{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=53",
    "query": {
      "condition": "Inborn Errors of Metabolism",
      "page": 53
    },
    "page_size": 10
  },
  "pagination": {
    "page": 53,
    "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=54&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism&page=52&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-19T14:22:06.740Z",
  "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": "NCT00294671",
      "title": "The Effect of Diflunisal on Familial Amyloidosis",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2",
        "PHASE3"
      ],
      "conditions": [
        "Familial Amyloid Polyneuropathy",
        "Familial Amyloidosis"
      ],
      "interventions": [
        {
          "name": "diflunisal",
          "type": "DRUG"
        },
        {
          "name": "placebo",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "DRUG",
        "OTHER"
      ],
      "sponsor": "Boston University",
      "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": 130,
      "start_date": "2006-02",
      "completion_date": "2012-12",
      "has_results": true,
      "last_update_posted_date": "2017-03-17",
      "last_synced_at": "2026-10-19T14:22:06.740Z",
      "location_count": 3,
      "location_summary": "Boston, Massachusetts • Rochester, Minnesota • New York, New York",
      "locations": [
        {
          "city": "Boston",
          "state": "Massachusetts"
        },
        {
          "city": "Rochester",
          "state": "Minnesota"
        },
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00294671"
    },
    {
      "nct_id": "NCT03181828",
      "title": "Manipulating the Gut Microbiome Study",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Urea Cycle Disorder"
      ],
      "interventions": [
        {
          "name": "Acetohydroxamic Acid Oral Tablet",
          "type": "DRUG"
        },
        {
          "name": "No treatment",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "DRUG",
        "OTHER"
      ],
      "sponsor": "Nicholas Ah Mew",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "60 Years",
        "sex": "ALL",
        "summary": "18 Years to 60 Years"
      },
      "enrollment_count": 4,
      "start_date": "2017-03-24",
      "completion_date": "2018-06-05",
      "has_results": true,
      "last_update_posted_date": "2021-12-15",
      "last_synced_at": "2026-10-19T14:22:06.740Z",
      "location_count": 1,
      "location_summary": "Washington D.C., District of Columbia",
      "locations": [
        {
          "city": "Washington D.C.",
          "state": "District of Columbia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03181828"
    },
    {
      "nct_id": "NCT00272844",
      "title": "Treatment of the Cholesterol Defect in Smith-Lemli-Opitz Syndrome",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Smith-Lemli-Opitz Syndrome"
      ],
      "interventions": [
        {
          "name": "crystalline cholesterol oil-based suspension",
          "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": 23,
      "start_date": "1998-01",
      "completion_date": "2011-07",
      "has_results": true,
      "last_update_posted_date": "2017-10-20",
      "last_synced_at": "2026-10-19T14:22:06.740Z",
      "location_count": 1,
      "location_summary": "Boston, Massachusetts",
      "locations": [
        {
          "city": "Boston",
          "state": "Massachusetts"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00272844"
    },
    {
      "nct_id": "NCT07643844",
      "title": "AAVrh10-PCCA Gene Therapy for Propionic Acidemia",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Propionic Acidemia"
      ],
      "interventions": [
        {
          "name": "AAVrh10-PCCA low dose",
          "type": "DRUG"
        },
        {
          "name": "AAVrh10-PCCA middle dose",
          "type": "DRUG"
        },
        {
          "name": "AAVrh10-PCCA high dose",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Mayo Clinic",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Months",
        "maximum_age": "2 Years",
        "sex": "ALL",
        "summary": "6 Months to 2 Years"
      },
      "enrollment_count": 9,
      "start_date": "2026-07-20",
      "completion_date": "2033-12",
      "has_results": false,
      "last_update_posted_date": "2026-08-21",
      "last_synced_at": "2026-10-19T14:22:06.740Z",
      "location_count": 1,
      "location_summary": "Rochester, Minnesota",
      "locations": [
        {
          "city": "Rochester",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07643844"
    },
    {
      "nct_id": "NCT03718234",
      "title": "Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Congenital Adrenal Hyperplasia",
        "Hyperplasia",
        "Adrenal Hyperplasia",
        "Congenital Disorders",
        "Adrenocortical Hyperfunction",
        "Disorders of Sex Development",
        "Urogenital Abnormalities",
        "Genetic Diseases, Inborn",
        "Steroid Metabolic Diseases, Inborn",
        "Adrenal Gland Disease",
        "Hydrocortisone"
      ],
      "interventions": [
        {
          "name": "Subcutaneous hydrocortisone",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "University of Minnesota",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "4 Years",
        "maximum_age": "18 Years",
        "sex": "ALL",
        "summary": "4 Years to 18 Years"
      },
      "enrollment_count": 11,
      "start_date": "2019-01-01",
      "completion_date": "2023-02-23",
      "has_results": true,
      "last_update_posted_date": "2025-06-25",
      "last_synced_at": "2026-10-19T14:22:06.740Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03718234"
    },
    {
      "nct_id": "NCT00694109",
      "title": "An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Lipid Metabolism, Inborn Errors",
