{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=47",
    "query": {
      "condition": "Inborn Errors of Metabolism Disorders",
      "page": 47
    },
    "page_size": 10
  },
  "pagination": {
    "page": 47,
    "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=48&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=46&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-11-15T10:13:59.850Z",
  "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": "NCT04201418",
      "title": "A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Hereditary Transthyretin-mediated (ATTRv) Amyloidosis",
        "Polyneuropathy"
      ],
      "interventions": [
        {
          "name": "Patisiran",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Alnylam Pharmaceuticals",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 67,
      "start_date": "2019-12-18",
      "completion_date": "2022-05-24",
      "has_results": false,
      "last_update_posted_date": "2022-06-06",
      "last_synced_at": "2026-11-15T10:13:59.850Z",
      "location_count": 27,
      "location_summary": "Los Angeles, California • San Diego, California • Aurora, Colorado + 22 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "San Diego",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Farmington",
          "state": "Connecticut"
        },
        {
          "city": "Jacksonville",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04201418"
    },
    {
      "nct_id": "NCT02616484",
      "title": "Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Pyruvate Dehydrogenase Complex Deficiency"
      ],
      "interventions": [
        {
          "name": "Dichloroacetate (DCA)",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "OTHER"
        },
        {
          "name": "Genotype",
          "type": "GENETIC"
        }
      ],
      "intervention_types": [
        "DRUG",
        "OTHER",
        "GENETIC"
      ],
      "sponsor": "Saol Therapeutics Inc",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Months",
        "maximum_age": "17 Years",
        "sex": "ALL",
        "summary": "6 Months to 17 Years"
      },
      "enrollment_count": 34,
      "start_date": "2020-07-14",
      "completion_date": "2025-08-27",
      "has_results": false,
      "last_update_posted_date": "2025-05-04",
      "last_synced_at": "2026-11-15T10:13:59.850Z",
      "location_count": 10,
      "location_summary": "Orange, California • Stanford, California • Washington D.C., District of Columbia + 7 more",
      "locations": [
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "Stanford",
          "state": "California"
        },
        {
          "city": "Washington D.C.",
          "state": "District of Columbia"
        },
        {
          "city": "Gainesville",
          "state": "Florida"
        },
        {
          "city": "Cleveland",
          "state": "Ohio"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02616484"
    },
    {
      "nct_id": "NCT00004443",
      "title": "Study of NTBC for Tyrosinemia I",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "NA"
      ],
      "conditions": [
        "Tyrosinemia I"
      ],
      "interventions": [
        {
          "name": "NTBC",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "University of Washington",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "17 Years",
        "sex": "ALL",
        "summary": "Up to 17 Years"
      },
      "enrollment_count": 20,
      "start_date": "1998-10",
      "completion_date": "2006-09",
      "has_results": false,
      "last_update_posted_date": "2015-03-25",
      "last_synced_at": "2026-11-15T10:13:59.850Z",
      "location_count": 2,
      "location_summary": "Portland, Oregon • Seattle, Washington",
      "locations": [
        {
          "city": "Portland",
          "state": "Oregon"
        },
        {
          "city": "Seattle",
          "state": "Washington"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00004443"
    },
    {
      "nct_id": "NCT02000310",
      "title": "Molecular and Cellular Mechanisms of Lysosomal Storage Diseases",
      "overall_status": "UNKNOWN",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Lysosomal Storage Disorders"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "O & O Alpan LLC",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "1 Day",
        "maximum_age": "100 Years",
        "sex": "ALL",
        "summary": "1 Day to 100 Years"
      },
      "enrollment_count": 80,
      "start_date": "2013-11",
      "completion_date": "2022-12",
      "has_results": false,
      "last_update_posted_date": "2021-02-23",
      "last_synced_at": "2026-11-15T10:13:59.850Z",
      "location_count": 1,
      "location_summary": "Fairfax, Virginia",
      "locations": [
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02000310"
    },
    {
      "nct_id": "NCT06247085",
      "title": "Pegtibatinase as a Treatment for Participants With Classical Homocystinuria (HCU) Receiving Standard of Care Treatment",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Homocystinuria"
      ],
      "interventions": [
        {
          "name": "Pegtibatinase",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "DRUG",
        "OTHER"
      ],
      "sponsor": "Travere Therapeutics, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "12 Years",
        "maximum_age": "65 Years",
        "sex": "ALL",
        "summary": "12 Years to 65 Years"
      },
      "enrollment_count": 70,
