{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=15",
    "query": {
      "condition": "Inborn Errors of Metabolism Disorders",
      "page": 15
    },
    "page_size": 10
  },
  "pagination": {
    "page": 15,
    "page_size": 10,
    "total_count": 1510,
    "total_pages": 151,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=16&page_size=10",
    "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Inborn+Errors+of+Metabolism+Disorders&page=14&page_size=10"
  },
  "source": "remote",
  "last_synced_at": "2026-10-23T00:08:58.372Z",
  "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": "NCT00076323",
      "title": "A Test to Predict the Hemolytic Potential of Drugs in G6PD Deficiency",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "NA"
      ],
      "conditions": [
        "Glucosephosphate Dehydrogenase Deficiency",
        "Favism"
      ],
      "interventions": [
        {
          "name": "Primaquine",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Walter Reed Army Institute of Research (WRAIR)",
      "sponsor_class": "FED",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "60 Years",
        "sex": "ALL",
        "summary": "18 Years to 60 Years"
      },
      "enrollment_count": 14,
      "start_date": "2003-12",
      "completion_date": "2006-11",
      "has_results": false,
      "last_update_posted_date": "2010-07-27",
      "last_synced_at": "2026-10-23T00:08:58.372Z",
      "location_count": 1,
      "location_summary": "Silver Spring, Maryland",
      "locations": [
        {
          "city": "Silver Spring",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00076323"
    },
    {
      "nct_id": "NCT04977388",
      "title": "NORTHERA (DROXIDOPA) for Dysautonomia in Adult Survivors of Menkes Disease and Occipital Horn Syndrome",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Menkes Disease",
        "Occipital Horn Syndrome"
      ],
      "interventions": [
        {
          "name": "Droxidopa",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "DRUG",
        "OTHER"
      ],
      "sponsor": "Stephen G. Kaler, MD",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": "50 Years",
        "sex": "ALL",
        "summary": "18 Years to 50 Years"
      },
      "enrollment_count": 3,
      "start_date": "2021-07-12",
      "completion_date": "2024-06-29",
      "has_results": true,
      "last_update_posted_date": "2026-04-13",
      "last_synced_at": "2026-10-23T00:08:58.372Z",
      "location_count": 1,
      "location_summary": "New York, New York",
      "locations": [
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04977388"
    },
    {
      "nct_id": "NCT03533673",
      "title": "AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Pompe Disease"
      ],
      "interventions": [
        {
          "name": "ACTUS-101",
          "type": "GENETIC"
        }
      ],
      "intervention_types": [
        "GENETIC"
      ],
      "sponsor": "AskBio Inc",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 7,
      "start_date": "2018-12-17",
      "completion_date": "2026-03-12",
      "has_results": false,
      "last_update_posted_date": "2026-04-13",
      "last_synced_at": "2026-10-23T00:08:58.372Z",
      "location_count": 1,
      "location_summary": "Durham, North Carolina",
      "locations": [
        {
          "city": "Durham",
          "state": "North Carolina"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03533673"
    },
    {
      "nct_id": "NCT00887562",
      "title": "Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis & Stroke-like Episodes",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "MELAS Syndrome"
      ],
      "interventions": [
        {
          "name": "Idebenone",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "DRUG",
        "OTHER"
      ],
      "sponsor": "Michio Hirano",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "8 Years",
        "maximum_age": "65 Years",
        "sex": "ALL",
        "summary": "8 Years to 65 Years"
      },
      "enrollment_count": 27,
      "start_date": "2009-05",
      "completion_date": "2012-07",
      "has_results": true,
      "last_update_posted_date": "2016-10-26",
      "last_synced_at": "2026-10-23T00:08:58.372Z",
      "location_count": 1,
      "location_summary": "New York, New York",
      "locations": [
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00887562"
    },
    {
      "nct_id": "NCT03047369",
      "title": "The Myelin Disorders Biorepository Project",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Leukodystrophy",
        "White Matter Disease",
        "Leukoencephalopathies",
        "4H Syndrome",
        "Adrenoleukodystrophy",
        "AMN",
        "ALD",
        "ALD Gene Mutation",
        "ALD (Adrenoleukodystrophy)",
        "X-linked Adrenoleukodystrophy",
        "X-ALD",
        "Adrenomyeloneuropathy",
        "Aicardi Goutieres Syndrome",
        "AGS",
        "Alexander Disease",
        "Alexanders Leukodystrophy",
        "AxD",
        "ADLD",
        "Canavan Disease",
        "CTX",
        "Cerebrotendinous Xanthomatoses",
        "Krabbe Disease",
        "GALC Deficiency",
        "Globoid Leukodystrophy",
        "TUBB4A-Related Leukodystrophy",
        "H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum",
        "HBSL",
        "HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity",
        "LBSL",
        "Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder)",
        "Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation",
        "ALSP",
        "CSF1R Gene Mutation",
        "HCC - Hypomyelination and Congenital Cataract",
        "MLC1",
        "Megalencephalic Leukoencephalopathy With Subcortical Cysts",
        "MLD",
        "Metachromatic Leukodystrophy",
        "PMD",
        "Pelizaeus-Merzbacher Disease",
        "PLP1 Null Syndrome",
        "PLP1 Gene Duplication | Blood or Tissue | Mutations",
        "Pelizaeus Merzbacher Like Disease",
        "Peroxisomal Biogenesis Disorder",
        "Zellweger Syndrome",
        "Refsum Disease",
        "Salla Disease",
        "Sialic Storage Disease",
        "Sjögren",
        "Sjogren-Larsson Syndrome",
        "Van Der Knapp Disease",
        "Vanishing White Matter Disease",
        "Charcot-Marie-Tooth",
        "CMT",
        "Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency",
        "Allan-Herndon-Dudley Syndrome",
        "Cadasil",
        "Cockayne Syndrome",
        "Multiple Sulfatase Deficiency",
        "Gangliosidoses",
        "GM2 Gangliosidosis",
        "BPAN",
