{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Gangliosidosis%2C+GM1",
    "query": {
      "condition": "Gangliosidosis, GM1"
    },
    "page_size": 10
  },
  "pagination": {
    "page": 1,
    "page_size": 10,
    "total_count": 21,
    "total_pages": 3,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Gangliosidosis%2C+GM1&page=2&page_size=10",
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-06-26T10:05:42.899Z",
  "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": "NCT06539169",
      "title": "FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Alpha-Thalassemia",
        "Beta-Thalassemia",
        "Amyloidosis",
        "Amyotrophic Lateral Sclerosis",
        "Creutzfeld-Jakob Disease",
        "Cystic Fibrosis",
        "Duchenne Muscular Dystrophy",
        "Early-Onset Alzheimer Disease",
        "Ehlers-Danlos Syndrome",
        "Huntington Disease",
        "Gaucher Disease",
        "GM1 Gangliosidosis",
        "Myasthenia Gravis",
        "Pompe Disease",
        "Sickle Cell Disease",
        "Transthyretin Amyloid Cardiomyopathy",
        "Rare Diseases"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "xCures",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 1000,
      "start_date": "2024-06-10",
      "completion_date": "2026-06-10",
      "has_results": false,
      "last_update_posted_date": "2024-11-14",
      "last_synced_at": "2026-06-26T10:05:42.899Z",
      "location_count": 1,
      "location_summary": "Los Altos, California",
      "locations": [
        {
          "city": "Los Altos",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06539169"
    },
    {
      "nct_id": "NCT04713475",
      "title": "Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis",
      "overall_status": "ACTIVE_NOT_RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "GM1 Gangliosidosis",
        "GM1 Gangliosidosis, Type I",
        "GM1 Gangliosidosis, Type 2",
        "Beta-Galactosidase-1 (GLB1) Deficiency"
      ],
      "interventions": [
        {
          "name": "PBGM01",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Gemma Biotherapeutics",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Month",
        "maximum_age": "24 Months",
        "sex": "ALL",
        "summary": "1 Month to 24 Months"
      },
      "enrollment_count": 26,
      "start_date": "2021-03-17",
      "completion_date": "2029-02",
      "has_results": false,
      "last_update_posted_date": "2025-05-21",
      "last_synced_at": "2026-06-26T10:05:42.899Z",
      "location_count": 4,
      "location_summary": "Oakland, California • Minneapolis, Minnesota • New Brunswick, New Jersey + 1 more",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        },
        {
          "city": "New Brunswick",
          "state": "New Jersey"
        },
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04713475"
    },
    {
      "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": "2026-06-24",
      "last_synced_at": "2026-06-26T10:05:42.899Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00029965"
    },
    {
      "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": "2026-06-26T10:05:42.899Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01626092"
    },
    {
      "nct_id": "NCT04273269",
      "title": "A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "GM1 Gangliosidosis"
      ],
      "interventions": [
        {
          "name": "LYS-GM101",
          "type": "GENETIC"
        }
      ],
      "intervention_types": [
        "GENETIC"
      ],
      "sponsor": "LYSOGENE",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "3 Years",
        "sex": "ALL",
        "summary": "Up to 3 Years"
      },
      "enrollment_count": 5,
      "start_date": "2021-05-11",
      "completion_date": "2023-05-22",
      "has_results": false,
      "last_update_posted_date": "2023-06-09",
      "last_synced_at": "2026-06-26T10:05:42.899Z",
      "location_count": 1,
      "location_summary": "Orange, California",
      "locations": [
        {
          "city": "Orange",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04273269"
    },
    {
      "nct_id": "NCT07054515",
      "title": "A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Niemann-Pick Type C Disease",
        "GM1 Gangliosidosis",
        "GM2 Gangliosidosis"
      ],
      "interventions": [
        {
          "name": "AZ-3102",
          "type": "DRUG"
        },
        {
          "name": "Placebo",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Azafaros B.V.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "4 Years",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "4 Years and older"
