{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=56",
    "query": {
      "condition": "Inborn Errors of Metabolism Disorders",
      "page": 56
    },
    "page_size": 10
  },
  "pagination": {
    "page": 56,
    "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=57&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=55&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-11-15T20:54:07.358Z",
  "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": "NCT03409744",
      "title": "Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Homozygous Familial Hypercholesterolemia"
      ],
      "interventions": [
        {
          "name": "evinacumab",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Regeneron Pharmaceuticals",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "12 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "12 Years and older"
      },
      "enrollment_count": 116,
      "start_date": "2018-03-13",
      "completion_date": "2023-04-13",
      "has_results": true,
      "last_update_posted_date": "2025-04-08",
      "last_synced_at": "2026-11-15T20:54:07.358Z",
      "location_count": 9,
      "location_summary": "Los Angeles, California • Boca Raton, Florida • Boston, Massachusetts + 5 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Boca Raton",
          "state": "Florida"
        },
        {
          "city": "Boston",
          "state": "Massachusetts"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Cincinnati",
          "state": "Ohio"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03409744"
    },
    {
      "nct_id": "NCT06079281",
      "title": "Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Hypophosphatasia"
      ],
      "interventions": [
        {
          "name": "ALXN1850",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Alexion Pharmaceuticals, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "12 Years",
        "maximum_age": "130 Years",
        "sex": "ALL",
        "summary": "12 Years to 130 Years"
      },
      "enrollment_count": 124,
      "start_date": "2024-01-03",
      "completion_date": "2028-03-29",
      "has_results": false,
      "last_update_posted_date": "2026-06-22",
      "last_synced_at": "2026-11-15T20:54:07.358Z",
      "location_count": 5,
      "location_summary": "Indianapolis, Indiana • Garden City, New York • Durham, North Carolina + 2 more",
      "locations": [
        {
          "city": "Indianapolis",
          "state": "Indiana"
        },
        {
          "city": "Garden City",
          "state": "New York"
        },
        {
          "city": "Durham",
          "state": "North Carolina"
        },
        {
          "city": "Columbus",
          "state": "Ohio"
        },
        {
          "city": "Nashville",
          "state": "Tennessee"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06079281"
    },
    {
      "nct_id": "NCT06270316",
      "title": "Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [
        {
          "name": "AMT-191",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "UniQure Biopharma B.V.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "65 Years",
        "sex": "MALE",
        "summary": "18 Years to 65 Years · Male only"
      },
      "enrollment_count": 20,
      "start_date": "2024-06-05",
      "completion_date": "2031-11-30",
      "has_results": false,
      "last_update_posted_date": "2026-09-24",
      "last_synced_at": "2026-11-15T20:54:07.358Z",
      "location_count": 10,
      "location_summary": "Birmingham, Alabama • Los Angeles, California • Atlanta, Georgia + 7 more",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06270316"
    },
    {
      "nct_id": "NCT05508009",
      "title": "Early Trial of Allogeneic Hematopoietic Stem Cell Transplantation for Patients Who Will Receive a Kidney Transplant From the Same Donor",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "SIOD",
        "Cystinosis",
        "FSGS",
        "SLE Nephritis",
        "CKD Stage 4"
      ],
      "interventions": [
        {
          "name": "Cyclophosphamide 1200 mg/Kg",
          "type": "DRUG"
        },
        {
          "name": "Fludarabine",
          "type": "DRUG"
        },
        {
          "name": "Cyclophosphamide 100 mg/Kg",
          "type": "DRUG"
        },
        {
          "name": "Total Body Irradiation",
          "type": "RADIATION"
        },
        {
          "name": "ATG",
          "type": "DRUG"
        },
        {
          "name": "Rituximab",
          "type": "DRUG"
        },
        {
          "name": "Melphalan",
          "type": "DRUG"
        },
        {
          "name": "CliniMACS® TCR α/β Reagent Kit and CliniMACS® CD19 System",
          "type": "DEVICE"
        },
        {
          "name": "Kidney Transplant",
          "type": "PROCEDURE"
        }
      ],
      "intervention_types": [
        "DRUG",
        "RADIATION",
        "DEVICE",
        "PROCEDURE"
      ],
      "sponsor": "Alice Bertaina",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Year",
        "maximum_age": "30 Years",
        "sex": "ALL",
        "summary": "1 Year to 30 Years"
      },
      "enrollment_count": 12,
      "start_date": "2023-01-10",
      "completion_date": "2034-10",
      "has_results": false,
      "last_update_posted_date": "2023-07-17",
      "last_synced_at": "2026-11-15T20:54:07.358Z",
      "location_count": 1,
      "location_summary": "Palo Alto, California",
      "locations": [
        {
          "city": "Palo Alto",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05508009"
    },
    {
      "nct_id": "NCT02785744",
