{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Metabolic+Inborn+Brain+Diseases",
    "query": {
      "condition": "Metabolic Inborn Brain Diseases"
    },
    "page_size": 10
  },
  "pagination": {
    "page": 1,
    "page_size": 10,
    "total_count": 783,
    "total_pages": 79,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Metabolic+Inborn+Brain+Diseases&page=2&page_size=10",
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-07-24T14:10:41.389Z",
  "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": "NCT07054515",
      "title": "A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Niemann-Pick Type C Disease",
        "GM1 Gangliosidosis",
        "GM2 Gangliosidosis"
      ],
      "interventions": [
        {
          "name": "AZ-3102",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Azafaros B.V.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "4 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "4 Years and older"
      },
      "enrollment_count": 147,
      "start_date": "2025-06-30",
      "completion_date": "2028-06-14",
      "has_results": false,
      "last_update_posted_date": "2026-07-21",
      "last_synced_at": "2026-07-24T14:10:41.389Z",
      "location_count": 5,
      "location_summary": "Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        },
        {
          "city": "Rochester",
          "state": "Minnesota"
        },
        {
          "city": "Dallas",
          "state": "Texas"
        },
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07054515"
    },
    {
      "nct_id": "NCT02595489",
      "title": "A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "X-linked Adrenoleukodystrophy"
      ],
      "interventions": [
        {
          "name": "vitamin D3",
          "type": "DIETARY_SUPPLEMENT"
        }
      ],
      "intervention_types": [
        "DIETARY_SUPPLEMENT"
      ],
      "sponsor": "Stanford University",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Months",
        "maximum_age": "25 Years",
        "sex": "MALE",
        "summary": "18 Months to 25 Years · Male only"
      },
      "enrollment_count": 21,
      "start_date": "2016-11-21",
      "completion_date": "2020-06-30",
      "has_results": false,
      "last_update_posted_date": "2022-06-09",
      "last_synced_at": "2026-07-24T14:10:41.389Z",
      "location_count": 1,
      "location_summary": "Palo Alto, California",
      "locations": [
        {
          "city": "Palo Alto",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02595489"
    },
    {
      "nct_id": "NCT03190837",
      "title": "A Long-term Follow-up Study of Gaucher Disease",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Gaucher Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Duke University",
      "sponsor_class": "OTHER",
      "healthy_volunteers": null,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 200,
      "start_date": "2017-06-12",
      "completion_date": "2027-01",
      "has_results": false,
      "last_update_posted_date": "2025-09-22",
      "last_synced_at": "2026-07-24T14:10:41.389Z",
      "location_count": 1,
      "location_summary": "Durham, North Carolina",
      "locations": [
        {
          "city": "Durham",
          "state": "North Carolina"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03190837"
    },
    {
      "nct_id": "NCT02233257",
      "title": "Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy",
      "overall_status": "NO_LONGER_AVAILABLE",
      "study_type": "EXPANDED_ACCESS",
      "phases": [],
      "conditions": [
        "X-linked Adrenoleukodystrophy"
      ],
      "interventions": [
        {
          "name": "Lorenzo's Oil",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "University of Minnesota",
      "sponsor_class": "OTHER",
      "healthy_volunteers": null,
      "eligibility": {
        "minimum_age": "18 Months",
        "maximum_age": "18 Years",
        "sex": "MALE",
        "summary": "18 Months to 18 Years · Male only"
      },
      "enrollment_count": null,
      "start_date": null,
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2018-01-23",
      "last_synced_at": "2026-07-24T14:10:41.389Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02233257"
    },
    {
      "nct_id": "NCT06054893",
      "title": "A Study to Find Out How BIIB141 (Omaveloxolone) is Processed in the Body and to Learn More About Its Safety in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Friedreich Ataxia"
      ],
      "interventions": [
        {
          "name": "Omaveloxolone",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Biogen",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": "15 Years",
        "sex": "ALL",
        "summary": "2 Years to 15 Years"
      },
      "enrollment_count": 33,
      "start_date": "2024-07-01",
      "completion_date": "2030-11-22",
