{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=45",
    "query": {
      "condition": "Inborn Errors of Metabolism",
      "page": 45
    },
    "page_size": 10
  },
  "pagination": {
    "page": 45,
    "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=46&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=44&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-18T14:55:53.067Z",
  "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": "NCT00075244",
      "title": "Alternative Dosing and Regimen of Replagal to Treat Fabry Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [
        {
          "name": "Replagal",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "National Institute of Neurological Disorders and Stroke (NINDS)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "MALE",
        "summary": "Male only"
      },
      "enrollment_count": 25,
      "start_date": "2004-01",
      "completion_date": "2005-11",
      "has_results": false,
      "last_update_posted_date": "2008-03-04",
      "last_synced_at": "2026-10-18T14:55:53.067Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00075244"
    },
    {
      "nct_id": "NCT04574830",
      "title": "Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Glycogen Storage Disease Type III (GSD III)",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Glycogen Storage Disease Type III"
      ],
      "interventions": [
        {
          "name": "No Intervention",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "Ultragenyx Pharmaceutical Inc",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "5 Years",
        "maximum_age": "65 Years",
        "sex": "ALL",
        "summary": "5 Years to 65 Years"
      },
      "enrollment_count": 18,
      "start_date": "2020-11-23",
      "completion_date": "2022-06-30",
      "has_results": false,
      "last_update_posted_date": "2022-09-13",
      "last_synced_at": "2026-10-18T14:55:53.067Z",
      "location_count": 4,
      "location_summary": "Irvine, California • Orange, California • Aurora, Colorado + 1 more",
      "locations": [
        {
          "city": "Irvine",
          "state": "California"
        },
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Houston",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04574830"
    },
    {
      "nct_id": "NCT07582484",
      "title": "Gene Therapy Trial for CLN6 Batten Disease",
      "overall_status": "NOT_YET_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "CLN6",
        "Batten Disease",
        "Batten's Disease",
        "Neuronal Ceroid Lipofuscinosis CLN6",
        "Neuronal Ceroid Lipofuscinosis"
      ],
      "interventions": [
        {
          "name": "scAAV9.CB.CLN6 (dose: 1.5E14 vector genomes)",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "The Charlotte and Gwenyth Gray Foundation",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "4 Months",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "4 Months and older"
      },
      "enrollment_count": 12,
      "start_date": "2026-08",
      "completion_date": "2028-08",
      "has_results": false,
      "last_update_posted_date": "2026-05-14",
      "last_synced_at": "2026-10-18T14:55:53.067Z",
      "location_count": 1,
      "location_summary": "La Jolla, California",
      "locations": [
        {
          "city": "La Jolla",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07582484"
    },
    {
      "nct_id": "NCT01721733",
      "title": "Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Leigh Syndrome"
      ],
      "interventions": [
        {
          "name": "Placebo",
          "type": "DRUG"
        },
        {
          "name": "EPI-743 15 mg/kg",
          "type": "DRUG"
        },
        {
          "name": "EPI-743 5 mg/kg",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "PTC Therapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Years",
        "maximum_age": "17 Years",
        "sex": "ALL",
        "summary": "6 Years to 17 Years"
      },
      "enrollment_count": 35,
      "start_date": "2012-10-31",
      "completion_date": "2015-05-31",
      "has_results": false,
      "last_update_posted_date": "2020-08-31",
      "last_synced_at": "2026-10-18T14:55:53.067Z",
      "location_count": 4,
      "location_summary": "Palo Alto, California • Akron, Ohio • Houston, Texas + 1 more",
      "locations": [
        {
          "city": "Palo Alto",
          "state": "California"
        },
        {
          "city": "Akron",
          "state": "Ohio"
        },
        {
          "city": "Houston",
          "state": "Texas"
        },
        {
          "city": "Seattle",
          "state": "Washington"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01721733"
    },
    {
      "nct_id": "NCT03119558",
      "title": "PET/MRI Evaluation of Cardiac Amyloid",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "EARLY_PHASE1"
