{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=11",
    "query": {
      "condition": "Inborn Errors of Metabolism",
      "page": 11
    },
    "page_size": 10
  },
  "pagination": {
    "page": 11,
    "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=12&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=10&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-14T09:43:30.584Z",
  "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": "NCT00001736",
      "title": "New Cysteamine Eye Drops Formulation to Treat Corneal Crystals in Cystinosis",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Cystinosis"
      ],
      "interventions": [
        {
          "name": "Cysteamine",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "National Eye Institute (NEI)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 51,
      "start_date": "1998-05",
      "completion_date": "2001-03",
      "has_results": false,
      "last_update_posted_date": "2008-03-04",
      "last_synced_at": "2026-10-14T09:43:30.584Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00001736"
    },
    {
      "nct_id": "NCT02254863",
      "title": "UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Adrenoleukodystrophy",
        "Batten Disease",
        "Mucopolysaccharidosis II",
        "Leukodystrophy, Globoid Cell",
        "Leukodystrophy, Metachromatic",
        "Neimann Pick Disease",
        "Pelizaeus-Merzbacher Disease",
        "Sandhoff Disease",
        "Tay-Sachs Disease",
        "Brain Diseases, Metabolic, Inborn",
        "Alpha-Mannosidosis",
        "Sanfilippo Mucopolysaccharidoses"
      ],
      "interventions": [
        {
          "name": "DUOC-01",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Joanne Kurtzberg, MD",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Week",
        "maximum_age": "22 Years",
        "sex": "ALL",
        "summary": "1 Week to 22 Years"
      },
      "enrollment_count": 40,
      "start_date": "2014-09",
      "completion_date": "2027-10",
      "has_results": false,
      "last_update_posted_date": "2026-09-03",
      "last_synced_at": "2026-10-14T09:43:30.584Z",
      "location_count": 1,
      "location_summary": "Durham, North Carolina",
      "locations": [
        {
          "city": "Durham",
          "state": "North Carolina"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02254863"
    },
    {
      "nct_id": "NCT05659901",
      "title": "Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Pelizaeus-Merzbacher Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Ionis Pharmaceuticals, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Months",
        "maximum_age": "17 Years",
        "sex": "MALE",
        "summary": "6 Months to 17 Years · Male only"
      },
      "enrollment_count": 32,
      "start_date": "2022-10-03",
      "completion_date": "2029-03",
      "has_results": false,
      "last_update_posted_date": "2026-10-02",
      "last_synced_at": "2026-10-14T09:43:30.584Z",
      "location_count": 3,
      "location_summary": "Atlanta, Georgia • Baltimore, Maryland • Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Baltimore",
          "state": "Maryland"
        },
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05659901"
    },
    {
      "nct_id": "NCT04849741",
      "title": "A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Alexander Disease"
      ],
      "interventions": [
        {
          "name": "zilganersen",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Ionis Pharmaceuticals, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": "65 Years",
        "sex": "ALL",
        "summary": "2 Years to 65 Years"
      },
      "enrollment_count": 54,
      "start_date": "2021-06-01",
      "completion_date": "2034-07",
      "has_results": false,
      "last_update_posted_date": "2026-08-21",
      "last_synced_at": "2026-10-14T09:43:30.584Z",
      "location_count": 4,
      "location_summary": "Palo Alto, California • Atlanta, Georgia • Boston, Massachusetts + 1 more",
      "locations": [
        {
          "city": "Palo Alto",
          "state": "California"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Boston",
          "state": "Massachusetts"
        },
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04849741"
    },
    {
      "nct_id": "NCT01387854",
      "title": "Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "MPS VI"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "BioMarin Pharmaceutical",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 59,
      "start_date": "2011-06",
      "completion_date": "2013-02",
      "has_results": false,
      "last_update_posted_date": "2014-04-02",
      "last_synced_at": "2026-10-14T09:43:30.584Z",
      "location_count": 1,
      "location_summary": "Oakland, California",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01387854"
    },
    {
      "nct_id": "NCT04977388",
