{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Metabolism%2C+Inborn+Errors&page=12",
    "query": {
      "condition": "Metabolism, Inborn Errors",
      "page": 12
    },
    "page_size": 10
  },
  "pagination": {
    "page": 12,
    "page_size": 10,
    "total_count": 1608,
    "total_pages": 161,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Metabolism%2C+Inborn+Errors&page=13&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Metabolism%2C+Inborn+Errors&page=11&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-14T17:33:52.241Z",
  "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": "NCT00814073",
      "title": "Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Indolent Systemic Mastocytosis"
      ],
      "interventions": [
        {
          "name": "Masitinib",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        },
        {
          "name": "Best Supportive Care",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "DRUG",
        "OTHER"
      ],
      "sponsor": "AB Science",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "75 Years",
        "sex": "ALL",
        "summary": "18 Years to 75 Years"
      },
      "enrollment_count": 135,
      "start_date": "2008-12",
      "completion_date": "2015-11",
      "has_results": false,
      "last_update_posted_date": "2019-12-03",
      "last_synced_at": "2026-10-14T17:33:52.241Z",
      "location_count": 2,
      "location_summary": "Sacramento, California • Houston, Texas",
      "locations": [
        {
          "city": "Sacramento",
          "state": "California"
        },
        {
          "city": "Houston",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00814073"
    },
    {
      "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": "2026-10-14T17:33:52.241Z",
      "location_count": 1,
      "location_summary": "Irvine, California",
      "locations": [
        {
          "city": "Irvine",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02654886"
    },
    {
      "nct_id": "NCT02025543",
      "title": "Confounder-Corrected Quantitative MRI Biomarker of Hepatic Iron Content",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Iron Overload",
        "Hemochromatosis",
        "Hemosiderosis"
      ],
      "interventions": [
        {
          "name": "MRI",
          "type": "DEVICE"
        }
      ],
      "intervention_types": [
        "DEVICE"
      ],
      "sponsor": "University of Wisconsin, Madison",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "8 Years",
        "maximum_age": "89 Years",
        "sex": "ALL",
        "summary": "8 Years to 89 Years"
      },
      "enrollment_count": 207,
      "start_date": "2015-08-12",
      "completion_date": "2019-09",
      "has_results": false,
      "last_update_posted_date": "2025-08-12",
      "last_synced_at": "2026-10-14T17:33:52.241Z",
      "location_count": 4,
      "location_summary": "Palo Alto, California • Baltimore, Maryland • Dallas, Texas + 1 more",
      "locations": [
        {
          "city": "Palo Alto",
          "state": "California"
        },
        {
          "city": "Baltimore",
          "state": "Maryland"
        },
        {
          "city": "Dallas",
          "state": "Texas"
        },
        {
          "city": "Madison",
          "state": "Wisconsin"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02025543"
    },
    {
      "nct_id": "NCT04722068",
      "title": "Regeneron 1331 Kinetics Sub-Study HoFH",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "NA"
      ],
      "conditions": [
        "Hypercholesterolemia, Familial"
      ],
      "interventions": [
        {
          "name": "Kinetics test",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "University of Pennsylvania",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 4,
      "start_date": "2016-10-07",
      "completion_date": "2021-01-01",
      "has_results": false,
      "last_update_posted_date": "2021-05-17",
      "last_synced_at": "2026-10-14T17:33:52.241Z",
      "location_count": 1,
      "location_summary": "Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04722068"
    },
    {
      "nct_id": "NCT06255782",
      "title": "An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Ornithine Transcarbamylase Deficiency",
        "Ornithine Transcarbamylase Deficiency Disease",
        "Ornithine Carbamoyltransferase Deficiency (Disorder)",
        "Urea Cycle Disorders, Inborn"
      ],
      "interventions": [
        {
          "name": "ECUR-506",
          "type": "GENETIC"
        }
      ],
      "intervention_types": [
        "GENETIC"
      ],
      "sponsor": "iECURE, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "24 Hours",
        "maximum_age": "7 Months",
        "sex": "MALE",
        "summary": "24 Hours to 7 Months · Male only"
      },
      "enrollment_count": 20,
