{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=53",
    "query": {
      "condition": "Inborn Errors of Metabolism Disorders",
      "page": 53
    },
    "page_size": 10
  },
  "pagination": {
    "page": 53,
    "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=54&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=52&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-11-15T17:05:53.010Z",
  "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": "NCT00962260",
      "title": "Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease",
      "overall_status": "NO_LONGER_AVAILABLE",
      "study_type": "EXPANDED_ACCESS",
      "phases": [],
      "conditions": [
        "Gaucher Disease"
      ],
      "interventions": [
        {
          "name": "Plant cell expressed recombinant glucocerebrosidase (prGCD)",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Pfizer",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": null,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": null,
      "start_date": null,
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2018-09-07",
      "last_synced_at": "2026-11-15T17:05:53.010Z",
      "location_count": 14,
      "location_summary": "La Jolla, California • Aurora, Colorado • Coral Springs, Florida + 11 more",
      "locations": [
        {
          "city": "La Jolla",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Coral Springs",
          "state": "Florida"
        },
        {
          "city": "Decatur",
          "state": "Georgia"
        },
        {
          "city": "Skokie",
          "state": "Illinois"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00962260"
    },
    {
      "nct_id": "NCT01899950",
      "title": "Longitudinal Study of Cognition With Niemann-Pick Disease, Type C",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Niemann-Pick Disease, Type C"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Mayo Clinic",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": "99 Years",
        "sex": "ALL",
        "summary": "2 Years to 99 Years"
      },
      "enrollment_count": 70,
      "start_date": "2011-03",
      "completion_date": "2014-09",
      "has_results": false,
      "last_update_posted_date": "2016-01-06",
      "last_synced_at": "2026-11-15T17:05:53.010Z",
      "location_count": 1,
      "location_summary": "Rochester, Minnesota",
      "locations": [
        {
          "city": "Rochester",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01899950"
    },
    {
      "nct_id": "NCT04980248",
      "title": "Study of ALXN1850 in Participants With Hypophosphatasia (HPP)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Hypophosphatasia"
      ],
      "interventions": [
        {
          "name": "ALXN1850",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Alexion Pharmaceuticals, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "64 Years",
        "sex": "ALL",
        "summary": "18 Years to 64 Years"
      },
      "enrollment_count": 15,
      "start_date": "2021-09-28",
      "completion_date": "2022-08-24",
      "has_results": true,
      "last_update_posted_date": "2024-12-27",
      "last_synced_at": "2026-11-15T17:05:53.010Z",
      "location_count": 4,
      "location_summary": "Las Vegas, Nevada • Columbus, Ohio • Nashville, Tennessee + 1 more",
      "locations": [
        {
          "city": "Las Vegas",
          "state": "Nevada"
        },
        {
          "city": "Columbus",
          "state": "Ohio"
        },
        {
          "city": "Nashville",
          "state": "Tennessee"
        },
        {
          "city": "Austin",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04980248"
    },
    {
      "nct_id": "NCT00430248",
      "title": "Efficacy and Safety of Oral Febuxostat in Participants With Gout",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Gout"
      ],
      "interventions": [
        {
          "name": "Febuxostat",
          "type": "DRUG"
        },
        {
          "name": "Allopurinol",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Takeda",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "85 Years",
        "sex": "ALL",
        "summary": "18 Years to 85 Years"
      },
      "enrollment_count": 2269,
      "start_date": "2007-02",
      "completion_date": "2008-03",
      "has_results": true,
      "last_update_posted_date": "2012-02-02",
      "last_synced_at": "2026-11-15T17:05:53.010Z",
      "location_count": 258,
      "location_summary": "Birmingham, Alabama • Hoover, Alabama • Hueytown, Alabama + 255 more",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Hoover",
          "state": "Alabama"
        },
        {
          "city": "Hueytown",
          "state": "Alabama"
        },
        {
          "city": "Huntsville",
          "state": "Alabama"
        },
        {
          "city": "Mobile",
          "state": "Alabama"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00430248"
    },
    {
      "nct_id": "NCT00000673",
      "title": "A Phase I/II Open-Labelled Trial of Intravitreal Ganciclovir Salvage Therapy for AIDS Patients With Active CMV Retinitis Who Are Intolerant of Systemic Therapy",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Cytomegalovirus Retinitis",
        "HIV Infections"
      ],
      "interventions": [
        {
          "name": "Ganciclovir",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "National Institute of Allergy and Infectious Diseases (NIAID)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "13 Years",
        "maximum_age": "65 Years",
        "sex": "ALL",
        "summary": "13 Years to 65 Years"
      },
      "enrollment_count": 38,
      "start_date": null,
      "completion_date": "1993-05",
      "has_results": false,
      "last_update_posted_date": "2021-10-27",
      "last_synced_at": "2026-11-15T17:05:53.010Z",
      "location_count": 2,
      "location_summary": "Miami, Florida • St Louis, Missouri",
      "locations": [
        {
          "city": "Miami",
          "state": "Florida"
        },
        {
          "city": "St Louis",
          "state": "Missouri"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00000673"
    },
    {
      "nct_id": "NCT04411654",
      "title": "Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Gaucher Disease, Type 2"
