{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Leukodystrophy",
    "query": {
      "condition": "Leukodystrophy"
    },
    "page_size": 10
  },
  "pagination": {
    "page": 1,
    "page_size": 10,
    "total_count": 58,
    "total_pages": 6,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Leukodystrophy&page=2&page_size=10",
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-05-21T23:33:34.333Z",
  "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": "NCT01594853",
      "title": "Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "NA"
      ],
      "conditions": [
        "X-linked Adrenoleukodystrophy"
      ],
      "interventions": [
        {
          "name": "exercise training",
          "type": "BEHAVIORAL"
        }
      ],
      "intervention_types": [
        "BEHAVIORAL"
      ],
      "sponsor": "Hugo W. Moser Research Institute at Kennedy Krieger, Inc.",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "21 Years",
        "maximum_age": "70 Years",
        "sex": "FEMALE",
        "summary": "21 Years to 70 Years · Female only"
      },
      "enrollment_count": 31,
      "start_date": "2012-05",
      "completion_date": "2014-10",
      "has_results": true,
      "last_update_posted_date": "2018-08-29",
      "last_synced_at": "2026-05-21T23:33:34.333Z",
      "location_count": 1,
      "location_summary": "Baltimore, Maryland",
      "locations": [
        {
          "city": "Baltimore",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01594853"
    },
    {
      "nct_id": "NCT00730314",
      "title": "Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Sickle Cell Disease",
        "Thalassemia",
        "Anemia",
        "Granuloma",
        "Wiskott-Aldrich Syndrome",
        "Chediak Higashi Syndrome",
        "Osteopetrosis",
        "Neutropenia",
        "Thrombocytopenia",
        "Hurler Disease",
        "Niemann-Pick Disease",
        "Fucosidosis"
      ],
      "interventions": [
        {
          "name": "Hematopoietic stem cell transplantation",
          "type": "PROCEDURE"
        }
      ],
      "intervention_types": [
        "PROCEDURE"
      ],
      "sponsor": "Children's Hospital Los Angeles",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "21 Years",
        "sex": "ALL",
        "summary": "Up to 21 Years"
      },
      "enrollment_count": 25,
      "start_date": "2008-08",
      "completion_date": "2015-08",
      "has_results": false,
      "last_update_posted_date": "2016-06-23",
      "last_synced_at": "2026-05-21T23:33:34.333Z",
      "location_count": 1,
      "location_summary": "Los Angeles, California",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00730314"
    },
    {
      "nct_id": "NCT03771898",
      "title": "A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Metachromatic Leukodystrophy (MLD)"
      ],
      "interventions": [
        {
          "name": "SHP611",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Shire",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Months",
        "maximum_age": "72 Months",
        "sex": "ALL",
        "summary": "6 Months to 72 Months"
      },
      "enrollment_count": 36,
      "start_date": "2019-05-13",
      "completion_date": "2026-05-01",
      "has_results": true,
      "last_update_posted_date": "2026-04-15",
      "last_synced_at": "2026-05-21T23:33:34.333Z",
      "location_count": 11,
      "location_summary": "Torrance, California • Aurora, Colorado • Atlanta, Georgia + 8 more",
      "locations": [
        {
          "city": "Torrance",
          "state": "California"
        },
        {
          "city": "Aurora",
          "state": "Colorado"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "Iowa City",
          "state": "Iowa"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03771898"
    },
    {
      "nct_id": "NCT04528355",
      "title": "Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Primary Immunodeficiency (PID)",
        "Congenital Bone Marrow Failure Syndromes",
        "Inherited Metabolic Disorders (IMD)",
        "Hereditary Anemias",
        "Inflammatory Conditions"
      ],
      "interventions": [
        {
          "name": "data collection",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Paul Szabolcs",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Months",
        "maximum_age": "60 Years",