      "has_results": false,
      "last_update_posted_date": "2026-05-11",
      "last_synced_at": "2026-07-24T14:10:41.389Z",
      "location_count": 1,
      "location_summary": "Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06054893"
    },
    {
      "nct_id": "NCT00286689",
      "title": "Effects of Growth Hormone in Chronically Ill Children",
      "overall_status": "WITHDRAWN",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "NA"
      ],
      "conditions": [
        "Hurler Syndrome",
        "Cerebral Palsy",
        "Juvenile Rheumatoid Arthritis",
        "Crohn Disease",
        "HIV Infections"
      ],
      "interventions": [
        {
          "name": "Growth Hormone",
          "type": "DRUG"
        },
        {
          "name": "Whole body Protein turnover",
          "type": "PROCEDURE"
        },
        {
          "name": "DEXA scan",
          "type": "PROCEDURE"
        }
      ],
      "intervention_types": [
        "DRUG",
        "PROCEDURE"
      ],
      "sponsor": "University of Texas Southwestern Medical Center",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "3 Years",
        "maximum_age": "17 Years",
        "sex": "ALL",
        "summary": "3 Years to 17 Years"
      },
      "enrollment_count": 0,
      "start_date": "2006-02-03",
      "completion_date": "2006-02-03",
      "has_results": false,
      "last_update_posted_date": "2018-12-14",
      "last_synced_at": "2026-07-24T14:10:41.389Z",
      "location_count": 1,
      "location_summary": "Dallas, Texas",
      "locations": [
        {
          "city": "Dallas",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00286689"
    },
    {
      "nct_id": "NCT04804566",
      "title": "Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [
        {
          "name": "This is a non-interventional study",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "Amicus Therapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 44,
      "start_date": "2023-03-01",
      "completion_date": "2023-11-01",
      "has_results": false,
      "last_update_posted_date": "2024-04-03",
      "last_synced_at": "2026-07-24T14:10:41.389Z",
      "location_count": 1,
      "location_summary": "Eagan, Minnesota",
      "locations": [
        {
          "city": "Eagan",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04804566"
    },
    {
      "nct_id": "NCT00410566",
      "title": "Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Acid Sphingomyelinase Deficiency",
        "Niemann-Pick Disease"
      ],
      "interventions": [
        {
          "name": "rhASM",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Genzyme, a Sanofi Company",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "65 Years",
        "sex": "ALL",
        "summary": "18 Years to 65 Years"
      },
      "enrollment_count": 11,
      "start_date": "2006-12",
      "completion_date": "2009-04",
      "has_results": false,
      "last_update_posted_date": "2015-03-19",
      "last_synced_at": "2026-07-24T14:10:41.389Z",
      "location_count": 1,
      "location_summary": "New York, New York",
      "locations": [
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00410566"
    },
    {
      "nct_id": "NCT01610089",
      "title": "Nitric Oxide Flux and Ureagenesis in Argininosuccinate Synthetase Deficiency (ASSD)(Citrullinemia I)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "NA"
      ],
      "conditions": [
        "Citrullinemia"
      ],
      "interventions": [
        {
          "name": "stable isotope infusion",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "Baylor College of Medicine",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 6,
      "start_date": "2012-12",
      "completion_date": "2013-09",
      "has_results": false,
      "last_update_posted_date": "2015-03-20",
      "last_synced_at": "2026-07-24T14:10:41.389Z",
      "location_count": 1,
      "location_summary": "Houston, Texas",
      "locations": [
        {
          "city": "Houston",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01610089"
    },
    {
      "nct_id": "NCT00302146",
      "title": "Positron Emission Tomography (PET) Imaging in People With Gaucher Mutations",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Parkinson Disease",
        "Gaucher Disease"
      ],
      "interventions": [
        {
          "name": "15-0 H20",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "National Human Genome Research Institute (NHGRI)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "110 Years",
        "sex": "ALL",
        "summary": "18 Years to 110 Years"
      },
      "enrollment_count": 64,
      "start_date": "2006-05-23",
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2026-06-18",
      "last_synced_at": "2026-07-24T14:10:41.389Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00302146"
    }
  ]
}