{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=46",
    "query": {
      "condition": "Inborn Errors of Metabolism Disorders",
      "page": 46
    },
    "page_size": 10
  },
  "pagination": {
    "page": 46,
    "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=47&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=45&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-11-15T07:52:02.289Z",
  "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": "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": "2026-11-15T07:52:02.289Z",
      "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": "NCT06910813",
      "title": "DFT383 in Pediatric Participants With Nephropathic Cystinosis",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Nephropathic Cystinosis"
      ],
      "interventions": [
        {
          "name": "DFT383",
          "type": "GENETIC"
        }
      ],
      "intervention_types": [
        "GENETIC"
      ],
      "sponsor": "Novartis Pharmaceuticals",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": "5 Years",
        "sex": "ALL",
        "summary": "2 Years to 5 Years"
      },
      "enrollment_count": 30,
      "start_date": "2025-06-02",
      "completion_date": "2044-05-28",
      "has_results": false,
      "last_update_posted_date": "2026-10-16",
      "last_synced_at": "2026-11-15T07:52:02.289Z",
      "location_count": 6,
      "location_summary": "Phoenix, Arizona • San Diego, California • Stanford, California + 3 more",
      "locations": [
        {
          "city": "Phoenix",
          "state": "Arizona"
        },
        {
          "city": "San Diego",
          "state": "California"
        },
        {
          "city": "Stanford",
          "state": "California"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06910813"
    },
    {
      "nct_id": "NCT02555579",
      "title": "Simplified Diet Approach in Phenylketonuria",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "NA"
      ],
      "conditions": [
        "Phenylketonuria"
      ],
      "interventions": [
        {
          "name": "Dietary Protein Counting and Free Fruits and Vegetables",
          "type": "BEHAVIORAL"
        }
      ],
      "intervention_types": [
        "BEHAVIORAL"
      ],
      "sponsor": "University of Nebraska",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "13 Years",
        "maximum_age": "65 Years",
        "sex": "ALL",
        "summary": "13 Years to 65 Years"
      },
      "enrollment_count": 33,
      "start_date": "2015-10-26",
      "completion_date": "2017-09-20",
      "has_results": false,
      "last_update_posted_date": "2023-09-07",
      "last_synced_at": "2026-11-15T07:52:02.289Z",
      "location_count": 4,
      "location_summary": "Omaha, Nebraska",
      "locations": [
        {
          "city": "Omaha",
          "state": "Nebraska"
        },
        {
          "city": "Omaha",
          "state": "Nebraska"
        },
        {
          "city": "Omaha",
          "state": "Nebraska"
        },
        {
          "city": "Omaha",
          "state": "Nebraska"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02555579"
    },
    {
      "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": "2026-11-15T07:52:02.289Z",
      "location_count": 1,
      "location_summary": "Palo Alto, California",
      "locations": [
        {
          "city": "Palo Alto",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02595489"
    },
    {
      "nct_id": "NCT03002974",
      "title": "A Study to Evaluate Efficacy and Safety of Anakinra in the Treatment of Acute Gouty Arthritis",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Acute Gouty Arthritis"
      ],
      "interventions": [
        {
          "name": "Anakinra 100 mg",
          "type": "DRUG"
        },
        {
          "name": "Triamcinolone Acetonide 40 mg",
          "type": "DRUG"
        },
        {
          "name": "Placebo to Anakinra 100 mg",
          "type": "DRUG"
        },
        {
          "name": "Placebo to Triamcinolone Acetonide 40 mg",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Swedish Orphan Biovitrum",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 165,
      "start_date": "2016-12",
      "completion_date": "2019-08",
      "has_results": true,
      "last_update_posted_date": "2020-07-15",
      "last_synced_at": "2026-11-15T07:52:02.289Z",
      "location_count": 37,
      "location_summary": "Birmingham, Alabama • Gulf Shores, Alabama • Mobile, Alabama + 32 more",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Gulf Shores",
          "state": "Alabama"
        },
        {
          "city": "Mobile",
          "state": "Alabama"
        },
        {
          "city": "Anaheim",
          "state": "California"
        },
        {
          "city": "Colorado Springs",
          "state": "Colorado"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03002974"
    },
    {
      "nct_id": "NCT02171104",
      "title": "MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Mucopolysaccharidosis Disorders",
        "Hurler Syndrome",
        "Hunter Syndrome",
        "Maroteaux Lamy Syndrome",
        "Sly Syndrome",
        "Alpha-Mannosidosis",
        "Fucosidosis",
        "Aspartylglucosaminuria",
        "Glycoprotein Metabolic Disorders",
        "Sphingolipidoses",
        "Recessive Leukodystrophies",
        "Globoid Cell Leukodystrophy",
        "Metachromatic Leukodystrophy",
        "Niemann-Pick B",
        "Niemann-Pick C Subtype 2",
        "Sphingomyelin Deficiency",
        "Peroxisomal Disorders",
        "Adrenoleukodystrophy With Cerebral Involvement",
        "Zellweger Syndrome",
        "Neonatal Adrenoleukodystrophy",
        "Infantile Refsum Disease",
        "Acyl-CoA Oxidase Deficiency",
        "D-Bifunctional Enzyme Deficiency",
        "Multifunctional Enzyme Deficiency",
        "Alpha-methylacyl-CoA Racmase Deficiency",
