{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Metabolism%2C+Inborn+Errors&page=19",
    "query": {
      "condition": "Metabolism, Inborn Errors",
      "page": 19
    },
    "page_size": 10
  },
  "pagination": {
    "page": 19,
    "page_size": 10,
    "total_count": 1608,
    "total_pages": 161,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Metabolism%2C+Inborn+Errors&page=20&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Metabolism%2C+Inborn+Errors&page=18&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-15T01:01:29.504Z",
  "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": "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": "2026-10-15T01:01:29.504Z",
      "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": "NCT01663896",
      "title": "Observational Study of OCT in a Patients Undergoing FFR",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Coronary Artery Disease"
      ],
      "interventions": [
        {
          "name": "OCT stent guidance",
          "type": "DEVICE"
        }
      ],
      "intervention_types": [
        "DEVICE"
      ],
      "sponsor": "Abbott Medical Devices",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 418,
      "start_date": "2012-12-11",
      "completion_date": "2015-10",
      "has_results": true,
      "last_update_posted_date": "2020-07-02",
      "last_synced_at": "2026-10-15T01:01:29.504Z",
      "location_count": 15,
      "location_summary": "Birmingham, Alabama • La Jolla, California • Orlando, Florida + 12 more",
      "locations": [
        {
          "city": "Birmingham",
          "state": "Alabama"
        },
        {
          "city": "La Jolla",
          "state": "California"
        },
        {
          "city": "Orlando",
          "state": "Florida"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Maywood",
          "state": "Illinois"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01663896"
    },
    {
      "nct_id": "NCT03952637",
      "title": "A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Lysosomal Diseases",
        "Gangliosidosis",
        "GM1"
      ],
      "interventions": [
        {
          "name": "AAV9-GLB1",
          "type": "BIOLOGICAL"
        },
        {
          "name": "Abdominal ultrasound",
          "type": "PROCEDURE"
        },
        {
          "name": "Rituximab",
          "type": "DRUG"
        },
        {
          "name": "Sirolimus",
          "type": "DRUG"
        },
        {
          "name": "Methylprednisolone",
          "type": "DRUG"
        },
        {
          "name": "Prednisone",
          "type": "DRUG"
        },
        {
          "name": "Audiology assessment with ABR",
          "type": "DIAGNOSTIC_TEST"
        },
        {
          "name": "Bone density scan (DEXA)",
          "type": "DIAGNOSTIC_TEST"
        },
        {
          "name": "Electrocardiogram (EKG)",
          "type": "DIAGNOSTIC_TEST"
        },
        {
          "name": "Echocardiogram",
          "type": "DIAGNOSTIC_TEST"
        },
        {
          "name": "Electroencephalogram (EEG) awake and extended overnight",
          "type": "OTHER"
        },
        {
          "name": "Laboratory tests",
          "type": "DIAGNOSTIC_TEST"
        },
        {
          "name": "Lumbar puncture",
          "type": "PROCEDURE"
        },
        {
          "name": "Brain MRI/MRS/fMRI",
          "type": "PROCEDURE"
        },
        {
          "name": "Neurocognitive testing",
          "type": "BEHAVIORAL"
        },
        {
          "name": "Neurology exam",
          "type": "OTHER"
        },
        {
          "name": "PICC or other Central line placement",
          "type": "BEHAVIORAL"
        },
        {
          "name": "Skeletal survey",
          "type": "PROCEDURE"
        },
        {
          "name": "Skin biopsy",
          "type": "PROCEDURE"
        },
        {
          "name": "Speech and modified barium swallow study",
          "type": "PROCEDURE"
        },
        {
          "name": "Ophthalmology exam",
          "type": "PROCEDURE"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL",
        "PROCEDURE",
        "DRUG",
        "DIAGNOSTIC_TEST",
        "OTHER",
        "BEHAVIORAL"
      ],
      "sponsor": "National Human Genome Research Institute (NHGRI)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Months",
        "maximum_age": "12 Years",
        "sex": "ALL",
        "summary": "6 Months to 12 Years"
      },
      "enrollment_count": 54,
      "start_date": "2019-08-19",
      "completion_date": "2030-01-01",
      "has_results": false,
      "last_update_posted_date": "2026-09-21",
      "last_synced_at": "2026-10-15T01:01:29.504Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03952637"
    },
    {
      "nct_id": "NCT03153319",
      "title": "Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Mucopolysaccharidosis I",
        "Mucopolysaccharidosis II",
        "Mucopolysaccharidosis VI"
      ],
      "interventions": [
        {
          "name": "Adalimumab Injection [Humira]",
          "type": "DRUG"
        },
        {
          "name": "Saline Solution for Injection",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "5 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "5 Years and older"
      },
      "enrollment_count": 14,
      "start_date": "2017-06-05",
      "completion_date": "2027-02",
      "has_results": false,
      "last_update_posted_date": "2026-02-02",
      "last_synced_at": "2026-10-15T01:01:29.504Z",
      "location_count": 1,
      "location_summary": "Torrance, California",
      "locations": [
        {
          "city": "Torrance",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03153319"
    },
    {
      "nct_id": "NCT05659901",
      "title": "Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Pelizaeus-Merzbacher Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Ionis Pharmaceuticals, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Months",
        "maximum_age": "17 Years",
        "sex": "MALE",
