{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Brain+Diseases%2C+Metabolic%2C+Inborn&page=3",
    "query": {
      "condition": "Brain Diseases, Metabolic, Inborn",
      "page": 3
    },
    "page_size": 10
  },
  "pagination": {
    "page": 3,
    "page_size": 10,
    "total_count": 791,
    "total_pages": 80,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Brain+Diseases%2C+Metabolic%2C+Inborn&page=4&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Brain+Diseases%2C+Metabolic%2C+Inborn&page=2&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-07T17:16:07.660Z",
  "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": "NCT01114737",
      "title": "Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Phenylketonuria"
      ],
      "interventions": [
        {
          "name": "Sapropterin dihydrochloride",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "BioMarin Pharmaceutical",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "8 Years",
        "maximum_age": "65 Years",
        "sex": "ALL",
        "summary": "8 Years to 65 Years"
      },
      "enrollment_count": 206,
      "start_date": "2010-08",
      "completion_date": "2013-03",
      "has_results": true,
      "last_update_posted_date": "2016-02-01",
      "last_synced_at": "2026-10-07T17:16:07.660Z",
      "location_count": 26,
      "location_summary": "La Jolla, California • Los Angeles, California • Palo Alto, California + 23 more",
      "locations": [
        {
          "city": "La Jolla",
          "state": "California"
        },
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Palo Alto",
          "state": "California"
        },
        {
          "city": "San Francisco",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01114737"
    },
    {
      "nct_id": "NCT01212744",
      "title": "Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Phenylketonuria"
      ],
      "interventions": [
        {
          "name": "rAvPAL-PEG",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "BioMarin Pharmaceutical",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "16 Years",
        "maximum_age": "70 Years",
        "sex": "ALL",
        "summary": "16 Years to 70 Years"
      },
      "enrollment_count": 16,
      "start_date": "2011-03",
      "completion_date": "2015-04",
      "has_results": true,
      "last_update_posted_date": "2019-02-26",
      "last_synced_at": "2026-10-07T17:16:07.660Z",
      "location_count": 9,
      "location_summary": "Aurora, Colorado • Gainesville, Florida • Louisville, Kentucky + 6 more",
      "locations": [
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Gainesville",
          "state": "Florida"
        },
        {
          "city": "Louisville",
          "state": "Kentucky"
        },
        {
          "city": "Columbia",
          "state": "Missouri"
        },
        {
          "city": "St Louis",
          "state": "Missouri"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01212744"
    },
    {
      "nct_id": "NCT03639844",
      "title": "BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study",
      "overall_status": "NO_LONGER_AVAILABLE",
      "study_type": "EXPANDED_ACCESS",
      "phases": [],
      "conditions": [
        "Hurler Syndrome",
        "Inherited Metabolic Disorder",
        "Lysosomal Storage Disorder",
        "Metachromatic Leukodystrophy",
        "Inborn Errors of Metabolism"
      ],
      "interventions": [
        {
          "name": "rivogenlecleucel",
          "type": "BIOLOGICAL"
        },
        {
          "name": "rimiducid",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL",
        "DRUG"
      ],
      "sponsor": "Bellicum Pharmaceuticals",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": null,
      "eligibility": {
        "minimum_age": "3 Months",
        "maximum_age": "21 Years",
        "sex": "ALL",
        "summary": "3 Months to 21 Years"
      },
      "enrollment_count": null,
      "start_date": null,
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2020-10-05",
      "last_synced_at": "2026-10-07T17:16:07.660Z",
      "location_count": 2,
      "location_summary": "Los Angeles, California • Palo Alto, California",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Palo Alto",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03639844"
    },
    {
      "nct_id": "NCT01855360",
      "title": "Doxycycline and TUDCA in Patients With Transthyretin Amyloid Cardiomyopathy",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Amyloidosis; Heart (Manifestation)",
        "Senile Cardiac Amyloidosis"
      ],
      "interventions": [
        {
          "name": "Tauroursodeoxycholic Acid and Doxycycline",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Brigham and Women's Hospital",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "90 Years",
        "sex": "ALL",
        "summary": "18 Years to 90 Years"
      },
      "enrollment_count": 40,
      "start_date": "2013-06",
      "completion_date": "2015-05-31",
      "has_results": true,
      "last_update_posted_date": "2020-12-04",
      "last_synced_at": "2026-10-07T17:16:07.660Z",
      "location_count": 1,
      "location_summary": "Boston, Massachusetts",
      "locations": [
        {
          "city": "Boston",
          "state": "Massachusetts"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01855360"
    },
    {
      "nct_id": "NCT00446550",
      "title": "A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Gaucher Disease",
        "Type 1 Gaucher Disease",
        "Gaucher Disease, Type 1"
      ],
      "interventions": [
