{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=29",
    "query": {
      "condition": "Inborn Errors of Metabolism Disorders",
      "page": 29
    },
    "page_size": 10
  },
  "pagination": {
    "page": 29,
    "page_size": 10,
    "total_count": 1511,
    "total_pages": 152,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=30&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=28&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-29T06:38:13.329Z",
  "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": "NCT00787865",
      "title": "Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Krabbe Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "University of Pittsburgh",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "17 Years",
        "sex": "ALL",
        "summary": "Up to 17 Years"
      },
      "enrollment_count": 100,
      "start_date": "2008-04",
      "completion_date": "2027-04",
      "has_results": false,
      "last_update_posted_date": "2026-03-06",
      "last_synced_at": "2026-10-29T06:38:13.329Z",
      "location_count": 1,
      "location_summary": "Pittsburgh, Pennsylvania",
      "locations": [
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00787865"
    },
    {
      "nct_id": "NCT02534844",
      "title": "VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2",
        "PHASE3"
      ],
      "conditions": [
        "Niemann-Pick Disease, Type C"
      ],
      "interventions": [
        {
          "name": "Parts A/B: Adrabetadex",
          "type": "DRUG"
        },
        {
          "name": "Parts A/B: Sham Control",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "DRUG",
        "OTHER"
      ],
      "sponsor": "Mandos LLC",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "4 Years",
        "maximum_age": "21 Years",
        "sex": "ALL",
        "summary": "4 Years to 21 Years"
      },
      "enrollment_count": 56,
      "start_date": "2015-10",
      "completion_date": "2018-03-28",
      "has_results": true,
      "last_update_posted_date": "2023-02-22",
      "last_synced_at": "2026-10-29T06:38:13.329Z",
      "location_count": 14,
      "location_summary": "Birmingham, Alabama • Orange, California • San Francisco, California + 11 more",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "San Francisco",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Gainesville",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02534844"
    },
    {
      "nct_id": "NCT01677286",
      "title": "Safety and Effect of Doxycycline in Patients With Amyloidosis",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Amyloidosis"
      ],
      "interventions": [
        {
          "name": "Doxycycline 100 mg po bid x 12 months",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Boston University",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 25,
      "start_date": "2012-07",
      "completion_date": "2015-12-15",
      "has_results": true,
      "last_update_posted_date": "2017-08-01",
      "last_synced_at": "2026-10-29T06:38:13.329Z",
      "location_count": 1,
      "location_summary": "Boston, Massachusetts",
      "locations": [
        {
          "city": "Boston",
          "state": "Massachusetts"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01677286"
    },
    {
      "nct_id": "NCT02422654",
      "title": "Taste Evaluation of Different Liquid Formulations With Eliglustat",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Gaucher Disease"
      ],
      "interventions": [
        {
          "name": "eliglustat",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Genzyme, a Sanofi Company",
      "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": 8,
      "start_date": "2015-04",
      "completion_date": "2015-05",
      "has_results": false,
      "last_update_posted_date": "2015-05-27",
      "last_synced_at": "2026-10-29T06:38:13.329Z",
      "location_count": 1,
      "location_summary": "Evansville, Indiana",
      "locations": [
        {
          "city": "Evansville",
          "state": "Indiana"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02422654"
    },
    {
      "nct_id": "NCT00483379",
      "title": "High Dose or High Dose Frequency Study of Alglucosidase Alfa",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE4"
      ],
      "conditions": [
        "Pompe Disease",
        "Glycogen Storage Disease Type II (GSD-II)",
        "Glycogenesis 2 Acid Maltase Deficiency"
      ],
      "interventions": [
        {
          "name": "alglucosidase alfa",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Genzyme, a Sanofi Company",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Months",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "6 Months and older"
      },
      "enrollment_count": 13,
      "start_date": "2007-05",
      "completion_date": "2010-07",
      "has_results": true,
      "last_update_posted_date": "2014-03-07",
      "last_synced_at": "2026-10-29T06:38:13.329Z",
      "location_count": 9,
