{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Brain+Diseases%2C+Metabolic%2C+Inborn",
    "query": {
      "condition": "Brain Diseases, Metabolic, Inborn"
    },
    "page_size": 10
  },
  "pagination": {
    "page": 1,
    "page_size": 10,
    "total_count": 776,
    "total_pages": 78,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Brain+Diseases%2C+Metabolic%2C+Inborn&page=2&page_size=10",
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-05-22T03:09:24.847Z",
  "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": "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": "2026-05-22T03:09:24.847Z",
      "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": "NCT06402123",
      "title": "A Phase 2b Study of Zagociguat in Patients With MELAS",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)"
      ],
      "interventions": [
        {
          "name": "zagociguat 15mg",
          "type": "DRUG"
        },
        {
          "name": "zagociguat 30mg",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Tisento Therapeutics",
      "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": 43,
      "start_date": "2024-09-10",
      "completion_date": "2026-06-20",
      "has_results": false,
      "last_update_posted_date": "2026-03-25",
      "last_synced_at": "2026-05-22T03:09:24.847Z",
      "location_count": 10,
      "location_summary": "La Jolla, California • Aurora, Colorado • Atlanta, Georgia + 5 more",
      "locations": [
        {
          "city": "La Jolla",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Boston",
          "state": "Massachusetts"
        },
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06402123"
    },
    {
      "nct_id": "NCT00001972",
      "title": "PET Scan of Brain Metabolism in Relation to Age and Disease",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Alzheimer's Disease",
        "Brain Neoplasm",
        "Niemann Pick Disease"
      ],
      "interventions": [
        {
          "name": "15 O Water",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "National Institute of Neurological Disorders and Stroke (NINDS)",
      "sponsor_class": "NIH",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 123,
      "start_date": "1994-09",
      "completion_date": "2002-08",
      "has_results": false,
      "last_update_posted_date": "2008-03-04",
      "last_synced_at": "2026-05-22T03:09:24.847Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00001972"
    },
    {
      "nct_id": "NCT00343577",
      "title": "Antiproteinuric Agents and Fabry Disease",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Fabry Disease",
        "Proteinuria"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "University of Alabama at Birmingham",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "14 Years",
        "maximum_age": "95 Years",
        "sex": "ALL",
        "summary": "14 Years to 95 Years"
      },
      "enrollment_count": 12,
      "start_date": "2001-01",
      "completion_date": "2006-12",
      "has_results": false,
      "last_update_posted_date": "2013-11-19",
      "last_synced_at": "2026-05-22T03:09:24.847Z",
      "location_count": 1,
      "location_summary": "Birmingham, Alabama",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00343577"
    },
    {
      "nct_id": "NCT01896102",
      "title": "A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2",
        "PHASE3"
      ],
      "conditions": [
        "Cerebral Adrenoleukodystrophy (CALD)"
      ],
      "interventions": [
        {
          "name": "Lenti-D Drug Product (eli-cel)",
          "type": "GENETIC"
        }
      ],
      "intervention_types": [
        "GENETIC"
      ],
      "sponsor": "Genetix Biotherapeutics Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "17 Years",
        "sex": "MALE",
        "summary": "Up to 17 Years · Male only"
      },
      "enrollment_count": 32,
      "start_date": "2013-08-21",
      "completion_date": "2021-03-26",
      "has_results": true,
      "last_update_posted_date": "2022-04-25",
      "last_synced_at": "2026-05-22T03:09:24.847Z",
      "location_count": 3,
      "location_summary": "Los Angeles, California • Boston, Massachusetts • Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Boston",
          "state": "Massachusetts"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01896102"
    },
    {
      "nct_id": "NCT01806051",
      "title": "A Pilot Study on Diurnal Variation",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "NA"
      ],
      "conditions": [
        "Phenylketonuria (PKU)"
      ],
      "interventions": [
        {
          "name": "Kuvan",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Dr. Linda Randolph",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "4 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "4 Years and older"
      },
      "enrollment_count": 6,
      "start_date": "2013-03",
      "completion_date": "2014-05",
      "has_results": true,
      "last_update_posted_date": "2016-04-20",
      "last_synced_at": "2026-05-22T03:09:24.847Z",
      "location_count": 1,
      "location_summary": "Los Angeles, California",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01806051"
    },
    {
      "nct_id": "NCT04221451",
      "title": "A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Tay-Sachs Disease",
        "Sandhoff Disease"
      ],
      "interventions": [
        {
          "name": "venglustat GZ402671",
          "type": "DRUG"
        },
        {
          "name": "placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Genzyme, a Sanofi Company",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "2 Years and older"
      },
      "enrollment_count": 75,
      "start_date": "2020-06-29",
      "completion_date": "2024-12-26",
      "has_results": true,
      "last_update_posted_date": "2026-01-28",
      "last_synced_at": "2026-05-22T03:09:24.847Z",
      "location_count": 5,
      "location_summary": "Los Angeles, California • Atlanta, Georgia • Bethesda, Maryland + 2 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Bethesda",
          "state": "Maryland"
        },
        {
          "city": "Boston",
          "state": "Massachusetts"
        },
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04221451"
    },
    {
      "nct_id": "NCT01594853",
      "title": "Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "NA"
      ],
      "conditions": [
        "X-linked Adrenoleukodystrophy"
      ],
      "interventions": [
        {
          "name": "exercise training",
          "type": "BEHAVIORAL"
        }
      ],
      "intervention_types": [
        "BEHAVIORAL"
      ],
      "sponsor": "Hugo W. Moser Research Institute at Kennedy Krieger, Inc.",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "21 Years",
        "maximum_age": "70 Years",
        "sex": "FEMALE",
        "summary": "21 Years to 70 Years · Female only"
      },
      "enrollment_count": 31,
      "start_date": "2012-05",
      "completion_date": "2014-10",
      "has_results": true,
      "last_update_posted_date": "2018-08-29",
      "last_synced_at": "2026-05-22T03:09:24.847Z",
      "location_count": 1,
      "location_summary": "Baltimore, Maryland",
      "locations": [
        {
          "city": "Baltimore",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01594853"
    },
    {
      "nct_id": "NCT04721366",
      "title": "A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Gaucher Disease"
      ],
      "interventions": [
        {
          "name": "Standard of Care",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "Takeda",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "5 Years",
        "sex": "ALL",
        "summary": "Up to 5 Years"
      },
      "enrollment_count": 11,
      "start_date": "2021-01-08",
      "completion_date": "2023-04-17",
      "has_results": false,
      "last_update_posted_date": "2024-03-29",
      "last_synced_at": "2026-05-22T03:09:24.847Z",
      "location_count": 1,
      "location_summary": "Fairfax, Virginia",
      "locations": [
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04721366"
    },
    {
      "nct_id": "NCT04422431",
      "title": "Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Wilson Disease"
      ],
      "interventions": [
        {
          "name": "Bis-Choline Tetrathiomolybdate",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Alexion 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": 31,
      "start_date": "2020-12-02",
      "completion_date": "2023-05-17",
      "has_results": true,
      "last_update_posted_date": "2024-10-18",
      "last_synced_at": "2026-05-22T03:09:24.847Z",
      "location_count": 3,
      "location_summary": "Sacramento, California • Ann Arbor, Michigan • Dallas, Texas",
      "locations": [
        {
          "city": "Sacramento",
          "state": "California"
        },
        {
          "city": "Ann Arbor",
          "state": "Michigan"
        },
        {
          "city": "Dallas",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04422431"
    }
  ]
}