenic Marginal Zone Lymphoma",
        "T-cell Large Granular Lymphocyte Leukemia",
        "Waldenstrom Macroglobulinemia",
        "Wiskott-Aldrich Syndrome"
      ],
      "interventions": [
        {
          "name": "fludarabine phosphate",
          "type": "DRUG"
        },
        {
          "name": "melphalan",
          "type": "DRUG"
        },
        {
          "name": "total-body irradiation",
          "type": "RADIATION"
        },
        {
          "name": "tacrolimus",
          "type": "DRUG"
        },
        {
          "name": "mycophenolate mofetil",
          "type": "DRUG"
        },
        {
          "name": "methotrexate",
          "type": "DRUG"
        },
        {
          "name": "laboratory biomarker analysis",
          "type": "OTHER"
        },
        {
          "name": "allogeneic hematopoietic stem cell transplantation",
          "type": "PROCEDURE"
        },
        {
          "name": "peripheral blood stem cell transplantation",
          "type": "PROCEDURE"
        }
      ],
      "intervention_types": [
        "DRUG",
        "RADIATION",
        "OTHER",
        "PROCEDURE"
      ],
      "sponsor": "Roswell Park Cancer Institute",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "3 Years",
        "maximum_age": "75 Years",
        "sex": "ALL",
        "summary": "3 Years to 75 Years"
      },
      "enrollment_count": 94,
      "start_date": "2012-02-28",
      "completion_date": "2019-08-29",
      "has_results": true,
      "last_update_posted_date": "2019-09-24",
      "last_synced_at": "2026-10-18T13:34:40.962Z",
      "location_count": 1,
      "location_summary": "Buffalo, New York",
      "locations": [
        {
          "city": "Buffalo",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01529827"
    },
    {
      "nct_id": "NCT04199000",
      "title": "Clinical and Basic Investigations Into Congenital Disorders of Glycosylation",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Congenital Disorders of Glycosylation"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Icahn School of Medicine at Mount Sinai",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 500,
      "start_date": "2019-10-08",
      "completion_date": "2030-07-31",
      "has_results": false,
      "last_update_posted_date": "2026-07-21",
      "last_synced_at": "2026-10-18T13:34:40.962Z",
      "location_count": 12,
      "location_summary": "San Diego, California • Aurora, Colorado • Jacksonville, Florida + 9 more",
      "locations": [
        {
          "city": "San Diego",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Jacksonville",
          "state": "Florida"
        },
        {
          "city": "New Orleans",
          "state": "Louisiana"
        },
        {
          "city": "Boston",
          "state": "Massachusetts"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04199000"
    },
    {
      "nct_id": "NCT05348564",
      "title": "Comparing Direct vs Indirect Methods for Cascade Screening",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "NA"
      ],
      "conditions": [
        "Long QT Syndrome",
        "Familial Hypercholesterolemia",
        "Ethics",
        "Genetic Testing"
      ],
      "interventions": [
        {
          "name": "Direct contact of family members for cascade screening of relevant variants",
          "type": "BEHAVIORAL"
        }
      ],
      "intervention_types": [
        "BEHAVIORAL"
      ],
      "sponsor": "University of Maryland, Baltimore",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 200,
      "start_date": "2023-05-15",
      "completion_date": "2027-10",
      "has_results": false,
      "last_update_posted_date": "2026-04-14",
      "last_synced_at": "2026-10-18T13:34:40.962Z",
      "location_count": 1,
      "location_summary": "Lancaster, Pennsylvania",
      "locations": [
        {
          "city": "Lancaster",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05348564"
    },
    {
      "nct_id": "NCT06164951",
      "title": "A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Achondroplasia"
      ],
      "interventions": [
        {
          "name": "Infigratinib 0.25 mg/kg/day",
          "type": "DRUG"
        },
        {
          "name": "Placebo Comparator 0.25 mg/kg/day",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "QED Therapeutics, a BridgeBio company",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "3 Years",
        "maximum_age": "17 Years",
        "sex": "ALL",
        "summary": "3 Years to 17 Years"
      },
      "enrollment_count": 114,
      "start_date": "2023-11-10",
      "completion_date": "2025-12-18",
      "has_results": false,
      "last_update_posted_date": "2026-03-17",
      "last_synced_at": "2026-10-18T13:34:40.962Z",
      "location_count": 7,
      "location_summary": "San Francisco, California • Aurora, Colorado • Baltimore, Maryland + 4 more",
      "locations": [
        {
          "city": "San Francisco",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Baltimore",
          "state": "Maryland"
        },
        {
          "city": "Columbia",
          "state": "Missouri"
        },
        {
          "city": "Cincinnati",
          "state": "Ohio"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06164951"
    },
    {
      "nct_id": "NCT00031434",
      "title": "Valganciclovir in Congenital CMV Infants",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Cytomegalovirus Infections"
      ],
      "interventions": [
        {
          "name": "Valganciclovir",
          "type": "DRUG"
        },
        {
          "name": "Ganciclovir",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "National Institute of Allergy and Infectious Diseases (NIAID)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "30 Days",
        "sex": "ALL",
        "summary": "Up to 30 Days"
      },
      "enrollment_count": 24,
      "start_date": "2002-07",
      "completion_date": "2007-07",
      "has_results": false,
      "last_update_posted_date": "2011-02-07",
      "last_synced_at": "2026-10-18T13:34:40.962Z",
      "location_count": 21,
      "location_summary": "Birmingham, Alabama • Little Rock, Arkansas • Los Angeles, California + 18 more",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Little Rock",
          "state": "Arkansas"
        },
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "Stanford",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00031434"
    },
    {
      "nct_id": "NCT02534844",
      "title": "VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2",
        "PHASE3"
      ],
      "conditions": [
        "Niemann-Pick Disease, Type C"
      ],
      "interventions": [
        {
          "name": "Parts A/B: Adrabetadex",
          "type": "DRUG"
        },
        {
          "name": "Parts A/B: Sham Control",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "DRUG",
        "OTHER"
      ],
      "sponsor": "Mandos LLC",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "4 Years",
        "maximum_age": "21 Years",
        "sex": "ALL",
        "summary": "4 Years to 21 Years"
      },
      "enrollment_count": 56,
      "start_date": "2015-10",
      "completion_date": "2018-03-28",
      "has_results": true,
      "last_update_posted_date": "2023-02-22",
      "last_synced_at": "2026-10-18T13:34:40.962Z",
      "location_count": 14,
      "location_summary": "Birmingham, Alabama • Orange, California • San Francisco, California + 11 more",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "San Francisco",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Gainesville",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02534844"
    }
  ]
}