        "Hypercholesterolemia, Autosomal Dominant",
        "Hyperlipidemias",
        "Metabolic Diseases",
        "Hyperlipoproteinemia Type II",
        "Metabolism, Inborn Errors",
        "Genetic Diseases, Inborn",
        "Infant, Newborn, Diseases",
        "Metabolic Disorder",
        "Congenital Abnormalities",
        "Hypercholesterolemia",
        "Hyperlipoproteinemias",
        "Dyslipidemias",
        "Lipid Metabolism Disorders"
      ],
      "interventions": [
        {
          "name": "Mipomersen Sodium",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Kastle Therapeutics, LLC",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "12 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "12 Years and older"
      },
      "enrollment_count": 144,
      "start_date": "2008-04",
      "completion_date": "2014-09",
      "has_results": true,
      "last_update_posted_date": "2016-09-09",
      "last_synced_at": "2026-10-19T14:22:06.740Z",
      "location_count": 20,
      "location_summary": "Mission Viejo, California • Newport Beach, California • Bridgeport, Connecticut + 17 more",
      "locations": [
        {
          "city": "Mission Viejo",
          "state": "California"
        },
        {
          "city": "Newport Beach",
          "state": "California"
        },
        {
          "city": "Bridgeport",
          "state": "Connecticut"
        },
        {
          "city": "Melbourne",
          "state": "Florida"
        },
        {
          "city": "Winter Park",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00694109"
    },
    {
      "nct_id": "NCT03609814",
      "title": "Study of Clofarabine and Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT)",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Hematologic Malignancies",
        "Nonmalignant Diseases",
        "Immunodeficiencies",
        "Hemoglobinopathies",
        "Genetic Inborn Errors of Metabolism",
        "Fanconi's Anemia",
        "Thalassemia",
        "Sickle Cell Disease"
      ],
      "interventions": [
        {
          "name": "Clofarabine",
          "type": "DRUG"
        },
        {
          "name": "Fludarabine Injection",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "University of California, San Francisco",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "17 Years",
        "sex": "ALL",
        "summary": "Up to 17 Years"
      },
      "enrollment_count": 30,
      "start_date": "2016-01-26",
      "completion_date": "2020-06-30",
      "has_results": false,
      "last_update_posted_date": "2021-10-04",
      "last_synced_at": "2026-10-19T14:22:06.740Z",
      "location_count": 1,
      "location_summary": "San Francisco, California",
      "locations": [
        {
          "city": "San Francisco",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03609814"
    },
    {
      "nct_id": "NCT00478647",
      "title": "Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2",
        "PHASE3"
      ],
      "conditions": [
        "Gaucher Disease"
      ],
      "interventions": [
        {
          "name": "GA-GCB (velaglucerase alfa)",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Shire",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "2 Years and older"
      },
      "enrollment_count": 40,
      "start_date": "2007-07-25",
      "completion_date": "2009-06-26",
      "has_results": true,
      "last_update_posted_date": "2021-06-10",
      "last_synced_at": "2026-10-19T14:22:06.740Z",
      "location_count": 11,
      "location_summary": "Los Angeles, California • Oakland, California • Decatur, Georgia + 8 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Decatur",
          "state": "Georgia"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00478647"
    },
    {
      "nct_id": "NCT07771621",
      "title": "PET Imaging With [18F]Fluselenamyl in People With Cardiac Amyloidosis",
      "overall_status": "ENROLLING_BY_INVITATION",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Cardiac Amyloidosis"
      ],
      "interventions": [
        {
          "name": "[18F]Fluselenamyl (FSA)",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Washington University School of Medicine",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 50,
      "start_date": "2026-01-14",
      "completion_date": "2030-12",
      "has_results": false,
      "last_update_posted_date": "2026-08-18",
      "last_synced_at": "2026-10-19T14:22:06.740Z",
      "location_count": 1,
      "location_summary": "St Louis, Missouri",
      "locations": [
        {
          "city": "St Louis",
          "state": "Missouri"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07771621"
    },
    {
      "nct_id": "NCT07653555",
      "title": "Cascade Testing In Identifying At-risk Family Members Of Individuals With Familial Hypercholesterolemia (FH) In PCP",
      "overall_status": "NOT_YET_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "NA"
      ],
      "conditions": [
        "Familial Hypercholesterolemia"
      ],
      "interventions": [
        {
          "name": "Cascade testing",
          "type": "OTHER"
        },
        {
          "name": "Standard of Care",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "Mayo Clinic",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Year",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "1 Year and older"
      },
      "enrollment_count": 480,
      "start_date": "2026-10-20",
      "completion_date": "2029-09-20",
      "has_results": false,
      "last_update_posted_date": "2026-09-18",
      "last_synced_at": "2026-10-19T14:22:06.740Z",
      "location_count": 1,
      "location_summary": "Rochester, Minnesota",
      "locations": [
        {
          "city": "Rochester",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07653555"
    }
  ]
}