{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=39",
    "query": {
      "condition": "Inborn Errors of Metabolism Disorders",
      "page": 39
    },
    "page_size": 10
  },
  "pagination": {
    "page": 39,
    "page_size": 10,
    "total_count": 1513,
    "total_pages": 152,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=40&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=38&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-11-05T23:03:01.954Z",
  "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": "NCT00995618",
      "title": "Study of Tranilast Alone or in Combination With Febuxostat in Patients With Hyperuricemia",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Gout",
        "Hyperuricemia"
      ],
      "interventions": [
        {
          "name": "Tranilast",
          "type": "DRUG"
        },
        {
          "name": "Febuxostat",
          "type": "DRUG"
        },
        {
          "name": "Combination - Tranilast and Febuxostat",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Nuon Therapeutics, Inc.",
      "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": 24,
      "start_date": "2009-09",
      "completion_date": "2010-04",
      "has_results": false,
      "last_update_posted_date": "2011-01-06",
      "last_synced_at": "2026-11-05T23:03:01.954Z",
      "location_count": 3,
      "location_summary": "Honolulu, Hawaii • Evansville, Indiana • Dallas, Texas",
      "locations": [
        {
          "city": "Honolulu",
          "state": "Hawaii"
        },
        {
          "city": "Evansville",
          "state": "Indiana"
        },
        {
          "city": "Dallas",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00995618"
    },
    {
      "nct_id": "NCT07478172",
      "title": "Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "NA"
      ],
      "conditions": [
        "Neuromuscular Diseases (NMD)",
        "Amyotrophic Lateral Sclerosis",
        "Myasthenia Gravis",
        "Lambert-eaton Myasthenic Syndrome",
        "Primary Lateral Sclerosis",
        "Spinal Muscular Atrophy",
        "Charcot Marie Tooth Disease (CMT)",
        "Fascioscapulohumeral Muscular Dystrophy",
        "Inclusion Body Myositis",
        "Mitochondrial Myopathy",
        "Nemaline Myopathy",
        "Centronuclear Myopathy",
        "Postpolio Syndrome",
        "Pompe Disease (Late-onset)",
        "Chronic Inflammatory Demyelinating Polyneuropathy",
        "Hereditary Spastic Paraplegia",
        "Postural Orthostatic Tachycardia Syndrome (POTS)",
        "Progressive Muscular Atrophy"
      ],
      "interventions": [
        {
          "name": "Whole-body Electrical Muscle Stimulation Exercise",
          "type": "DEVICE"
        }
      ],
      "intervention_types": [
        "DEVICE"
      ],
      "sponsor": "University of Missouri-Columbia",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 50,
      "start_date": "2026-03-10",
      "completion_date": "2031-01-07",
      "has_results": false,
      "last_update_posted_date": "2026-03-17",
      "last_synced_at": "2026-11-05T23:03:01.954Z",
      "location_count": 1,
      "location_summary": "Columbia, Missouri",
      "locations": [
        {
          "city": "Columbia",
          "state": "Missouri"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07478172"
    },
    {
      "nct_id": "NCT02791230",
      "title": "Long-term Safety of Tafamidis in Subjects With Transthyretin Cardiomyopathy",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Transthyretin (TTR) Amyloid Cardiomyopathy"
      ],
      "interventions": [
        {
          "name": "Tafamidis",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Pfizer",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 1733,
      "start_date": "2016-06-13",
      "completion_date": "2023-11-02",
      "has_results": true,
      "last_update_posted_date": "2025-02-03",
      "last_synced_at": "2026-11-05T23:03:01.954Z",
      "location_count": 41,
      "location_summary": "Birmingham, Alabama • Beverly Hills, California • La Jolla, California + 20 more",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Beverly Hills",
          "state": "California"
        },
        {
          "city": "Beverly Hills",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02791230"
    },
    {
      "nct_id": "NCT04713475",
      "title": "Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "GM1 Gangliosidosis",
        "GM1 Gangliosidosis, Type I",
        "GM1 Gangliosidosis, Type 2",
        "Beta-Galactosidase-1 (GLB1) Deficiency"
      ],
      "interventions": [
        {
          "name": "PBGM01",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Gemma Biotherapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Month",
        "maximum_age": "24 Months",
        "sex": "ALL",
        "summary": "1 Month to 24 Months"
      },
      "enrollment_count": 26,
      "start_date": "2021-03-17",
      "completion_date": "2029-02",
      "has_results": false,
      "last_update_posted_date": "2026-06-30",
      "last_synced_at": "2026-11-05T23:03:01.954Z",
      "location_count": 4,
      "location_summary": "Oakland, California • Minneapolis, Minnesota • New Brunswick, New Jersey + 1 more",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        },
        {
          "city": "New Brunswick",
          "state": "New Jersey"
        },
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04713475"
    },
    {
      "nct_id": "NCT04138277",
      "title": "A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Pompe Disease (Late-onset)"
      ],
      "interventions": [
        {
          "name": "AT2221",
          "type": "DRUG"
        },
        {
          "name": "ATB200",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "DRUG",
        "BIOLOGICAL"
      ],
      "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": 119,
      "start_date": "2019-12-18",
      "completion_date": "2024-12-31",
