{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=43",
    "query": {
      "condition": "Inborn Errors of Metabolism",
      "page": 43
    },
    "page_size": 10
  },
  "pagination": {
    "page": 43,
    "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=44&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=42&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-18T03:16:42.778Z",
  "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": "NCT01161576",
      "title": "Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Batten Disease",
        "Late-Infantile Neuronal Ceroid Lipofuscinosis"
      ],
      "interventions": [
        {
          "name": "AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies",
          "type": "BIOLOGICAL"
        },
        {
          "name": "AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Weill Medical College of Cornell University",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": "18 Years",
        "sex": "ALL",
        "summary": "2 Years to 18 Years"
      },
      "enrollment_count": 12,
      "start_date": "2010-08-19",
      "completion_date": "2020-12-31",
      "has_results": false,
      "last_update_posted_date": "2021-02-02",
      "last_synced_at": "2026-10-18T03:16:42.778Z",
      "location_count": 1,
      "location_summary": "New York, New York",
      "locations": [
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01161576"
    },
    {
      "nct_id": "NCT02437253",
      "title": "Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Mucopolysaccharidosis Type I",
        "Mucopolysaccharidosis Type II",
        "Mucopolysaccharidosis Type VI"
      ],
      "interventions": [
        {
          "name": "Adalimumab",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "DRUG",
        "OTHER"
      ],
      "sponsor": "Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "5 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "5 Years and older"
      },
      "enrollment_count": 2,
      "start_date": "2015-05",
      "completion_date": "2016-07",
      "has_results": true,
      "last_update_posted_date": "2017-09-15",
      "last_synced_at": "2026-10-18T03:16:42.778Z",
      "location_count": 1,
      "location_summary": "Torrance, California",
      "locations": [
        {
          "city": "Torrance",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02437253"
    },
    {
      "nct_id": "NCT03454893",
      "title": "Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [
        {
          "name": "AVR-RD-01",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "AVROBIO",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "16 Years",
        "maximum_age": "50 Years",
        "sex": "MALE",
        "summary": "16 Years to 50 Years · Male only"
      },
      "enrollment_count": 15,
      "start_date": "2018-02-21",
      "completion_date": "2022-03-14",
      "has_results": true,
      "last_update_posted_date": "2024-01-05",
      "last_synced_at": "2026-10-18T03:16:42.778Z",
      "location_count": 2,
      "location_summary": "Hackensack, New Jersey • Pittsburgh, Pennsylvania",
      "locations": [
        {
          "city": "Hackensack",
          "state": "New Jersey"
        },
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03454893"
    },
    {
      "nct_id": "NCT01428258",
      "title": "Phase 2 Study of Glycomacropeptide Versus Amino Acid Diet for Management of Phenylketonuria",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "NA"
      ],
      "conditions": [
        "Phenylketonuria"
      ],
      "interventions": [
        {
          "name": "GMP Diet/GMP Medical Foods",
          "type": "OTHER"
        },
        {
          "name": "AA Diet/AA Medical Foods",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "University of Wisconsin, Madison",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "12 Years",
        "maximum_age": "45 Years",
        "sex": "ALL",
        "summary": "12 Years to 45 Years"
      },
      "enrollment_count": 32,
      "start_date": "2011-09",
      "completion_date": "2016-05",
      "has_results": true,
      "last_update_posted_date": "2018-08-24",
      "last_synced_at": "2026-10-18T03:16:42.778Z",
      "location_count": 2,
      "location_summary": "Boston, Massachusetts • Madison, Wisconsin",
      "locations": [
        {
          "city": "Boston",
          "state": "Massachusetts"
        },
        {
          "city": "Madison",
          "state": "Wisconsin"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01428258"
    },
    {
      "nct_id": "NCT05430191",
      "title": "Equitable Implementation of Cascade Screening for Familial Hypercholesterolemia",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Familial Hypercholesterolemia"
      ],
      "interventions": [
        {
