{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=8",
    "query": {
      "condition": "Inborn Errors of Metabolism",
      "page": 8
    },
    "page_size": 10
  },
  "pagination": {
    "page": 8,
    "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=9&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=7&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-14T05:30:28.436Z",
  "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": "NCT04903223",
      "title": "Liver Adiposity Effects on Pediatric Statin",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Cholesterol; Lipidosis"
      ],
      "interventions": [
        {
          "name": "Rosuvastatin 10mg",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Children's Mercy Hospital Kansas City",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "8 Years",
        "maximum_age": "21 Years",
        "sex": "ALL",
        "summary": "8 Years to 21 Years"
      },
      "enrollment_count": 50,
      "start_date": "2021-04-01",
      "completion_date": "2027-03-31",
      "has_results": false,
      "last_update_posted_date": "2026-06-25",
      "last_synced_at": "2026-10-14T05:30:28.436Z",
      "location_count": 1,
      "location_summary": "Kansas City, Missouri",
      "locations": [
        {
          "city": "Kansas City",
          "state": "Missouri"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04903223"
    },
    {
      "nct_id": "NCT04270682",
      "title": "Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "CTX"
      ],
      "interventions": [
        {
          "name": "Blinded CDCA 250 mg TID",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        },
        {
          "name": "Open-Label CDCA 250 mg TID",
          "type": "DRUG"
        },
        {
          "name": "Rescue Medication CDCA 250 mg TID",
          "type": "DRUG"
        },
        {
          "name": "CDCA Weight-Based Dose TID",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Mirum Pharmaceuticals, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Month",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "1 Month and older"
      },
      "enrollment_count": 19,
      "start_date": "2020-01-31",
      "completion_date": "2023-10-04",
      "has_results": true,
      "last_update_posted_date": "2024-10-28",
      "last_synced_at": "2026-10-14T05:30:28.436Z",
      "location_count": 9,
      "location_summary": "Aurora, Colorado • Orlando, Florida • Iowa City, Iowa + 5 more",
      "locations": [
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Orlando",
          "state": "Florida"
        },
        {
          "city": "Iowa City",
          "state": "Iowa"
        },
        {
          "city": "New Orleans",
          "state": "Louisiana"
        },
        {
          "city": "New Orleans",
          "state": "Louisiana"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04270682"
    },
    {
      "nct_id": "NCT03514420",
      "title": "Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Familial Partial Lipodystrophy"
      ],
      "interventions": [
        {
          "name": "AKCEA-ANGPTL3-LRx",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Akcea Therapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 4,
      "start_date": "2018-06-15",
      "completion_date": "2019-08-21",
      "has_results": true,
      "last_update_posted_date": "2021-02-16",
      "last_synced_at": "2026-10-14T05:30:28.436Z",
      "location_count": 1,
      "location_summary": "Ann Arbor, Michigan",
      "locations": [
        {
          "city": "Ann Arbor",
          "state": "Michigan"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03514420"
    },
    {
      "nct_id": "NCT04470713",
      "title": "Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "GM1 Gangliosidosis",
        "GM2 Gangliosidosis",
        "Gaucher Disease, Type 2",
        "Tay-Sachs Disease",
        "AB Variant Gangliosidosis GM2",
        "Sandhoff Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Idorsia Pharmaceuticals Ltd.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "0 Months",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "0 Months and older"
      },
      "enrollment_count": 226,
      "start_date": "2019-07-31",
      "completion_date": "2021-10-30",
      "has_results": false,
      "last_update_posted_date": "2021-11-08",
      "last_synced_at": "2026-10-14T05:30:28.436Z",
      "location_count": 3,
      "location_summary": "Chicago, Illinois • Rochester, Minnesota • Fairfax, Virginia",
      "locations": [
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "Rochester",
          "state": "Minnesota"
        },
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04470713"
    },
    {
      "nct_id": "NCT04174105",
      "title": "Gene Transfer Study in Patients With Late Onset Pompe Disease",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Pompe Disease (Late-onset)"
      ],
      "interventions": [
        {
          "name": "zocaglusagene nuzaparvovec",
          "type": "GENETIC"
        }
      ],
      "intervention_types": [
        "GENETIC"
      ],
      "sponsor": "Astellas Gene Therapies",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "80 Years",
        "sex": "ALL",
        "summary": "18 Years to 80 Years"
      },
      "enrollment_count": 11,
      "start_date": "2020-10-27",
      "completion_date": "2035-02-28",
      "has_results": false,
      "last_update_posted_date": "2026-09-02",
      "last_synced_at": "2026-10-14T05:30:28.436Z",
      "location_count": 3,
      "location_summary": "Orange, California • Palo Alto, California • Salt Lake City, Utah",
      "locations": [
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "Palo Alto",
          "state": "California"
        },
        {
          "city": "Salt Lake City",
          "state": "Utah"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04174105"