      "title": "Genzyme Osteopenia/Osteoporosis Study",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Gaucher Disease"
      ],
      "interventions": [
        {
          "name": "Gaucher disease DNA mutation analysis",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "NYU Langone Health",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 76,
      "start_date": "2016-04-01",
      "completion_date": "2019-09-01",
      "has_results": false,
      "last_update_posted_date": "2021-03-23",
      "last_synced_at": "2026-11-15T20:54:07.358Z",
      "location_count": 1,
      "location_summary": "New York, New York",
      "locations": [
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02785744"
    },
    {
      "nct_id": "NCT02432144",
      "title": "A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Sly Syndrome",
        "MPS VII",
        "Mucopolysaccharidosis",
        "Mucopolysaccharidosis VII"
      ],
      "interventions": [
        {
          "name": "UX003",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Ultragenyx Pharmaceutical Inc",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "5 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "5 Years and older"
      },
      "enrollment_count": 12,
      "start_date": "2015-11-10",
      "completion_date": "2019-01-14",
      "has_results": true,
      "last_update_posted_date": "2020-07-30",
      "last_synced_at": "2026-11-15T20:54:07.358Z",
      "location_count": 4,
      "location_summary": "Oakland, California • Orange, California • Minneapolis, Minnesota + 1 more",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        },
        {
          "city": "Houston",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02432144"
    },
    {
      "nct_id": "NCT06843811",
      "title": "Sirolimus for Leigh Syndrome",
      "overall_status": "ENROLLING_BY_INVITATION",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Leigh Syndrome"
      ],
      "interventions": [
        {
          "name": "Sirolimus",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Matthew Demczko",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Months",
        "maximum_age": "55 Years",
        "sex": "ALL",
        "summary": "6 Months to 55 Years"
      },
      "enrollment_count": 15,
      "start_date": "2025-02-27",
      "completion_date": "2027-12",
      "has_results": false,
      "last_update_posted_date": "2026-03-31",
      "last_synced_at": "2026-11-15T20:54:07.358Z",
      "location_count": 1,
      "location_summary": "Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06843811"
    },
    {
      "nct_id": "NCT04573309",
      "title": "Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Wilson Disease"
      ],
      "interventions": [
        {
          "name": "ALXN1840",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Alexion Pharmaceuticals, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 9,
      "start_date": "2020-09-07",
      "completion_date": "2022-06-07",
      "has_results": true,
      "last_update_posted_date": "2024-06-24",
      "last_synced_at": "2026-11-15T20:54:07.358Z",
      "location_count": 1,
      "location_summary": "New Haven, Connecticut",
      "locations": [
        {
          "city": "New Haven",
          "state": "Connecticut"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04573309"
    },
    {
      "nct_id": "NCT00158600",
      "title": "A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Pompe Disease (Late-onset)",
        "Glycogen Storage Disease Type II (GSD-II)",
        "Acid Maltase Deficiency Disease",
        "Glycogenosis 2"
      ],
      "interventions": [
        {
          "name": "alglucosidase alfa",
          "type": "BIOLOGICAL"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL",
        "DRUG"
      ],
      "sponsor": "Genzyme, a Sanofi Company",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "8 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "8 Years and older"
      },
      "enrollment_count": 90,
      "start_date": "2005-09",
      "completion_date": "2007-09",
      "has_results": true,
      "last_update_posted_date": "2015-04-28",
      "last_synced_at": "2026-11-15T20:54:07.358Z",
      "location_count": 5,
      "location_summary": "Beverly Hills, California • Washington D.C., District of Columbia • St Louis, Missouri + 2 more",
      "locations": [
        {
          "city": "Beverly Hills",
          "state": "California"
        },
        {
          "city": "Washington D.C.",
          "state": "District of Columbia"
        },
        {
          "city": "St Louis",
          "state": "Missouri"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00158600"
    },
    {
      "nct_id": "NCT07247058",
      "title": "Isturisa Treatment in Mild Autonomous Cortisol Secretion( MACS)",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE4"
      ],
      "conditions": [
        "Mild Autonomous Cortisol Secretion (MACS)"
      ],
      "interventions": [
        {
          "name": "Osilodrostat (Isturisa)",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Johns Hopkins University",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 10,
      "start_date": "2026-02-02",
      "completion_date": "2029-03",
      "has_results": false,
      "last_update_posted_date": "2026-10-23",
      "last_synced_at": "2026-11-15T20:54:07.358Z",
      "location_count": 1,
      "location_summary": "Baltimore, Maryland",
      "locations": [
        {
          "city": "Baltimore",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07247058"
    }
  ]
}