          "name": "Cascade Screening",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "Northwestern University",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 22,
      "start_date": "2022-07-20",
      "completion_date": "2023-06-20",
      "has_results": false,
      "last_update_posted_date": "2023-09-29",
      "last_synced_at": "2026-10-18T03:16:42.778Z",
      "location_count": 1,
      "location_summary": "Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05430191"
    },
    {
      "nct_id": "NCT06340685",
      "title": "Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Pyruvate Dehydrogenase Complex Deficiency"
      ],
      "interventions": [
        {
          "name": "Triheptanoin",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Jirair Krikor Bedoyan",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Year",
        "maximum_age": "17 Years",
        "sex": "ALL",
        "summary": "1 Year to 17 Years"
      },
      "enrollment_count": 6,
      "start_date": "2024-07-11",
      "completion_date": "2029-06-30",
      "has_results": false,
      "last_update_posted_date": "2026-02-17",
      "last_synced_at": "2026-10-18T03:16:42.778Z",
      "location_count": 1,
      "location_summary": "Pittsburgh, Pennsylvania",
      "locations": [
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06340685"
    },
    {
      "nct_id": "NCT04829435",
      "title": "Study 102: Multiple Ascending Dose Study of ALLN-346 (Engineered Urate Oxidase)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Hyperuricemia",
        "Gout"
      ],
      "interventions": [
        {
          "name": "ALLN-346",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Allena Pharmaceuticals",
      "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": 18,
      "start_date": "2021-04-21",
      "completion_date": "2021-06-01",
      "has_results": false,
      "last_update_posted_date": "2021-07-01",
      "last_synced_at": "2026-10-18T03:16:42.778Z",
      "location_count": 1,
      "location_summary": "Cincinnati, Ohio",
      "locations": [
        {
          "city": "Cincinnati",
          "state": "Ohio"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04829435"
    },
    {
      "nct_id": "NCT00688597",
      "title": "Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Pompe Disease"
      ],
      "interventions": [
        {
          "name": "Duvoglustat",
          "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": 3,
      "start_date": "2008-12-08",
      "completion_date": "2009-12-14",
      "has_results": true,
      "last_update_posted_date": "2025-09-11",
      "last_synced_at": "2026-10-18T03:16:42.778Z",
      "location_count": 1,
      "location_summary": "Decatur, Georgia",
      "locations": [
        {
          "city": "Decatur",
          "state": "Georgia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00688597"
    },
    {
      "nct_id": "NCT07082543",
      "title": "A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Gangliosidoses, GM2",
        "Gangliosidosis, GM1"
      ],
      "interventions": [
        {
          "name": "AZ-3102",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Azafaros B.V.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "4 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "4 Years and older"
      },
      "enrollment_count": 75,
      "start_date": "2025-06-30",
      "completion_date": "2028-03-06",
      "has_results": false,
      "last_update_posted_date": "2026-09-22",
      "last_synced_at": "2026-10-18T03:16:42.778Z",
      "location_count": 5,
      "location_summary": "Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        },
        {
          "city": "Rochester",
          "state": "Minnesota"
        },
        {
          "city": "Dallas",
          "state": "Texas"
        },
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07082543"
    },
    {
      "nct_id": "NCT04193592",
      "title": "Efficacy and Safety of Pirfenidone Treatment in HPS-ILD",
      "overall_status": "UNKNOWN",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Hermansky Pudlak Syndrome",
        "Interstitial Lung Disease"
      ],
      "interventions": [
        {
          "name": "Pirfenidone",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Jesse Roman",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "75 Years",
        "sex": "ALL",
        "summary": "18 Years to 75 Years"
      },
      "enrollment_count": 50,
      "start_date": "2019-12-01",
      "completion_date": "2022-12-31",
      "has_results": false,
      "last_update_posted_date": "2019-12-10",
      "last_synced_at": "2026-10-18T03:16:42.778Z",
      "location_count": 1,
      "location_summary": "Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04193592"
    }
  ]
}