{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inherited+Metabolic+Disorders+%28Inborn+Errors+of+Metabolism%29",
    "query": {
      "condition": "Inherited Metabolic Disorders (Inborn Errors of Metabolism)"
    },
    "page_size": 10
  },
  "pagination": {
    "page": 1,
    "page_size": 10,
    "total_count": 1578,
    "total_pages": 158,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inherited+Metabolic+Disorders+%28Inborn+Errors+of+Metabolism%29&page=2&page_size=10",
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-06-10T04:05:54.711Z",
  "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": "NCT05238519",
      "title": "Improved Diagnosis of Familial Hypercholesterolemia Across the Northland (ID-FH)",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Familial Hypercholesterolemia",
        "Cholesterol, Elevated",
        "Genetic Disease"
      ],
      "interventions": [
        {
          "name": "Motivational interview (MI)",
          "type": "BEHAVIORAL"
        }
      ],
      "intervention_types": [
        "BEHAVIORAL"
      ],
      "sponsor": "Essentia Health",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": "75 Years",
        "sex": "ALL",
        "summary": "2 Years to 75 Years"
      },
      "enrollment_count": 300,
      "start_date": "2022-02-15",
      "completion_date": "2026-06",
      "has_results": false,
      "last_update_posted_date": "2026-01-16",
      "last_synced_at": "2026-06-10T04:05:54.711Z",
      "location_count": 2,
      "location_summary": "Duluth, Minnesota",
      "locations": [
        {
          "city": "Duluth",
          "state": "Minnesota"
        },
        {
          "city": "Duluth",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05238519"
    },
    {
      "nct_id": "NCT04022434",
      "title": "Investigation of Supplemental L-alanine in the Management of Dietary Fructose Intolerance",
      "overall_status": "UNKNOWN",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "NA"
      ],
      "conditions": [
        "Fructose Intolerance"
      ],
      "interventions": [
        {
          "name": "Placedo",
          "type": "DIETARY_SUPPLEMENT"
        },
        {
          "name": "Alanine",
          "type": "DIETARY_SUPPLEMENT"
        }
      ],
      "intervention_types": [
        "DIETARY_SUPPLEMENT"
      ],
      "sponsor": "Augusta University",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 65,
      "start_date": "2014-01-07",
      "completion_date": "2020-12-30",
      "has_results": false,
      "last_update_posted_date": "2020-04-15",
      "last_synced_at": "2026-06-10T04:05:54.711Z",
      "location_count": 1,
      "location_summary": "Augusta, Georgia",
      "locations": [
        {
          "city": "Augusta",
          "state": "Georgia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04022434"
    },
    {
      "nct_id": "NCT00532844",
      "title": "A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Endothelial Dysfunction"
      ],
      "interventions": [
        {
          "name": "Sapropterin Dihydrochloride",
          "type": "DRUG"
        },
        {
          "name": "Sapropterin Dihydrochloride and Vitamin C",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "BioMarin Pharmaceutical",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "75 Years",
        "sex": "ALL",
        "summary": "18 Years to 75 Years"
      },
      "enrollment_count": 52,
      "start_date": "2007-09",
      "completion_date": "2009-03",
      "has_results": true,
      "last_update_posted_date": "2021-05-17",
      "last_synced_at": "2026-06-10T04:05:54.711Z",
      "location_count": 1,
      "location_summary": "Hackensack, New Jersey",
      "locations": [
        {
          "city": "Hackensack",
          "state": "New Jersey"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00532844"
    },
    {
      "nct_id": "NCT00003336",
      "title": "Pilot Study Of Unrelated UCB Transplant for Non-Malignant Hematologic Conditions",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Leukemia",
        "Myelodysplastic Syndromes",
        "Myelodysplastic/Myeloproliferative Diseases"
      ],
      "interventions": [
        {
          "name": "anti-thymocyte globulin",
          "type": "BIOLOGICAL"
        },
        {
          "name": "busulfan",
          "type": "DRUG"
        },
        {
          "name": "cyclophosphamide",
          "type": "DRUG"
        },
        {
          "name": "melphalan",
          "type": "DRUG"
        },
        {
          "name": "methylprednisolone",
          "type": "DRUG"
        },
        {
          "name": "bone marrow ablation with stem cell support",
          "type": "PROCEDURE"
        },
        {
          "name": "umbilical cord blood transplantation",
          "type": "PROCEDURE"
        },
        {
          "name": "radiation therapy",
          "type": "RADIATION"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL",
        "DRUG",
        "PROCEDURE",
        "RADIATION"
      ],
      "sponsor": "Case Comprehensive Cancer Center",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "55 Years",
        "sex": "ALL",
        "summary": "Up to 55 Years"
