{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Leukodystrophies",
    "query": {
      "condition": "Leukodystrophies"
    },
    "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=Leukodystrophies&page=2&page_size=10",
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-07-24T04:15:15.613Z",
  "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": "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": "2026-07-24T04:15:15.613Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01626092"
    },
    {
      "nct_id": "NCT02843555",
      "title": "Natural History of the Leukodystrophies",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Leukodystrophies"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Baylor Research Institute",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 10,
      "start_date": "2019-01-23",
      "completion_date": "2019-01-23",
      "has_results": false,
      "last_update_posted_date": "2019-03-22",
      "last_synced_at": "2026-07-24T04:15:15.613Z",
      "location_count": 1,
      "location_summary": "Dallas, Texas",
      "locations": [
        {
          "city": "Dallas",
          "state": "Texas"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02843555"
    },
    {
      "nct_id": "NCT03639844",
      "title": "BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study",
      "overall_status": "NO_LONGER_AVAILABLE",
      "study_type": "EXPANDED_ACCESS",
      "phases": [],
      "conditions": [
        "Hurler Syndrome",
        "Inherited Metabolic Disorder",
        "Lysosomal Storage Disorder",
        "Metachromatic Leukodystrophy",
        "Inborn Errors of Metabolism"
      ],
      "interventions": [
        {
          "name": "rivogenlecleucel",
          "type": "BIOLOGICAL"
        },
        {
          "name": "rimiducid",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL",
        "DRUG"
      ],
      "sponsor": "Bellicum Pharmaceuticals",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": null,
      "eligibility": {
        "minimum_age": "3 Months",
        "maximum_age": "21 Years",
        "sex": "ALL",
        "summary": "3 Months to 21 Years"
      },
      "enrollment_count": null,
      "start_date": null,
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2020-10-05",
      "last_synced_at": "2026-07-24T04:15:15.613Z",
      "location_count": 2,
      "location_summary": "Los Angeles, California • Palo Alto, California",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Palo Alto",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03639844"
    },
    {
      "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": "2026-07-24T04:15:15.613Z",
      "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": "NCT04833907",
      "title": "rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease",
      "overall_status": "ENROLLING_BY_INVITATION",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Canavan Disease"
      ],
      "interventions": [
        {
          "name": "rAAV-Olig001-ASPA",
          "type": "DRUG"
        },
        {
          "name": "Levetiracetam",
          "type": "DRUG"
        },
        {
          "name": "Prednisone",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Myrtelle Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "3 Months",
        "maximum_age": "60 Months",
        "sex": "ALL",
        "summary": "3 Months to 60 Months"
      },
      "enrollment_count": 24,
      "start_date": "2021-04-01",
      "completion_date": "2027-08-31",
      "has_results": false,
      "last_update_posted_date": "2025-11-14",
      "last_synced_at": "2026-07-24T04:15:15.613Z",
      "location_count": 1,
      "location_summary": "Dayton, Ohio",
      "locations": [
        {
          "city": "Dayton",
          "state": "Ohio"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04833907"
    },
    {
      "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": "2026-07-24T04:15:15.613Z",
      "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": "NCT04628364",
      "title": "The Natural History of Metachromatic Leukodystrophy Study (HOME Study)",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Metachromatic Leukodystrophy"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "National Organization for Rare Disorders",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 21,
      "start_date": "2020-10-01",
      "completion_date": "2025-03-30",
      "has_results": false,
      "last_update_posted_date": "2025-06-08",
      "last_synced_at": "2026-07-24T04:15:15.613Z",
      "location_count": 1,
      "location_summary": "Danbury, Connecticut",
      "locations": [
        {
          "city": "Danbury",
          "state": "Connecticut"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04628364"
    },
    {
      "nct_id": "NCT07222371",
      "title": "An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Genetic Disease",
        "TUBB4A-Related Leukodystrophy"
      ],
      "interventions": [
        {
          "name": "nL-TUBB4-001",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "University of California, San Diego",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 1,
      "start_date": "2025-09-19",
      "completion_date": "2027-09",
      "has_results": false,
      "last_update_posted_date": "2025-10-29",
      "last_synced_at": "2026-07-24T04:15:15.613Z",
      "location_count": 1,
      "location_summary": "San Diego, California",
      "locations": [
        {
          "city": "San Diego",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07222371"
    },
    {
      "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": "2026-07-24T04:15:15.613Z",
      "location_count": 1,
      "location_summary": "Pittsburgh, Pennsylvania",
      "locations": [
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00787865"
    },
    {
      "nct_id": "NCT04126005",
      "title": "Natural History Study of Patients With Canavan Disease (CANinform Study)",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Canavan Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Aspa Therapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 67,
      "start_date": "2019-10-10",
      "completion_date": "2025-08-31",
      "has_results": false,
      "last_update_posted_date": "2026-04-17",
      "last_synced_at": "2026-07-24T04:15:15.613Z",
      "location_count": 2,
      "location_summary": "Oakland, California • Boston, Massachusetts",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Boston",
          "state": "Massachusetts"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04126005"
    }
  ]
}