      "start_date": "2023-12-28",
      "completion_date": "2027-09",
      "has_results": false,
      "last_update_posted_date": "2026-11-09",
      "last_synced_at": "2026-11-15T10:13:59.850Z",
      "location_count": 13,
      "location_summary": "Phoenix, Arizona • New Haven, Connecticut • Atlanta, Georgia + 10 more",
      "locations": [
        {
          "city": "Phoenix",
          "state": "Arizona"
        },
        {
          "city": "New Haven",
          "state": "Connecticut"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "Portland",
          "state": "Maine"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06247085"
    },
    {
      "nct_id": "NCT00795197",
      "title": "A Screening Study Evaluating Disease Status of Gaucher Type I Patients",
      "overall_status": "WITHDRAWN",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Gaucher Disease, Type 1"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Genzyme, a Sanofi Company",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "16 Years",
        "maximum_age": "65 Years",
        "sex": "ALL",
        "summary": "16 Years to 65 Years"
      },
      "enrollment_count": 0,
      "start_date": "2008-12",
      "completion_date": "2009-12",
      "has_results": false,
      "last_update_posted_date": "2015-05-08",
      "last_synced_at": "2026-11-15T10:13:59.850Z",
      "location_count": 1,
      "location_summary": "New York, New York",
      "locations": [
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00795197"
    },
    {
      "nct_id": "NCT04093349",
      "title": "A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Pompe Disease",
        "Pompe Disease (Late-onset)",
        "Glycogen Storage Disease Type 2",
        "Glycogen Storage Disease Type II",
        "LOPD",
        "Lysosomal Storage Diseases",
        "Acid Maltase Deficiency"
      ],
      "interventions": [
        {
          "name": "SPK-3006",
          "type": "GENETIC"
        }
      ],
      "intervention_types": [
        "GENETIC"
      ],
      "sponsor": "Spark Therapeutics, Inc.",
      "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": "2020-10-01",
      "completion_date": "2032-04",
      "has_results": false,
      "last_update_posted_date": "2024-11-27",
      "last_synced_at": "2026-11-15T10:13:59.850Z",
      "location_count": 10,
      "location_summary": "Phoenix, Arizona • Orange, California • Atlanta, Georgia + 7 more",
      "locations": [
        {
          "city": "Phoenix",
          "state": "Arizona"
        },
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Kansas City",
          "state": "Kansas"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04093349"
    },
    {
      "nct_id": "NCT01509768",
      "title": "Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Sanfilippo Syndrome Type B"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Shire",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Year",
        "maximum_age": "10 Years",
        "sex": "ALL",
        "summary": "1 Year to 10 Years"
      },
      "enrollment_count": 19,
      "start_date": "2012-04-09",
      "completion_date": "2013-10-10",
      "has_results": false,
      "last_update_posted_date": "2021-03-17",
      "last_synced_at": "2026-11-15T10:13:59.850Z",
      "location_count": 2,
      "location_summary": "Oakland, California • Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01509768"
    },
    {
      "nct_id": "NCT06410976",
      "title": "Prospective Clinical Assessment Study in Children With Hypochondroplasia",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Hypochondroplasia"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "QED Therapeutics, a BridgeBio company",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "30 Months",
        "maximum_age": "16 Years",
        "sex": "ALL",
        "summary": "30 Months to 16 Years"
      },
      "enrollment_count": 150,
      "start_date": "2024-06-05",
      "completion_date": "2026-10",
      "has_results": false,
      "last_update_posted_date": "2026-07-16",
      "last_synced_at": "2026-11-15T10:13:59.850Z",
      "location_count": 8,
      "location_summary": "Oakland, California • Aurora, Colorado • Washington D.C., District of Columbia + 5 more",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Washington D.C.",
          "state": "District of Columbia"
        },
        {
          "city": "Baltimore",
          "state": "Maryland"
        },
        {
          "city": "Columbia",
          "state": "Missouri"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06410976"
    },
    {
      "nct_id": "NCT06193304",
      "title": "A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Gaucher's Disease"
      ],
      "interventions": [
        {
          "name": "Eliglustat",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Sanofi",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "55 Years",
        "sex": "ALL",
        "summary": "18 Years to 55 Years"
      },
      "enrollment_count": 6,
      "start_date": "2014-08-25",
      "completion_date": "2014-09-21",
      "has_results": false,
      "last_update_posted_date": "2024-01-05",
      "last_synced_at": "2026-11-15T10:13:59.850Z",
      "location_count": 1,
      "location_summary": "Evansville, Indiana",
      "locations": [
        {
          "city": "Evansville",
          "state": "Indiana"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06193304"
    }
  ]
}