        "summary": "6 Months to 17 Years · Male only"
      },
      "enrollment_count": 32,
      "start_date": "2022-10-03",
      "completion_date": "2029-03",
      "has_results": false,
      "last_update_posted_date": "2026-10-02",
      "last_synced_at": "2026-10-15T01:01:29.504Z",
      "location_count": 3,
      "location_summary": "Atlanta, Georgia • Baltimore, Maryland • Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Baltimore",
          "state": "Maryland"
        },
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05659901"
    },
    {
      "nct_id": "NCT00106912",
      "title": "Study to Collect Data on Fabry Disease Patients With Enhanceable Alpha-Galactosidase A Activity",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Fabry Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "National Institute of Neurological Disorders and Stroke (NINDS)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "MALE",
        "summary": "18 Years and older · Male only"
      },
      "enrollment_count": 35,
      "start_date": "2005-03-28",
      "completion_date": "2008-03-27",
      "has_results": false,
      "last_update_posted_date": "2017-07-02",
      "last_synced_at": "2026-10-15T01:01:29.504Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00106912"
    },
    {
      "nct_id": "NCT05228145",
      "title": "Gene Therapy Study for Children With CLN5 Batten Disease",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Neuronal Ceroid Lipofuscinosis CLN5"
      ],
      "interventions": [
        {
          "name": "NGN-101",
          "type": "GENETIC"
        }
      ],
      "intervention_types": [
        "GENETIC"
      ],
      "sponsor": "Neurogene Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "3 Years",
        "maximum_age": "9 Years",
        "sex": "ALL",
        "summary": "3 Years to 9 Years"
      },
      "enrollment_count": 6,
      "start_date": "2022-01-31",
      "completion_date": "2028-11",
      "has_results": false,
      "last_update_posted_date": "2024-08-12",
      "last_synced_at": "2026-10-15T01:01:29.504Z",
      "location_count": 1,
      "location_summary": "Rochester, New York",
      "locations": [
        {
          "city": "Rochester",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05228145"
    },
    {
      "nct_id": "NCT01003912",
      "title": "Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases",
      "overall_status": "WITHDRAWN",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Lysosomal Storage Diseases",
        "Inborn Errors of Metabolism"
      ],
      "interventions": [
        {
          "name": "ALD-601",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Joanne Kurtzberg, MD",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "FEMALE",
        "summary": "18 Years and older · Female only"
      },
      "enrollment_count": 0,
      "start_date": "2009-10",
      "completion_date": "2017-03",
      "has_results": false,
      "last_update_posted_date": "2017-06-29",
      "last_synced_at": "2026-10-15T01:01:29.504Z",
      "location_count": 2,
      "location_summary": "Durham, North Carolina",
      "locations": [
        {
          "city": "Durham",
          "state": "North Carolina"
        },
        {
          "city": "Durham",
          "state": "North Carolina"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01003912"
    },
    {
      "nct_id": "NCT01461304",
      "title": "Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism",
      "overall_status": "NO_LONGER_AVAILABLE",
      "study_type": "EXPANDED_ACCESS",
      "phases": [],
      "conditions": [
        "Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency",
        "Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2)",
        "Mitochondrial Trifunctional Protein Deficiency",
        "Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency",
        "Glycogen Storage Disorders",
        "Pyruvate Carboxylase Deficiency Disease",
        "ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of",
        "Barth Syndrome"
      ],
      "interventions": [
        {
          "name": "triheptanoin",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Jerry Vockley, MD, PhD",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Month",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "1 Month and older"
      },
      "enrollment_count": null,
      "start_date": null,
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2021-12-10",
      "last_synced_at": "2026-10-15T01:01:29.504Z",
      "location_count": 1,
      "location_summary": "Pittsburgh, Pennsylvania",
      "locations": [
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01461304"
    },
    {
      "nct_id": "NCT03729362",
      "title": "A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Pompe Disease (Late-onset)"
      ],
      "interventions": [
        {
          "name": "Cipaglucosidase Alfa",
          "type": "BIOLOGICAL"
        },
        {
          "name": "Miglustat",
          "type": "DRUG"
        },
        {
          "name": "Alglucosidase Alfa",
          "type": "BIOLOGICAL"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL",
        "DRUG"
      ],
      "sponsor": "Amicus Therapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 125,
      "start_date": "2018-12-04",
      "completion_date": "2021-01-15",
      "has_results": true,
      "last_update_posted_date": "2025-09-11",
      "last_synced_at": "2026-10-15T01:01:29.504Z",
      "location_count": 25,
      "location_summary": "Phoenix, Arizona • Little Rock, Arkansas • Irvine, California + 21 more",
      "locations": [
        {
          "city": "Phoenix",
          "state": "Arizona"
        },
        {
          "city": "Little Rock",
          "state": "Arkansas"
        },
        {
          "city": "Irvine",
          "state": "California"
        },
        {
          "city": "Gainesville",
          "state": "Florida"
        },
        {
          "city": "Tampa",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03729362"
    }
  ]
}