        "sex": "ALL",
        "summary": "2 Months to 60 Years"
      },
      "enrollment_count": 50,
      "start_date": "2020-08-20",
      "completion_date": "2028-06-30",
      "has_results": false,
      "last_update_posted_date": "2026-01-13",
      "last_synced_at": "2026-05-21T23:33:34.333Z",
      "location_count": 1,
      "location_summary": "Pittsburgh, Pennsylvania",
      "locations": [
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04528355"
    },
    {
      "nct_id": "NCT04693598",
      "title": "Gene Transfer Clinical Trial for Krabbe Disease",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Krabbe Disease"
      ],
      "interventions": [
        {
          "name": "FBX-101",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Forge Biologics, Inc",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Day",
        "maximum_age": "12 Months",
        "sex": "ALL",
        "summary": "1 Day to 12 Months"
      },
      "enrollment_count": 6,
      "start_date": "2021-11-05",
      "completion_date": "2026-11",
      "has_results": false,
      "last_update_posted_date": "2025-01-30",
      "last_synced_at": "2026-05-21T23:33:34.333Z",
      "location_count": 1,
      "location_summary": "Ann Arbor, Michigan",
      "locations": [
        {
          "city": "Ann Arbor",
          "state": "Michigan"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04693598"
    },
    {
      "nct_id": "NCT02171104",
      "title": "MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Mucopolysaccharidosis Disorders",
        "Hurler Syndrome",
        "Hunter Syndrome",
        "Maroteaux Lamy Syndrome",
        "Sly Syndrome",
        "Alpha-Mannosidosis",
        "Fucosidosis",
        "Aspartylglucosaminuria",
        "Glycoprotein Metabolic Disorders",
        "Sphingolipidoses",
        "Recessive Leukodystrophies",
        "Globoid Cell Leukodystrophy",
        "Metachromatic Leukodystrophy",
        "Niemann-Pick B",
        "Niemann-Pick C Subtype 2",
        "Sphingomyelin Deficiency",
        "Peroxisomal Disorders",
        "Adrenoleukodystrophy With Cerebral Involvement",
        "Zellweger Syndrome",
        "Neonatal Adrenoleukodystrophy",
        "Infantile Refsum Disease",
        "Acyl-CoA Oxidase Deficiency",
        "D-Bifunctional Enzyme Deficiency",
        "Multifunctional Enzyme Deficiency",
        "Alpha-methylacyl-CoA Racmase Deficiency",
        "Mitochondrial Neurogastrointestingal Encephalopathy",
        "Severe Osteopetrosis",
        "Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation)",
        "Inherited Metabolic Disorders"
      ],
      "interventions": [
        {
          "name": "Stem Cell Transplantation",
          "type": "BIOLOGICAL"
        },
        {
          "name": "IMD Preparative Regimen",
          "type": "DRUG"
        },
        {
          "name": "Osteopetrosis Only Preparative Regimen",
          "type": "DRUG"
        },
        {
          "name": "Osteopetrosis Haploidentical Only Preparative Regimen",
          "type": "DRUG"
        },
        {
          "name": "cALD SR-A (Standard-Risk, Regimen A)",
          "type": "DRUG"
        },
        {
          "name": "cALD SR-B (Standard-Risk, Regimen B)",
          "type": "DRUG"
        },
        {
          "name": "cALD HR-D (High-Risk, Regimen C)",
          "type": "DRUG"
        },
        {
          "name": "cALD HR-D (High-Risk, Regimen D)",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL",
        "DRUG"
      ],
      "sponsor": "Masonic Cancer Center, University of Minnesota",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "55 Years",
        "sex": "ALL",
        "summary": "Up to 55 Years"
      },
      "enrollment_count": 149,
      "start_date": "2014-07-10",
      "completion_date": "2029-07-14",
      "has_results": false,
      "last_update_posted_date": "2026-01-07",
      "last_synced_at": "2026-05-21T23:33:34.333Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02171104"
    },
    {
      "nct_id": "NCT03639285",
      "title": "Natural History, Diagnosis, and Outcomes for Leukodystrophies",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Leukodystrophy"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "University of Utah",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 600,