        "Labrune Syndrome",
        "LCC",
        "Mucopolysaccharidoses",
        "TBCK-Related Intellectual Disability Syndrome"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Children's Hospital of Philadelphia",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 12000,
      "start_date": "2016-12-08",
      "completion_date": "2030-12-08",
      "has_results": false,
      "last_update_posted_date": "2025-10-23",
      "last_synced_at": "2026-10-23T00:08:58.372Z",
      "location_count": 23,
      "location_summary": "Los Angeles, California • Orange, California • Palo Alto, California + 18 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "Palo Alto",
          "state": "California"
        },
        {
          "city": "Sacramento",
          "state": "California"
        },
        {
          "city": "San Diego",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03047369"
    },
    {
      "nct_id": "NCT01451879",
      "title": "Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Pompe Disease"
      ],
      "interventions": [
        {
          "name": "Rituximab",
          "type": "DRUG"
        },
        {
          "name": "Miglustat",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "University of Florida",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "65 Years",
        "sex": "ALL",
        "summary": "Up to 65 Years"
      },
      "enrollment_count": 11,
      "start_date": "2008-10",
      "completion_date": "2017-10",
      "has_results": false,
      "last_update_posted_date": "2017-12-08",
      "last_synced_at": "2026-10-23T00:08:58.372Z",
      "location_count": 1,
      "location_summary": "Gainesville, Florida",
      "locations": [
        {
          "city": "Gainesville",
          "state": "Florida"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01451879"
    },
    {
      "nct_id": "NCT03687242",
      "title": "Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Congenital Adrenal Hyperplasia",
        "CAH - Congenital Adrenal Hyperplasia",
        "CAH - 21-Hydroxylase Deficiency"
      ],
      "interventions": [
        {
          "name": "SPR001",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Spruce Biosciences",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 11,
      "start_date": "2018-09-06",
      "completion_date": "2019-08-09",
      "has_results": true,
      "last_update_posted_date": "2025-04-01",
      "last_synced_at": "2026-10-23T00:08:58.372Z",
      "location_count": 8,
      "location_summary": "Orange, California • San Diego, California • Atlanta, Georgia + 5 more",
      "locations": [
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "San Diego",
          "state": "California"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Indianapolis",
          "state": "Indiana"
        },
        {
          "city": "Ann Arbor",
          "state": "Michigan"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03687242"
    },
    {
      "nct_id": "NCT00065299",
      "title": "Low Phenylalanine Diet for Mothers With Phenylketonuria (PKU)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "NA"
      ],
      "conditions": [
        "Phenylketonuria"
      ],
      "interventions": [
        {
          "name": "Restricted phenylalanine diet",
          "type": "BEHAVIORAL"
        }
      ],
      "intervention_types": [
        "BEHAVIORAL"
      ],
      "sponsor": "Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)",
      "sponsor_class": "NIH",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "0 Years",
        "maximum_age": null,
        "sex": "FEMALE",
        "summary": "0 Years and older · Female only"
      },
      "enrollment_count": 572,
      "start_date": "1984-05",
      "completion_date": "2000-04",
      "has_results": false,
      "last_update_posted_date": "2005-06-24",
      "last_synced_at": "2026-10-23T00:08:58.372Z",
      "location_count": 3,
      "location_summary": "Los Angeles, California • Chicago, Illinois • Galveston, Texas",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "Galveston",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00065299"
    },
    {
      "nct_id": "NCT00668564",
      "title": "Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Hurler's Syndrome",
        "Maroteaux-Lamy Syndrome",
        "Sly Syndrome",
        "Alpha Mannosidosis",
        "Fucosidosis",
        "Aspartylglucosaminuria",
        "Sphingolipidoses",
        "Krabbe Disease",
        "Wolman's Disease",
        "Niemann-Pick Disease Type B",
        "Niemann-Pick Disease, Type C"
      ],
      "interventions": [
        {
          "name": "Stem Cell Transplantation",
          "type": "PROCEDURE"
        },
        {
          "name": "Cyclophosphamide",
          "type": "DRUG"
        },
        {
          "name": "Campath-1H",
          "type": "DRUG"
        },
        {
          "name": "Busulfan",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "PROCEDURE",
        "DRUG"
      ],
      "sponsor": "Masonic Cancer Center, University of Minnesota",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "21 Years",
        "sex": "ALL",
        "summary": "Up to 21 Years"
      },
      "enrollment_count": 18,
      "start_date": "2008-03",
      "completion_date": "2010-02",
      "has_results": true,
      "last_update_posted_date": "2017-12-28",
      "last_synced_at": "2026-10-23T00:08:58.372Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00668564"
    },
    {
      "nct_id": "NCT01498263",
      "title": "Inherited Diseases, Caregiving, and Social Networks",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Undiagnosed Disease",
        "Healthy Volunteer (Adult With Typically Developing Child)",
        "Inherited Neurodegenerative Disorders",
        "Inherited Metabolic Disorders (Inborn Errors of Metabolism)",
        "Alzheimer's Disease and Related Dementias"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "National Human Genome Research Institute (NHGRI)",
      "sponsor_class": "NIH",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "18 Years and older"
      },
      "enrollment_count": 682,
      "start_date": "2012-01-09",
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2026-10-02",
      "last_synced_at": "2026-10-23T00:08:58.372Z",
      "location_count": 2,
      "location_summary": "Bethesda, Maryland • Memphis, Tennessee",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        },
        {
          "city": "Memphis",
          "state": "Tennessee"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01498263"
    }
  ]
}