      "start_date": "2024-04-08",
      "completion_date": "2027-12",
      "has_results": false,
      "last_update_posted_date": "2026-09-30",
      "last_synced_at": "2026-10-14T17:33:52.241Z",
      "location_count": 6,
      "location_summary": "Los Angeles, California • Aurora, Colorado • Atlanta, Georgia + 3 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06255782"
    },
    {
      "nct_id": "NCT06258213",
      "title": "A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Oral ABP-745 Administration in Healthy Volunteers",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Gout",
        "Acute Gout Flare",
        "Gout Flares"
      ],
      "interventions": [
        {
          "name": "ABP-745",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Atom Therapeutics Co., Ltd",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "55 Years",
        "sex": "ALL",
        "summary": "18 Years to 55 Years"
      },
      "enrollment_count": 90,
      "start_date": "2024-01-23",
      "completion_date": "2024-07-29",
      "has_results": false,
      "last_update_posted_date": "2025-08-17",
      "last_synced_at": "2026-10-14T17:33:52.241Z",
      "location_count": 1,
      "location_summary": "San Antonio, Texas",
      "locations": [
        {
          "city": "San Antonio",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06258213"
    },
    {
      "nct_id": "NCT07748403",
      "title": "A Study of the Safety and Efficacy of Prime Editing (PM577) in Participants With Wilson Disease (WD)",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Wilson Disease",
        "Wilson's Disease",
        "Wilsons Disease"
      ],
      "interventions": [
        {
          "name": "PM577a",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Prime Medicine, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "12 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "12 Years and older"
      },
      "enrollment_count": 42,
      "start_date": "2026-09",
      "completion_date": "2028-12",
      "has_results": false,
      "last_update_posted_date": "2026-10-01",
      "last_synced_at": "2026-10-14T17:33:52.241Z",
      "location_count": 3,
      "location_summary": "New Haven, Connecticut • Chicago, Illinois • San Antonio, Texas",
      "locations": [
        {
          "city": "New Haven",
          "state": "Connecticut"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "San Antonio",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07748403"
    },
    {
      "nct_id": "NCT00104260",
      "title": "Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Phenylketonurias"
      ],
      "interventions": [
        {
          "name": "sapropterin dihydrochloride",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "BioMarin Pharmaceutical",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "8 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "8 Years and older"
      },
      "enrollment_count": 700,
      "start_date": "2004-12",
      "completion_date": "2005-11",
      "has_results": false,
      "last_update_posted_date": "2007-04-09",
      "last_synced_at": "2026-10-14T17:33:52.241Z",
      "location_count": 13,
      "location_summary": "Los Angeles, California • Oakland, California • New Haven, Connecticut + 10 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "New Haven",
          "state": "Connecticut"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "Boston",
          "state": "Massachusetts"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00104260"
    },
    {
      "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": "2026-10-14T17:33:52.241Z",
      "location_count": 1,
      "location_summary": "Buffalo, New York",
      "locations": [
        {
          "city": "Buffalo",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01529827"
    },
    {
      "nct_id": "NCT01421888",
      "title": "The NIH UNI Study: Urea Cycle Disorders, Nutrition and Immunity",
      "overall_status": "TERMINATED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Argininosuccinic Aciduria",
        "Carbamoyl-Phosphate Synthase I Deficiency",
        "Citrullinemia",
        "Ornithine Carbamoyltransferase Deficiency",
        "Hyperargininemia",
        "N-Acetylglutamate Synthase Deficiency"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "National Human Genome Research Institute (NHGRI)",
      "sponsor_class": "NIH",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": "99 Years",
        "sex": "ALL",
        "summary": "2 Years to 99 Years"
      },
      "enrollment_count": 4,
      "start_date": "2011-08-08",
      "completion_date": "2013-04-17",
      "has_results": false,
      "last_update_posted_date": "2019-12-09",
      "last_synced_at": "2026-10-14T17:33:52.241Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01421888"
    }
  ]
}