      "start_date": "2007-01-19",
      "completion_date": "2050-12-31",
      "has_results": false,
      "last_update_posted_date": "2026-01-13",
      "last_synced_at": "2026-05-21T23:33:34.333Z",
      "location_count": 1,
      "location_summary": "Salt Lake City, Utah",
      "locations": [
        {
          "city": "Salt Lake City",
          "state": "Utah"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03639285"
    },
    {
      "nct_id": "NCT04008849",
      "title": "A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456",
      "overall_status": "TERMINATED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Inherited Metabolic Disorders (IMD)"
      ],
      "interventions": [
        {
          "name": "Safety and efficacy assessments",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "Magenta Therapeutics, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "16 Years",
        "sex": "ALL",
        "summary": "Up to 16 Years"
      },
      "enrollment_count": 3,
      "start_date": "2019-05-21",
      "completion_date": "2020-10-08",
      "has_results": false,
      "last_update_posted_date": "2021-03-15",
      "last_synced_at": "2026-05-21T23:33:34.333Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04008849"
    },
    {
      "nct_id": "NCT03333200",
      "title": "Longitudinal Study of Neurodegenerative Disorders",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "MLD",
        "Krabbe Disease",
        "ALD",
        "MPS I",
        "MPS II",
        "MPS III",
        "Vanishing White Matter Disease",
        "GM3 Gangliosidosis",
        "PKAN",
        "Tay-Sachs Disease",
        "NP Deficiency",
        "Osteopetrosis",
        "Alpha-Mannosidosis",
        "Sandhoff Disease",
        "Niemann-Pick Diseases",
        "MPS IV",
        "Gaucher Disease",
        "GAN",
        "GM1 Gangliosidoses",
        "Morquio Disease",
        "S-Adenosylhomocysteine Hydrolase Deficiency",
        "Batten Disease",
        "Pelizaeus-Merzbacher Disease",
        "Leukodystrophy",
        "Lysosomal Storage Diseases",
        "Purine Nucleoside Phosphorylase Deficiency",
        "Multiple Sulfatase Deficiency Disease"
      ],
      "interventions": [
        {
          "name": "Palliative Care",
          "type": "OTHER"
        },
        {
          "name": "Hematopoetic Stem Cell Transplantation",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "OTHER",
        "BIOLOGICAL"
      ],
      "sponsor": "University of Pittsburgh",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 1500,
      "start_date": "2012-01-11",
      "completion_date": "2035-01",
      "has_results": false,
      "last_update_posted_date": "2026-02-09",
      "last_synced_at": "2026-05-21T23:33:34.333Z",
      "location_count": 1,
      "location_summary": "Pittsburgh, Pennsylvania",
      "locations": [
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03333200"
    },
    {
      "nct_id": "NCT01626092",
      "title": "Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "NA"
      ],
      "conditions": [
        "Lysosomal Storage Disease",
        "Peroxisomal Disorder"
      ],
      "interventions": [
        {
          "name": "Campath-1H",
          "type": "DRUG"
        },
        {
          "name": "Clofarabine",
          "type": "DRUG"
        },
        {
          "name": "Melphalan",
          "type": "DRUG"
        },
        {
          "name": "Total Body Irradiation with Marrow Boosting",
          "type": "RADIATION"
        },
        {
          "name": "Hematopoietic stem cell transplantation",
          "type": "BIOLOGICAL"
        },
        {
          "name": "Cyclosporine A",
          "type": "DRUG"
        },
        {
          "name": "Mycophenolate mofetil",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG",
        "RADIATION",
        "BIOLOGICAL"
      ],
      "sponsor": "Masonic Cancer Center, University of Minnesota",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "55 Years",
        "sex": "ALL",
        "summary": "Up to 55 Years"
      },
      "enrollment_count": 3,
      "start_date": "2012-07-11",
      "completion_date": "2013-11",
      "has_results": true,
      "last_update_posted_date": "2017-12-05",
      "last_synced_at": "2026-05-21T23:33:34.333Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01626092"
    }
  ]
}