      ],
      "interventions": [
        {
          "name": "LY3884961",
          "type": "GENETIC"
        },
        {
          "name": "Methylprednisolone",
          "type": "DRUG"
        },
        {
          "name": "Sirolimus",
          "type": "DRUG"
        },
        {
          "name": "Prednisone",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "GENETIC",
        "DRUG"
      ],
      "sponsor": "Prevail Therapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "0 Months",
        "maximum_age": "24 Months",
        "sex": "ALL",
        "summary": "0 Months to 24 Months"
      },
      "enrollment_count": 7,
      "start_date": "2021-06-29",
      "completion_date": "2027-02",
      "has_results": false,
      "last_update_posted_date": "2026-10-22",
      "last_synced_at": "2026-11-15T17:05:53.010Z",
      "location_count": 5,
      "location_summary": "Oakland, California • Minneapolis, Minnesota • New York, New York + 2 more",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        },
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04411654"
    },
    {
      "nct_id": "NCT07531251",
      "title": "Clinical Trial in Patients With Barth Syndrome- 4TAZPower",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE4"
      ],
      "conditions": [
        "Barth Syndrome"
      ],
      "interventions": [
        {
          "name": "Elamipretide",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Stealth BioTherapeutics Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "5 Years",
        "maximum_age": "55 Years",
        "sex": "MALE",
        "summary": "5 Years to 55 Years · Male only"
      },
      "enrollment_count": 48,
      "start_date": "2026-07-02",
      "completion_date": "2029-11-30",
      "has_results": false,
      "last_update_posted_date": "2026-11-09",
      "last_synced_at": "2026-11-15T17:05:53.010Z",
      "location_count": 1,
      "location_summary": "Needham, Massachusetts",
      "locations": [
        {
          "city": "Needham",
          "state": "Massachusetts"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07531251"
    },
    {
      "nct_id": "NCT06034405",
      "title": "Analysis of Lumbar Spine Stenosis Specimens for Identification of Transthyretin Cardiac Amyloidosis",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Lumbar Spinal Stenosis",
        "Cardiac Amyloidosis",
        "ATTR Amyloidosis Wild Type",
        "ATTR Gene Mutation",
        "ATTRV122I Amyloidosis",
        "Cardiomyopathy, Hypertrophic"
      ],
      "interventions": [
        {
          "name": "Tc99-PYP or Tc99-HDMP Scan",
          "type": "DIAGNOSTIC_TEST"
        }
      ],
      "intervention_types": [
        "DIAGNOSTIC_TEST"
      ],
      "sponsor": "Columbia University",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "50 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "50 Years and older"
      },
      "enrollment_count": 1663,
      "start_date": "2023-09-19",
      "completion_date": "2028-05",
      "has_results": false,
      "last_update_posted_date": "2026-06-04",
      "last_synced_at": "2026-11-15T17:05:53.010Z",
      "location_count": 6,
      "location_summary": "Beverly Hills, California • Boston, Massachusetts • Rochester, Minnesota + 2 more",
      "locations": [
        {
          "city": "Beverly Hills",
          "state": "California"
        },
        {
          "city": "Boston",
          "state": "Massachusetts"
        },
        {
          "city": "Boston",
          "state": "Massachusetts"
        },
        {
          "city": "Rochester",
          "state": "Minnesota"
        },
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06034405"
    },
    {
      "nct_id": "NCT00872729",
      "title": "Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Cystinosis"
      ],
      "interventions": [
        {
          "name": "Cystagon®",
          "type": "DRUG"
        },
        {
          "name": "RP103",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Amgen",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 9,
      "start_date": "2009-05",
      "completion_date": "2009-10",
      "has_results": true,
      "last_update_posted_date": "2024-12-19",
      "last_synced_at": "2026-11-15T17:05:53.010Z",
      "location_count": 1,
      "location_summary": "San Diego, California",
      "locations": [
        {
          "city": "San Diego",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00872729"
    },
    {
      "nct_id": "NCT06839456",
      "title": "Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Leukemia",
        "High Risk Acute Lymphoblastic Leukemia",
        "High Risk Acute Myeloid Leukemia",
        "Relapse Leukemia",
        "MDS (Myelodysplastic Syndrome)",
        "Relapsed Non-Hodgkin Lymphoma",
        "Acquired Aplastic Anemia",
        "Inherited BMF Syndrome",
        "Immunodeficiency",
        "Primary Immune Regulatory Disorder",
        "Hemoglobinopathies",
        "Bone Marrow Failure",
        "Inborn Errors of Metabolism",
        "HLH"
      ],
      "interventions": [
        {
          "name": "Phase 1 Dose Level 1",
          "type": "DEVICE"
        },
        {
          "name": "Phase 1 Dose Level 2",
          "type": "DEVICE"
        },
        {
          "name": "Phase 1 Dose Level 3",
          "type": "DEVICE"
        },
        {
          "name": "Phase 2 Maximum Tolerated Dose determined in Phase 1",
          "type": "DEVICE"
        },
        {
          "name": "Phase 2 Established Dose from prior study, NCT03810196",
          "type": "DEVICE"
        }
      ],
      "intervention_types": [
        "DEVICE"
      ],
      "sponsor": "Children's Hospital of Philadelphia",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Month",
        "maximum_age": "25 Years",
        "sex": "ALL",
        "summary": "1 Month to 25 Years"
      },
      "enrollment_count": 100,
      "start_date": "2025-03-21",
      "completion_date": "2032-03",
      "has_results": false,
      "last_update_posted_date": "2026-04-15",
      "last_synced_at": "2026-11-15T17:05:53.010Z",
      "location_count": 1,
      "location_summary": "Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06839456"
    }
  ]
}