      },
      "enrollment_count": 6,
      "start_date": "1998-01",
      "completion_date": "2006-02",
      "has_results": false,
      "last_update_posted_date": "2010-06-11",
      "last_synced_at": "2026-06-10T04:05:54.711Z",
      "location_count": 1,
      "location_summary": "Cleveland, Ohio",
      "locations": [
        {
          "city": "Cleveland",
          "state": "Ohio"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00003336"
    },
    {
      "nct_id": "NCT03811028",
      "title": "A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Sanfilippo Syndrome Type A (MPS IIIA)"
      ],
      "interventions": [
        {
          "name": "SOBI003",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Swedish Orphan Biovitrum",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Months",
        "maximum_age": "78 Months",
        "sex": "ALL",
        "summary": "18 Months to 78 Months"
      },
      "enrollment_count": 6,
      "start_date": "2019-01-19",
      "completion_date": "2021-05-07",
      "has_results": true,
      "last_update_posted_date": "2022-02-25",
      "last_synced_at": "2026-06-10T04:05:54.711Z",
      "location_count": 2,
      "location_summary": "Oakland, California • Chapel Hill, North Carolina",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Chapel Hill",
          "state": "North Carolina"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03811028"
    },
    {
      "nct_id": "NCT01885936",
      "title": "Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Pompe Disease"
      ],
      "interventions": [
        {
          "name": "Albuterol",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Duke University",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 16,
      "start_date": "2013-06",
      "completion_date": "2016-12-16",
      "has_results": true,
      "last_update_posted_date": "2019-07-15",
      "last_synced_at": "2026-06-10T04:05:54.711Z",
      "location_count": 1,
      "location_summary": "Durham, North Carolina",
      "locations": [
        {
          "city": "Durham",
          "state": "North Carolina"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01885936"
    },
    {
      "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": "2026-06-10T04:05:54.711Z",
      "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": "NCT00808106",
      "title": "Clinical, Cellular, and Molecular Investigation Into Oculocutaneous Albinism",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Oculocutaneous Albinism"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "National Human Genome Research Institute (NHGRI)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Year",
        "maximum_age": "80 Years",
        "sex": "ALL",
        "summary": "1 Year to 80 Years"
      },
      "enrollment_count": 206,
      "start_date": "2008-12-11",
      "completion_date": "2019-12-31",
      "has_results": false,
      "last_update_posted_date": "2020-01-06",
      "last_synced_at": "2026-06-10T04:05:54.711Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00808106"
    },
    {
      "nct_id": "NCT00879034",
      "title": "A Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Progeria",
        "Hutchinson-Gilford Syndrome"
      ],
      "interventions": [
        {
          "name": "Lonafarnib",
          "type": "DRUG"
        },
        {
          "name": "Zoledronic Acid",
          "type": "DRUG"
        },
        {
          "name": "Pravastatin",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Boston Children's Hospital",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 5,
      "start_date": "2009-03",
      "completion_date": "2009-04",
      "has_results": true,
      "last_update_posted_date": "2019-06-13",
      "last_synced_at": "2026-06-10T04:05:54.711Z",
      "location_count": 1,
      "location_summary": "Boston, Massachusetts",
      "locations": [
        {
          "city": "Boston",
          "state": "Massachusetts"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00879034"
    },
    {
      "nct_id": "NCT04908319",
      "title": "Hepatic Histopathology in Urea Cycle Disorders",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Urea Cycle Disorder",
        "Ornithine Transcarbamylase Deficiency",
        "Citrullinemia 1",
        "ARGI Deficiency",
        "ASL Deficiency",
        "Argininosuccinic Aciduria",
        "ASS Deficiency",
        "Hyperargininemia",
        "Carbamyl Phosphate Synthetase Deficiency",
        "NAGS Deficiency"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Baylor College of Medicine",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 70,
      "start_date": "2022-02-24",
      "completion_date": "2026-06-30",
      "has_results": false,
      "last_update_posted_date": "2025-07-20",
      "last_synced_at": "2026-06-10T04:05:54.711Z",
      "location_count": 2,
      "location_summary": "Washington D.C., District of Columbia • Houston, Texas",
      "locations": [
        {
          "city": "Washington D.C.",
          "state": "District of Columbia"
        },
        {
          "city": "Houston",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04908319"
    }
  ]
}