      "has_results": true,
      "last_update_posted_date": "2026-03-05",
      "last_synced_at": "2026-11-05T23:03:01.954Z",
      "location_count": 18,
      "location_summary": "Little Rock, Arkansas • Irvine, California • Gainesville, Florida + 14 more",
      "locations": [
        {
          "city": "Little Rock",
          "state": "Arkansas"
        },
        {
          "city": "Irvine",
          "state": "California"
        },
        {
          "city": "Gainesville",
          "state": "Florida"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Indianapolis",
          "state": "Indiana"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04138277"
    },
    {
      "nct_id": "NCT00136981",
      "title": "Carotid B-Mode Ultrasound Study to Compare Anti-Atherosclerotic Effect of Torcetrpib/Atorvastatin to Atorvastatin Alone.",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Hypercholesterolemia, Familial",
        "Hyperlipidemia"
      ],
      "interventions": [
        {
          "name": "torcetrapib/atorvastatin",
          "type": "DRUG"
        },
        {
          "name": "atorvastatin",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Pfizer",
      "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": 800,
      "start_date": "2003-12",
      "completion_date": "2006-11",
      "has_results": false,
      "last_update_posted_date": "2015-04-22",
      "last_synced_at": "2026-11-05T23:03:01.954Z",
      "location_count": 7,
      "location_summary": "Hartford, Connecticut • Boston, Massachusetts • Minneapolis, Minnesota + 4 more",
      "locations": [
        {
          "city": "Hartford",
          "state": "Connecticut"
        },
        {
          "city": "Boston",
          "state": "Massachusetts"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Houston",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00136981"
    },
    {
      "nct_id": "NCT05146830",
      "title": "A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04",
      "overall_status": "ENROLLING_BY_INVITATION",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Cystinosis"
      ],
      "interventions": [
        {
          "name": "Safety and Efficacy Assessments",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "Stephanie Cherqui",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "14 Years",
        "maximum_age": "50 Years",
        "sex": "ALL",
        "summary": "14 Years to 50 Years"
      },
      "enrollment_count": 50,
      "start_date": "2022-11-14",
      "completion_date": "2036-11-30",
      "has_results": false,
      "last_update_posted_date": "2024-07-03",
      "last_synced_at": "2026-11-05T23:03:01.954Z",
      "location_count": 1,
      "location_summary": "La Jolla, California",
      "locations": [
        {
          "city": "La Jolla",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05146830"
    },
    {
      "nct_id": "NCT02536911",
      "title": "A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Gaucher Disease"
      ],
      "interventions": [
        {
          "name": "eliglustat",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Genzyme, a Sanofi Company",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "79 Years",
        "sex": "ALL",
        "summary": "18 Years to 79 Years"
      },
      "enrollment_count": 24,
      "start_date": "2015-09",
      "completion_date": "2016-12",
      "has_results": false,
      "last_update_posted_date": "2017-02-10",
      "last_synced_at": "2026-11-05T23:03:01.954Z",
      "location_count": 2,
      "location_summary": "Miami, Florida • Knoxville, Tennessee",
      "locations": [
        {
          "city": "Miami",
          "state": "Florida"
        },
        {
          "city": "Knoxville",
          "state": "Tennessee"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02536911"
    },
    {
      "nct_id": "NCT00272792",
      "title": "Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Phenylketonurias"
      ],
      "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": "4 Years",
        "maximum_age": "12 Years",
        "sex": "ALL",
        "summary": "4 Years to 12 Years"
      },
      "enrollment_count": 45,
      "start_date": "2006-02",
      "completion_date": "2006-11",
      "has_results": true,
      "last_update_posted_date": "2015-08-13",
      "last_synced_at": "2026-11-05T23:03:01.954Z",
      "location_count": 9,
      "location_summary": "Los Angeles, California • Oakland, California • Sacramento, California + 6 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Sacramento",
          "state": "California"
        },
        {
          "city": "San Jose",
          "state": "California"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00272792"
    },
    {
      "nct_id": "NCT03921541",
      "title": "Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Arginase I Deficiency",
        "Hyperargininemia"
      ],
      "interventions": [
        {
          "name": "Pegzilarginase",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Immedica Pharma AB",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "2 Years and older"
      },
      "enrollment_count": 32,
      "start_date": "2019-05-01",
      "completion_date": "2023-02-01",
      "has_results": true,
      "last_update_posted_date": "2026-08-21",
      "last_synced_at": "2026-11-05T23:03:01.954Z",
      "location_count": 17,
      "location_summary": "Rogers, Arkansas • Orange, California • Stanford, California + 14 more",
      "locations": [
        {
          "city": "Rogers",
          "state": "Arkansas"
        },
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "Stanford",
          "state": "California"
        },
        {
          "city": "Washington D.C.",
          "state": "District of Columbia"
        },
        {
          "city": "Gainesville",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03921541"
    }
  ]
}