{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=52",
    "query": {
      "condition": "Inborn Errors of Metabolism",
      "page": 52
    },
    "page_size": 10
  },
  "pagination": {
    "page": 52,
    "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=53&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=51&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-19T15:25:15.636Z",
  "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": "NCT00730236",
      "title": "A Safety and Efficacy Study of AEGR-733 to Treat Homozygous Familial Hypercholesterolemia (FH)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Homozygous Familial Hypercholesterolemia"
      ],
      "interventions": [
        {
          "name": "AEGR-733",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Aegerion Pharmaceuticals, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 29,
      "start_date": "2007-12",
      "completion_date": "2011-10",
      "has_results": true,
      "last_update_posted_date": "2018-03-20",
      "last_synced_at": "2026-10-19T15:25:15.636Z",
      "location_count": 2,
      "location_summary": "Los Angeles, California • Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00730236"
    },
    {
      "nct_id": "NCT05588167",
      "title": "Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Niemann-Pick Disease, Type C"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "3 Months",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "3 Months and older"
      },
      "enrollment_count": 100,
      "start_date": "2022-11-28",
      "completion_date": "2027-09-30",
      "has_results": false,
      "last_update_posted_date": "2026-10-15",
      "last_synced_at": "2026-10-19T15:25:15.636Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05588167"
    },
    {
      "nct_id": "NCT00004307",
      "title": "Study of Treatment and Metabolism in Patients With Urea Cycle Disorders",
      "overall_status": "UNKNOWN",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Amino Acid Metabolism, Inborn Errors"
      ],
      "interventions": [
        {
          "name": "Protein and calorie controlled diet",
          "type": "BEHAVIORAL"
        },
        {
          "name": "Ornithine transcarbamylase vector",
          "type": "GENETIC"
        }
      ],
      "intervention_types": [
        "BEHAVIORAL",
        "GENETIC"
      ],
      "sponsor": "National Center for Research Resources (NCRR)",
      "sponsor_class": "NIH",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "6 Months",
        "maximum_age": "64 Years",
        "sex": "ALL",
        "summary": "6 Months to 64 Years"
      },
      "enrollment_count": 66,
      "start_date": "1999-12",
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2005-06-24",
      "last_synced_at": "2026-10-19T15:25:15.636Z",
      "location_count": 1,
      "location_summary": "Houston, Texas",
      "locations": [
        {
          "city": "Houston",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00004307"
    },
    {
      "nct_id": "NCT05063994",
      "title": "Comparison of Chronocort Versus Standard Hydrocortisone Replacement Therapy in Participants Aged 16 Years and Over With Congenital Adrenal Hyperplasia",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Congenital Adrenal Hyperplasia"
      ],
      "interventions": [
        {
          "name": "Chronocort",
          "type": "DRUG"
        },
        {
          "name": "Cortef",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "DRUG",
        "OTHER"
      ],
      "sponsor": "Neurocrine UK Limited",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "16 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "16 Years and older"
      },
      "enrollment_count": 55,
      "start_date": "2022-05-24",
      "completion_date": "2024-02-02",
      "has_results": true,
      "last_update_posted_date": "2026-10-02",
      "last_synced_at": "2026-10-19T15:25:15.636Z",
      "location_count": 11,
      "location_summary": "Los Angeles, California • Orange, California • Jacksonville, Florida + 8 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "Jacksonville",
          "state": "Florida"
        },
        {
          "city": "Iowa City",
          "state": "Iowa"
        },
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05063994"
    },
    {
      "nct_id": "NCT03378531",
      "title": "A Study of AEB1102 (Pegzilarginase) in Patients With Arginase 1 Deficiency",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Arginase I Deficiency",
        "Hyperargininemia"
      ],
      "interventions": [
        {
          "name": "AEB1102",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Immedica Pharma AB",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "2 Years and older"
      },
      "enrollment_count": 14,
      "start_date": "2017-12-07",
      "completion_date": "2022-12-15",
      "has_results": true,
      "last_update_posted_date": "2026-09-24",