      "title": "NORTHERA (DROXIDOPA) for Dysautonomia in Adult Survivors of Menkes Disease and Occipital Horn Syndrome",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Menkes Disease",
        "Occipital Horn Syndrome"
      ],
      "interventions": [
        {
          "name": "Droxidopa",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "DRUG",
        "OTHER"
      ],
      "sponsor": "Stephen G. Kaler, MD",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "50 Years",
        "sex": "ALL",
        "summary": "18 Years to 50 Years"
      },
      "enrollment_count": 3,
      "start_date": "2021-07-12",
      "completion_date": "2024-06-29",
      "has_results": true,
      "last_update_posted_date": "2026-04-13",
      "last_synced_at": "2026-10-14T09:43:30.584Z",
      "location_count": 1,
      "location_summary": "New York, New York",
      "locations": [
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04977388"
    },
    {
      "nct_id": "NCT07712003",
      "title": "Natural History to Assess Disease in Patients With MPS IIIC",
      "overall_status": "NOT_YET_RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Sanfilippo Syndrome Type C",
        "Mucopolysaccharidosis (MPS) IIIC"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Phoenix Nest",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Year",
        "maximum_age": "25 Years",
        "sex": "ALL",
        "summary": "1 Year to 25 Years"
      },
      "enrollment_count": 35,
      "start_date": "2026-11-01",
      "completion_date": "2029-02-01",
      "has_results": false,
      "last_update_posted_date": "2026-07-17",
      "last_synced_at": "2026-10-14T09:43:30.584Z",
      "location_count": 1,
      "location_summary": "Dallas, Texas",
      "locations": [
        {
          "city": "Dallas",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07712003"
    },
    {
      "nct_id": "NCT00675103",
      "title": "Re-exposure Study of Pegloticase Intravenous (i.v.) in Symptomatic Gout Patients",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Chronic Gout Refractory to Conventional Therapy"
      ],
      "interventions": [
        {
          "name": "pegloticase 8 mg i.v.",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Savient Pharmaceuticals",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 7,
      "start_date": "2008-05",
      "completion_date": "2009-04",
      "has_results": true,
      "last_update_posted_date": "2011-06-28",
      "last_synced_at": "2026-10-14T09:43:30.584Z",
      "location_count": 4,
      "location_summary": "Chicago, Illinois • Wheaton, Maryland • Durham, North Carolina + 1 more",
      "locations": [
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "Wheaton",
          "state": "Maryland"
        },
        {
          "city": "Durham",
          "state": "North Carolina"
        },
        {
          "city": "Lake Oswego",
          "state": "Oregon"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00675103"
    },
    {
      "nct_id": "NCT03403205",
      "title": "Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Wilson Disease"
      ],
      "interventions": [
        {
          "name": "ALXN1840",
          "type": "DRUG"
        },
        {
          "name": "SoC Therapy",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Alexion Pharmaceuticals, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "12 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "12 Years and older"
      },
      "enrollment_count": 214,
      "start_date": "2018-02-22",
      "completion_date": "2023-06-30",
      "has_results": true,
      "last_update_posted_date": "2024-06-17",
      "last_synced_at": "2026-10-14T09:43:30.584Z",
      "location_count": 6,
      "location_summary": "Los Angeles, California • New Haven, Connecticut • Chicago, Illinois + 3 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "New Haven",
          "state": "Connecticut"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "Ann Arbor",
          "state": "Michigan"
        },
        {
          "city": "Houston",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03403205"
    },
    {
      "nct_id": "NCT03882437",
      "title": "Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Danon Disease"
      ],
      "interventions": [
        {
          "name": "RP-A501",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Rocket Pharmaceuticals Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "8 Years",
        "maximum_age": null,
        "sex": "MALE",
        "summary": "8 Years and older · Male only"
      },
      "enrollment_count": 7,
      "start_date": "2019-04-17",
      "completion_date": "2025-07-03",
      "has_results": false,
      "last_update_posted_date": "2026-10-06",
      "last_synced_at": "2026-10-14T09:43:30.584Z",
      "location_count": 3,
      "location_summary": "La Jolla, California • Aurora, Colorado • Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "La Jolla",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03882437"
    }
  ]
}