{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Sandhoff+Disease",
    "query": {
      "condition": "Sandhoff Disease"
    },
    "page_size": 10
  },
  "pagination": {
    "page": 1,
    "page_size": 10,
    "total_count": 20,
    "total_pages": 2,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Sandhoff+Disease&page=2&page_size=10",
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-05-21T23:39:43.529Z",
  "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": "NCT04221451",
      "title": "A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Tay-Sachs Disease",
        "Sandhoff Disease"
      ],
      "interventions": [
        {
          "name": "venglustat GZ402671",
          "type": "DRUG"
        },
        {
          "name": "placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Genzyme, a Sanofi Company",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "2 Years and older"
      },
      "enrollment_count": 75,
      "start_date": "2020-06-29",
      "completion_date": "2024-12-26",
      "has_results": true,
      "last_update_posted_date": "2026-01-28",
      "last_synced_at": "2026-05-21T23:39:43.529Z",
      "location_count": 5,
      "location_summary": "Los Angeles, California • Atlanta, Georgia • Bethesda, Maryland + 2 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Bethesda",
          "state": "Maryland"
        },
        {
          "city": "Boston",
          "state": "Massachusetts"
        },
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04221451"
    },
    {
      "nct_id": "NCT00029965",
      "title": "Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Neurological Regression",
        "Myoclonus",
        "Cherry Red Spot",
        "Brain Atrophy"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "National Human Genome Research Institute (NHGRI)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Day",
        "maximum_age": "100 Years",
        "sex": "ALL",
        "summary": "1 Day to 100 Years"
      },
      "enrollment_count": 200,
      "start_date": "2002-02-06",
      "completion_date": null,
      "has_results": false,
      "last_update_posted_date": "2026-04-29",
      "last_synced_at": "2026-05-21T23:39:43.529Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00029965"
    },
    {
      "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": "2026-05-21T23:39:43.529Z",
      "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": "2026-05-21T23:39:43.529Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01626092"
    },
    {
      "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": "2026-05-21T23:39:43.529Z",
      "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": "NCT01869270",
      "title": "Gene Therapy for Tay-Sachs Disease",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Tay Sachs Disease",
        "Sandhoff Disease",
        "Late Onset Tay Sachs Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "University of Minnesota",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 4,
      "start_date": "2010-12",
      "completion_date": "2014-08",
      "has_results": false,
      "last_update_posted_date": "2014-12-04",
      "last_synced_at": "2026-05-21T23:39:43.529Z",
      "location_count": 2,
      "location_summary": "Tampa, Florida • Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Tampa",
          "state": "Florida"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01869270"
    },
    {
      "nct_id": "NCT02030015",
      "title": "Synergistic Enteral Regimen for Treatment of the Gangliosidoses",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE4"
      ],
      "conditions": [
        "GM1 Gangliosidoses",
        "GM2 Gangliosidoses",
        "Tay-Sachs Disease",
        "Sandhoff Disease"
      ],
      "interventions": [
        {
          "name": "miglustat",
          "type": "DRUG"
        },
        {
          "name": "Ketogenic Diet",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "DRUG",
        "OTHER"
      ],
      "sponsor": "University of Minnesota",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "204 Months",
        "sex": "ALL",
        "summary": "Up to 204 Months"
      },
      "enrollment_count": 16,
      "start_date": "2015-12-22",
      "completion_date": "2019-07-31",
      "has_results": true,
      "last_update_posted_date": "2021-04-14",
      "last_synced_at": "2026-05-21T23:39:43.529Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02030015"
    },
    {
      "nct_id": "NCT00654433",
      "title": "ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Inherited Metabolic Diseases",
        "Lysosomal Storage Disorders",
        "Peroxisomal Storage Diseases",
        "Inborn Errors of Metabolism",
        "Mucopolysaccharidosis"
      ],
      "interventions": [
        {
          "name": "ALD-101",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Aldagen",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "16 Years",
        "sex": "ALL",
        "summary": "Up to 16 Years"
      },
      "enrollment_count": 40,
      "start_date": "2008-03",
      "completion_date": "2011-11",
      "has_results": false,
      "last_update_posted_date": "2014-07-08",
      "last_synced_at": "2026-05-21T23:39:43.529Z",
      "location_count": 3,
      "location_summary": "Los Angeles, California • New York, New York • Durham, North Carolina",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Durham",
          "state": "North Carolina"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00654433"
    },
    {
      "nct_id": "NCT00668187",
      "title": "A Natural History Study of the Gangliosidoses",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Tay-Sachs Disease",
        "Sandhoff Disease",
        "Late Onset Tay-Sachs Disease",
        "GM1 Gangliosidosis",
        "GM2 Gangliosidosis"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "University of Minnesota",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 52,
      "start_date": "2010-12",
      "completion_date": "2027-03-01",
      "has_results": false,
      "last_update_posted_date": "2026-03-04",
      "last_synced_at": "2026-05-21T23:39:43.529Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00668187"
    },
    {
      "nct_id": "NCT03759665",
      "title": "N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "GM2 Gangliosidosis",
        "Tay-Sachs Disease",
        "Sandhoff Disease"
      ],
      "interventions": [
        {
          "name": "IB1001",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "IntraBio Inc",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "6 Years and older"
      },
      "enrollment_count": 30,
      "start_date": "2019-06-07",
      "completion_date": "2023-01-09",
      "has_results": true,
      "last_update_posted_date": "2024-03-12",
      "last_synced_at": "2026-05-21T23:39:43.529Z",
      "location_count": 3,
      "location_summary": "Los Angeles, California • Rochester, Minnesota • New York, New York",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Rochester",
          "state": "Minnesota"
        },
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03759665"
    }
  ]
}