      },
      "enrollment_count": 147,
      "start_date": "2025-06-30",
      "completion_date": "2027-11-04",
      "has_results": false,
      "last_update_posted_date": "2026-06-18",
      "last_synced_at": "2026-06-26T10:05:42.899Z",
      "location_count": 5,
      "location_summary": "Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        },
        {
          "city": "Rochester",
          "state": "Minnesota"
        },
        {
          "city": "Dallas",
          "state": "Texas"
        },
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT07054515"
    },
    {
      "nct_id": "NCT00383448",
      "title": "HSCT for High Risk Inherited Inborn Errors",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE2"
      ],
      "conditions": [
        "Adrenoleukodystrophy",
        "Metachromatic Leukodystrophy",
        "Globoid Cell Leukodystrophy",
        "Tay Sachs Disease",
        "Sandhoffs Disease",
        "Wolman Disease",
        "I-Cell Disease",
        "Sanfilippo Syndrome",
        "GM1 Gangliosidosis"
      ],
      "interventions": [
        {
          "name": "Clofarabine",
          "type": "DRUG"
        },
        {
          "name": "Total body Irradiation",
          "type": "PROCEDURE"
        },
        {
          "name": "Melphalan",
          "type": "DRUG"
        },
        {
          "name": "Hematopoietic Stem Cell Transplantation",
          "type": "BIOLOGICAL"
        },
        {
          "name": "Alemtuzumab",
          "type": "DRUG"
        },
        {
          "name": "mycophenylate mofetil",
          "type": "DRUG"
        },
        {
          "name": "Cyclosporine A",
          "type": "DEVICE"
        },
        {
          "name": "Hydroxyurea",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG",
        "PROCEDURE",
        "BIOLOGICAL",
        "DEVICE"
      ],
      "sponsor": "Masonic Cancer Center, University of Minnesota",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "70 Years",
        "sex": "ALL",
        "summary": "Up to 70 Years"
      },
      "enrollment_count": 38,
      "start_date": "2006-09",
      "completion_date": "2014-09",
      "has_results": true,
      "last_update_posted_date": "2019-07-11",
      "last_synced_at": "2026-06-26T10:05:42.899Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00383448"
    },
    {
      "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": "2026-06-26T10:05:42.899Z",
      "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": "NCT05109793",
      "title": "GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "GM1 Gangliosidosis",
        "Sandhoff Disease",
        "Tay-Sachs Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Azafaros B.V.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": "20 Years",
        "sex": "ALL",
        "summary": "2 Years to 20 Years"
      },
      "enrollment_count": 31,
      "start_date": "2022-02-22",
      "completion_date": "2025-09-16",
      "has_results": false,
      "last_update_posted_date": "2025-09-29",
      "last_synced_at": "2026-06-26T10:05:42.899Z",
      "location_count": 2,
      "location_summary": "Oakland, California • Rochester, Minnesota",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Rochester",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05109793"
    },
    {
      "nct_id": "NCT04470713",
      "title": "Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "GM1 Gangliosidosis",
        "GM2 Gangliosidosis",
        "Gaucher Disease, Type 2",
        "Tay-Sachs Disease",
        "AB Variant Gangliosidosis GM2",
        "Sandhoff Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Idorsia Pharmaceuticals Ltd.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "0 Months",
        "maximum_age": null,
        "sex": "ALL",
        "summary": "0 Months and older"
      },
      "enrollment_count": 226,
      "start_date": "2019-07-31",
      "completion_date": "2021-10-30",
      "has_results": false,
      "last_update_posted_date": "2021-11-08",
      "last_synced_at": "2026-06-26T10:05:42.899Z",
      "location_count": 3,
      "location_summary": "Chicago, Illinois • Rochester, Minnesota • Fairfax, Virginia",
      "locations": [
        {
          "city": "Chicago",
          "state": "Illinois"
        },
        {
          "city": "Rochester",
          "state": "Minnesota"
        },
        {
          "city": "Fairfax",
          "state": "Virginia"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04470713"
    }
  ]
}