      "location_summary": "Birmingham, Alabama • Stanford, California • Washington D.C., District of Columbia + 6 more",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Stanford",
          "state": "California"
        },
        {
          "city": "Washington D.C.",
          "state": "District of Columbia"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "Kansas City",
          "state": "Kansas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00483379"
    },
    {
      "nct_id": "NCT00002273",
      "title": "A Study of Ganciclovir in the Treatment of Cytomegalovirus of the Large Intestine in Patients With AIDS",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "NA"
      ],
      "conditions": [
        "Colitis",
        "HIV Infections"
      ],
      "interventions": [
        {
          "name": "Ganciclovir",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Hoffmann-La Roche",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "21 Years",
        "maximum_age": null,
        "sex": "MALE",
        "summary": "21 Years and older · Male only"
      },
      "enrollment_count": null,
      "start_date": null,
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2005-06-24",
      "last_synced_at": "2026-10-29T06:38:13.329Z",
      "location_count": 1,
      "location_summary": "Palo Alto, California",
      "locations": [
        {
          "city": "Palo Alto",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00002273"
    },
    {
      "nct_id": "NCT00004294",
      "title": "Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Gaucher's Disease"
      ],
      "interventions": [
        {
          "name": "human glucocerebrosidase gene into autologous peripheral blood stem cells",
          "type": "GENETIC"
        }
      ],
      "intervention_types": [
        "GENETIC"
      ],
      "sponsor": "National Center for Research Resources (NCRR)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "65 Years",
        "sex": "ALL",
        "summary": "18 Years to 65 Years"
      },
      "enrollment_count": 15,
      "start_date": "1999-11",
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2005-06-24",
      "last_synced_at": "2026-10-29T06:38:13.329Z",
      "location_count": 1,
      "location_summary": "Pittsburgh, Pennsylvania",
      "locations": [
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00004294"
    },
    {
      "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": "2026-10-29T06:38:13.329Z",
      "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": "NCT02128490",
      "title": "Efficacy and Safety of Extended Release and Immediate Release Febuxostat in Participants With Gout and Moderate Renal Impairment",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Gout",
        "Moderate Renal Impairment"
      ],
      "interventions": [
        {
          "name": "Febuxostat IR",
          "type": "DRUG"
        },
        {
          "name": "Febuxostat XR",
          "type": "DRUG"
        },
        {
          "name": "Febuxostat placebo",
          "type": "DRUG"
        },
        {
          "name": "Colchicine",
          "type": "DRUG"
        },
        {
          "name": "Naproxen",
          "type": "DRUG"
        },
        {
          "name": "Lansoprazole",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Takeda",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 189,
      "start_date": "2014-05",
      "completion_date": "2015-10",
      "has_results": true,
      "last_update_posted_date": "2016-11-03",
      "last_synced_at": "2026-10-29T06:38:13.329Z",
      "location_count": 89,
      "location_summary": "Birmingham, Alabama • Muscle Shoals, Alabama • Phoenix, Arizona + 75 more",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "Muscle Shoals",
          "state": "Alabama"
        },
        {
          "city": "Phoenix",
          "state": "Arizona"
        },
        {
          "city": "Little Rock",
          "state": "Arkansas"
        },
        {
          "city": "Little Rock",
          "state": "Arkansas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02128490"
    },
    {
      "nct_id": "NCT05299710",
      "title": "Ovarian Tissue Cryopreservation in Pre-Pubertal (OTC-Pre Pubertal)",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Cancers"
      ],
      "interventions": [
        {
          "name": "Laparoscopic surgery",
          "type": "PROCEDURE"
        }
      ],
      "intervention_types": [
        "PROCEDURE"
      ],
      "sponsor": "Erin Rowell",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "11 Years",
        "sex": "FEMALE",
        "summary": "Up to 11 Years · Female only"
      },
      "enrollment_count": 250,
      "start_date": "2018-05-29",
      "completion_date": "2035-01",
      "has_results": false,
      "last_update_posted_date": "2026-09-03",
      "last_synced_at": "2026-10-29T06:38:13.329Z",
      "location_count": 1,
      "location_summary": "Chicago, Illinois",
      "locations": [
        {
          "city": "Chicago",
          "state": "Illinois"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05299710"
    }
  ]
}