      "last_synced_at": "2026-10-19T15:25:15.636Z",
      "location_count": 4,
      "location_summary": "Stanford, California • Gainsville, Florida • New York, New York + 1 more",
      "locations": [
        {
          "city": "Stanford",
          "state": "California"
        },
        {
          "city": "Gainsville",
          "state": "Florida"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Dallas",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03378531"
    },
    {
      "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": "2026-10-19T15:25:15.636Z",
      "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": "NCT01257737",
      "title": "To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE4"
      ],
      "conditions": [
        "Urea Cycle Disorders"
      ],
      "interventions": [
        {
          "name": "HPN-100",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Amgen",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Year",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "1 Year and older"
      },
      "enrollment_count": 88,
      "start_date": "2010-10-04",
      "completion_date": "2017-02-16",
      "has_results": true,
      "last_update_posted_date": "2024-08-22",
      "last_synced_at": "2026-10-19T15:25:15.636Z",
      "location_count": 15,
      "location_summary": "Los Angeles, California • Palo Alto, California • Aurora, Colorado + 12 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Palo Alto",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Washington D.C.",
          "state": "District of Columbia"
        },
        {
          "city": "Portland",
          "state": "Maine"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01257737"
    },
    {
      "nct_id": "NCT00000805",
      "title": "A Pharmacokinetic and Tolerance Study of Oral Ganciclovir in HIV-Infected Children With Asymptomatic Cytomegalovirus Infection and Low CD4 Cell Counts or Quiescent Cytomegalovirus Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Cytomegalovirus Infections",
        "HIV Infections"
      ],
      "interventions": [
        {
          "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": "14 Days",
        "maximum_age": "20 Years",
        "sex": "ALL",
        "summary": "14 Days to 20 Years"
      },
      "enrollment_count": 32,
      "start_date": null,
      "completion_date": "1998-03",
      "has_results": false,
      "last_update_posted_date": "2021-11-03",
      "last_synced_at": "2026-10-19T15:25:15.636Z",
      "location_count": 18,
      "location_summary": "Los Angeles, California • Oakland, California • San Diego, California + 10 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "San Diego",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00000805"
    },
    {
      "nct_id": "NCT04639336",
      "title": "Cognitive and Neurological Pathologies in Pompe Disease",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Pompe Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Duke University",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "0 Years",
        "maximum_age": "18 Years",
        "sex": "ALL",
        "summary": "0 Years to 18 Years"
      },
      "enrollment_count": 45,
      "start_date": "2016-08-03",
      "completion_date": "2029-03",
      "has_results": false,
      "last_update_posted_date": "2026-03-04",
      "last_synced_at": "2026-10-19T15:25:15.636Z",
      "location_count": 1,
      "location_summary": "Durham, North Carolina",
      "locations": [
        {
          "city": "Durham",
          "state": "North Carolina"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04639336"
    },
    {
      "nct_id": "NCT05345171",
      "title": "Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "OTC Deficiency"
      ],
      "interventions": [
        {
          "name": "DTX301",
          "type": "GENETIC"
        },
        {
          "name": "Placebo",
          "type": "OTHER"
        },
        {
          "name": "Oral Corticosteroids",
          "type": "DRUG"
        },
        {
          "name": "Placebo for oral corticosteroids",
          "type": "DRUG"
        },
        {
          "name": "Sodium Acetate",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "GENETIC",
        "OTHER",
        "DRUG"
      ],
      "sponsor": "Ultragenyx Pharmaceutical Inc",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "12 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "12 Years and older"
      },
      "enrollment_count": 37,
      "start_date": "2022-10-18",
      "completion_date": "2031-03",
      "has_results": false,
      "last_update_posted_date": "2026-09-09",
      "last_synced_at": "2026-10-19T15:25:15.636Z",
      "location_count": 4,
      "location_summary": "Los Angeles, California • Aurora, Colorado • Chicago, Illinois + 1 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "Cleveland",
          "state": "Ohio"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05345171"
    }
  ]
}