    },
    {
      "nct_id": "NCT01275066",
      "title": "A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "MPS IV A"
      ],
      "interventions": [
        {
          "name": "BMN 110 Weekly",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        },
        {
          "name": "BMN 110 Every Other Week",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "BioMarin Pharmaceutical",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "5 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "5 Years and older"
      },
      "enrollment_count": 177,
      "start_date": "2011-02",
      "completion_date": "2012-08",
      "has_results": true,
      "last_update_posted_date": "2014-07-07",
      "last_synced_at": "2026-10-14T05:30:28.436Z",
      "location_count": 6,
      "location_summary": "Oakland, California • Wilmington, Delaware • Washington D.C., District of Columbia + 3 more",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Wilmington",
          "state": "Delaware"
        },
        {
          "city": "Washington D.C.",
          "state": "District of Columbia"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01275066"
    },
    {
      "nct_id": "NCT05797857",
      "title": "Exercise Training in Transthyretin Cardiac Amyloidosis",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "NA"
      ],
      "conditions": [
        "Amyloid Cardiomyopathy",
        "Transthyretin Cardiac Amyloidosis"
      ],
      "interventions": [
        {
          "name": "Exercise Training",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "Brigham and Women's Hospital",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "60 Years",
        "maximum_age": "90 Years",
        "sex": "ALL",
        "summary": "60 Years to 90 Years"
      },
      "enrollment_count": 40,
      "start_date": "2024-07-10",
      "completion_date": "2030-06-30",
      "has_results": false,
      "last_update_posted_date": "2025-03-18",
      "last_synced_at": "2026-10-14T05:30:28.436Z",
      "location_count": 1,
      "location_summary": "Boston, Massachusetts",
      "locations": [
        {
          "city": "Boston",
          "state": "Massachusetts"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05797857"
    },
    {
      "nct_id": "NCT02468570",
      "title": "A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Phenylketonuria"
      ],
      "interventions": [
        {
          "name": "Administration of CANTAB and Subject Global Assessment",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "BioMarin Pharmaceutical",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "70 Years",
        "sex": "ALL",
        "summary": "18 Years to 70 Years"
      },
      "enrollment_count": 9,
      "start_date": "2015-08-05",
      "completion_date": "2017-02-01",
      "has_results": true,
      "last_update_posted_date": "2020-06-04",
      "last_synced_at": "2026-10-14T05:30:28.436Z",
      "location_count": 4,
      "location_summary": "Lexington, Kentucky • Omaha, Nebraska • Oklahoma City, Oklahoma + 1 more",
      "locations": [
        {
          "city": "Lexington",
          "state": "Kentucky"
        },
        {
          "city": "Omaha",
          "state": "Nebraska"
        },
        {
          "city": "Oklahoma City",
          "state": "Oklahoma"
        },
        {
          "city": "Portland",
          "state": "Oregon"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02468570"
    },
    {
      "nct_id": "NCT05952869",
      "title": "A Study of Enlicitide Decanoate (MK-0616 Oral PCSK9 Inhibitor) in Adults With Heterozygous Familial Hypercholesterolemia (MK-0616-017/CORALreef HeFH)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Hypercholesterolemia",
        "Familial Hypercholesterolemia"
      ],
      "interventions": [
        {
          "name": "Enlicitide Decanoate",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Merck Sharp & Dohme LLC",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 303,
      "start_date": "2023-08-08",
      "completion_date": "2025-04-07",
      "has_results": true,
      "last_update_posted_date": "2026-02-24",
      "last_synced_at": "2026-10-14T05:30:28.436Z",
      "location_count": 13,
      "location_summary": "Daphne, Alabama • Boca Raton, Florida • Cutler Bay, Florida + 10 more",
      "locations": [
        {
          "city": "Daphne",
          "state": "Alabama"
        },
        {
          "city": "Boca Raton",
          "state": "Florida"
        },
        {
          "city": "Cutler Bay",
          "state": "Florida"
        },
        {
          "city": "Port Orange",
          "state": "Florida"
        },
        {
          "city": "Winter Park",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05952869"
    },
    {
      "nct_id": "NCT00417612",
      "title": "Effectiveness of Paricalcitol in Reducing Parathyroid Hormone (PTH) Levels in X-linked Hypophosphatemic Rickets",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Hypophosphatemia, Familial",
        "Hyperparathyroidism"
      ],
      "interventions": [
        {
          "name": "Paricalcitol",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "DRUG",
        "OTHER"
      ],
      "sponsor": "Yale University",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "9 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "9 Years and older"
      },
      "enrollment_count": 33,
      "start_date": "2007-01",
      "completion_date": "2012-08",
      "has_results": true,
      "last_update_posted_date": "2020-03-17",
      "last_synced_at": "2026-10-14T05:30:28.436Z",
      "location_count": 1,
      "location_summary": "New Haven, Connecticut",
      "locations": [
        {
          "city": "New Haven",
          "state": "Connecticut"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00417612"
    }
  ]
}