        "Mitochondrial Neurogastrointestingal Encephalopathy",
        "Severe Osteopetrosis",
        "Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation)",
        "Inherited Metabolic Disorders"
      ],
      "interventions": [
        {
          "name": "Stem Cell Transplantation",
          "type": "BIOLOGICAL"
        },
        {
          "name": "IMD Preparative Regimen",
          "type": "DRUG"
        },
        {
          "name": "Osteopetrosis Only Preparative Regimen",
          "type": "DRUG"
        },
        {
          "name": "Osteopetrosis Haploidentical Only Preparative Regimen",
          "type": "DRUG"
        },
        {
          "name": "cALD SR-A (Standard-Risk, Regimen A)",
          "type": "DRUG"
        },
        {
          "name": "cALD SR-B (Standard-Risk, Regimen B)",
          "type": "DRUG"
        },
        {
          "name": "cALD HR-D (High-Risk, Regimen C)",
          "type": "DRUG"
        },
        {
          "name": "cALD HR-D (High-Risk, Regimen D)",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL",
        "DRUG"
      ],
      "sponsor": "Masonic Cancer Center, University of Minnesota",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "55 Years",
        "sex": "ALL",
        "summary": "Up to 55 Years"
      },
      "enrollment_count": 149,
      "start_date": "2014-07-10",
      "completion_date": "2031-07-14",
      "has_results": false,
      "last_update_posted_date": "2026-11-03",
      "last_synced_at": "2026-11-15T07:52:02.289Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02171104"
    },
    {
      "nct_id": "NCT00819585",
      "title": "A Study of the Efficacy of Canakinumab in Prevention of Acute Flares in Chronic Gout Patients Initiating Allopurinol Therapy (Core Study) and a Long-term Study of the Efficacy and Safety of Canakinumab in Patients With Gout (Extension Study)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Gout"
      ],
      "interventions": [
        {
          "name": "Canakinumab",
          "type": "DRUG"
        },
        {
          "name": "Colchicine",
          "type": "DRUG"
        },
        {
          "name": "Allopurinol",
          "type": "DRUG"
        },
        {
          "name": "Placebo Matching Canakinumab",
          "type": "DRUG"
        },
        {
          "name": "Placebo Matching Colchicine",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Novartis Pharmaceuticals",
      "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": 432,
      "start_date": "2008-12",
      "completion_date": "2010-08",
      "has_results": true,
      "last_update_posted_date": "2018-07-17",
      "last_synced_at": "2026-11-15T07:52:02.289Z",
      "location_count": 19,
      "location_summary": "Huntington Beach, California • San Diego, California • Jupiter, Florida + 16 more",
      "locations": [
        {
          "city": "Huntington Beach",
          "state": "California"
        },
        {
          "city": "San Diego",
          "state": "California"
        },
        {
          "city": "Jupiter",
          "state": "Florida"
        },
        {
          "city": "Honolulu",
          "state": "Hawaii"
        },
        {
          "city": "Overland Park",
          "state": "Kansas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00819585"
    },
    {
      "nct_id": "NCT00001410",
      "title": "PEG-Glucocerebrosidase for the Treatment of Gaucher Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Gaucher's Disease"
      ],
      "interventions": [
        {
          "name": "Lysodase",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "National Institute of Mental Health (NIMH)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 18,
      "start_date": "1993-10",
      "completion_date": "2001-12",
      "has_results": false,
      "last_update_posted_date": "2008-03-04",
      "last_synced_at": "2026-11-15T07:52:02.289Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00001410"
    },
    {
      "nct_id": "NCT01358474",
      "title": "Detecting Dopaminergic Deficits in Individuals At-risk for Parkinsonism",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Parkinson Disease",
        "Gaucher Disease",
        "Idiopathic Rapid Eye Movement Sleep Disorder"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "University of Minnesota",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "85 Years",
        "sex": "ALL",
        "summary": "18 Years to 85 Years"
      },
      "enrollment_count": 56,
      "start_date": "2011-07",
      "completion_date": "2018-12",
      "has_results": false,
      "last_update_posted_date": "2019-04-18",
      "last_synced_at": "2026-11-15T07:52:02.289Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01358474"
    },
    {
      "nct_id": "NCT00081497",
      "title": "A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE4"
      ],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [
        {
          "name": "agalsidase beta",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Genzyme, a Sanofi Company",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "16 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "16 Years and older"
      },
      "enrollment_count": 67,
      "start_date": "2004-01",
      "completion_date": "2005-09",
      "has_results": true,
      "last_update_posted_date": "2015-04-02",
      "last_synced_at": "2026-11-15T07:52:02.289Z",
      "location_count": 18,
      "location_summary": "Birmingham, Alabama • Los Angeles, California • San Francisco, California + 15 more",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "San Francisco",
          "state": "California"
        },
        {
          "city": "West Hartford",
          "state": "Connecticut"
        },
        {
          "city": "Coral Springs",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00081497"
    }
  ]
}