      ],
      "conditions": [
        "Cardiac Amyloidosis"
      ],
      "interventions": [
        {
          "name": "18F-Florbetaben (Neuraceq®) PET/MRI",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Stanford University",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 9,
      "start_date": "2016-05-26",
      "completion_date": "2018-08-28",
      "has_results": false,
      "last_update_posted_date": "2019-07-17",
      "last_synced_at": "2026-10-18T14:55:53.067Z",
      "location_count": 1,
      "location_summary": "Stanford, California",
      "locations": [
        {
          "city": "Stanford",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03119558"
    },
    {
      "nct_id": "NCT02605603",
      "title": "SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease",
      "overall_status": "UNKNOWN",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Gaucher Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Lysosomal and Rare Disorders Research and Treatment Center, Inc.",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 30,
      "start_date": "2015-05",
      "completion_date": "2020-05",
      "has_results": false,
      "last_update_posted_date": "2019-08-13",
      "last_synced_at": "2026-10-18T14:55:53.067Z",
      "location_count": 1,
      "location_summary": "Fairfax, Virginia",
      "locations": [
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02605603"
    },
    {
      "nct_id": "NCT00484991",
      "title": "Sapropterin Expanded Access Program",
      "overall_status": "APPROVED_FOR_MARKETING",
      "study_type": "EXPANDED_ACCESS",
      "phases": [],
      "conditions": [
        "Phenylketonuria"
      ],
      "interventions": [
        {
          "name": "Sapropterin dihydrochloride",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "BioMarin Pharmaceutical",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": null,
      "eligibility": {
        "minimum_age": "9 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "9 Years and older"
      },
      "enrollment_count": null,
      "start_date": null,
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2008-04-15",
      "last_synced_at": "2026-10-18T14:55:53.067Z",
      "location_count": 36,
      "location_summary": "Los Angeles, California • Orange, California • San Diego, California + 33 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "San Diego",
          "state": "California"
        },
        {
          "city": "Stanford",
          "state": "California"
        },
        {
          "city": "Denver",
          "state": "Colorado"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00484991"
    },
    {
      "nct_id": "NCT00927810",
      "title": "Long Term Study of Canakinumab (ACZ885) in Patients With Gout",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Gouty Arthritis"
      ],
      "interventions": [
        {
          "name": "Canakinumab",
          "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": 341,
      "start_date": "2009-06-05",
      "completion_date": "2010-08-04",
      "has_results": true,
      "last_update_posted_date": "2021-07-02",
      "last_synced_at": "2026-10-18T14:55:53.067Z",
      "location_count": 1,
      "location_summary": "Seattle, Washington",
      "locations": [
        {
          "city": "Seattle",
          "state": "Washington"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00927810"
    },
    {
      "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": "2026-10-18T14:55:53.067Z",
      "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": "NCT04776161",
      "title": "Medication Adherence Patterns in Rheumatic Diseases: A Behavioral Trial",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "NA"
      ],
      "conditions": [
        "Rheumatic Diseases",
        "Gout",
        "Adherence, Medication",
        "Lupus Erythematosus, Systemic"
      ],
      "interventions": [
        {
          "name": "Cue-Reward Intervention",
          "type": "BEHAVIORAL"
        },
        {
          "name": "Cue-Reward Intervention with possible intensification",
          "type": "BEHAVIORAL"
        }
      ],
      "intervention_types": [
        "BEHAVIORAL"
      ],
      "sponsor": "Brigham and Women's Hospital",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "85 Years",
        "sex": "ALL",
        "summary": "18 Years to 85 Years"
      },
      "enrollment_count": 62,
      "start_date": "2021-08-02",
      "completion_date": "2022-11-10",
      "has_results": true,
      "last_update_posted_date": "2023-10-04",
      "last_synced_at": "2026-10-18T14:55:53.067Z",
      "location_count": 1,
      "location_summary": "Boston, Massachusetts",
      "locations": [
        {
          "city": "Boston",
          "state": "Massachusetts"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04776161"
    }
  ]
}