        {
          "name": "afegostat tartrate",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Amicus Therapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "74 Years",
        "sex": "ALL",
        "summary": "18 Years to 74 Years"
      },
      "enrollment_count": 19,
      "start_date": "2008-06-11",
      "completion_date": "2009-08-20",
      "has_results": true,
      "last_update_posted_date": "2018-08-15",
      "last_synced_at": "2026-10-07T17:16:07.660Z",
      "location_count": 7,
      "location_summary": "Beverly Hills, California • Coral Springs, Florida • Decatur, Georgia + 4 more",
      "locations": [
        {
          "city": "Beverly Hills",
          "state": "California"
        },
        {
          "city": "Coral Springs",
          "state": "Florida"
        },
        {
          "city": "Decatur",
          "state": "Georgia"
        },
        {
          "city": "Iowa City",
          "state": "Iowa"
        },
        {
          "city": "Kansas City",
          "state": "Kansas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00446550"
    },
    {
      "nct_id": "NCT07180355",
      "title": "A Study of SGT-212 Gene Therapy in Friedreich's Ataxia",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Friedreich's Ataxia (FA)"
      ],
      "interventions": [
        {
          "name": "SGT-212",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Solid Biosciences Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "40 Years",
        "sex": "ALL",
        "summary": "18 Years to 40 Years"
      },
      "enrollment_count": 10,
      "start_date": "2025-10-22",
      "completion_date": "2032-02-29",
      "has_results": false,
      "last_update_posted_date": "2026-09-29",
      "last_synced_at": "2026-10-07T17:16:07.660Z",
      "location_count": 3,
      "location_summary": "Los Angeles, California • Columbus, Ohio • Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Columbus",
          "state": "Ohio"
        },
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07180355"
    },
    {
      "nct_id": "NCT01581424",
      "title": "Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "University of Minnesota",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Year",
        "maximum_age": "75 Years",
        "sex": "ALL",
        "summary": "1 Year to 75 Years"
      },
      "enrollment_count": 50,
      "start_date": "2010-10",
      "completion_date": "2030-07-31",
      "has_results": false,
      "last_update_posted_date": "2026-08-14",
      "last_synced_at": "2026-10-07T17:16:07.660Z",
      "location_count": 2,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01581424"
    },
    {
      "nct_id": "NCT03056794",
      "title": "Natural History and Advanced Genetic Study of Pyruvate Dehydrogenase Complex Deficiencies",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Pyruvate Dehydrogenase Complex Deficiency Disease"
      ],
      "interventions": [
        {
          "name": "No intervention",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "University of Pittsburgh",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 150,
      "start_date": "2015-09",
      "completion_date": "2030-12",
      "has_results": false,
      "last_update_posted_date": "2026-10-05",
      "last_synced_at": "2026-10-07T17:16:07.660Z",
      "location_count": 1,
      "location_summary": "Pittsburgh, Pennsylvania",
      "locations": [
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03056794"
    },
    {
      "nct_id": "NCT01347073",
      "title": "Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Urea Cycle Disorders"
      ],
      "interventions": [
        {
          "name": "HPN-100",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Amgen",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "29 Days",
        "maximum_age": "6 Years",
        "sex": "ALL",
        "summary": "29 Days to 6 Years"
      },
      "enrollment_count": 23,
      "start_date": "2011-07",
      "completion_date": "2013-03",
      "has_results": true,
      "last_update_posted_date": "2024-07-11",
      "last_synced_at": "2026-10-07T17:16:07.660Z",
      "location_count": 8,
      "location_summary": "Los Angeles, California • Washington D.C., District of Columbia • Portland, Maine + 5 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Washington D.C.",
          "state": "District of Columbia"
        },
        {
          "city": "Portland",
          "state": "Maine"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        },
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01347073"
    },
    {
      "nct_id": "NCT04020913",
      "title": "Skeletal Muscle Effects of GH in Boys",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Growth Hormone Deficiency",
        "Idiopathic Short Stature"
      ],
      "interventions": [
        {
          "name": "Somatropin injection",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Nemours Children's Clinic",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "6 Years",
        "maximum_age": "11 Years",
        "sex": "MALE",
        "summary": "6 Years to 11 Years · Male only"
      },
      "enrollment_count": 45,
      "start_date": "2019-07-22",
      "completion_date": "2025-10-30",
      "has_results": false,
      "last_update_posted_date": "2025-11-04",
      "last_synced_at": "2026-10-07T17:16:07.660Z",
      "location_count": 1,
      "location_summary": "Jacksonville, Florida",
      "locations": [
        {
          "city": "Jacksonville",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04020913"
    }
  ]
}