{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=GM1+Gangliosidosis%2C+Type+2",
    "query": {
      "condition": "GM1 Gangliosidosis, Type 2"
    },
    "page_size": 10
  },
  "pagination": {
    "page": 1,
    "page_size": 10,
    "total_count": 8,
    "total_pages": 1,
    "next_page_url": null,
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-07-23T03:07:22.703Z",
  "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": "NCT03952637",
      "title": "A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1",
        "PHASE2"
      ],
      "conditions": [
        "Lysosomal Diseases",
        "Gangliosidosis",
        "GM1"
      ],
      "interventions": [
        {
          "name": "AAV9-GLB1",
          "type": "BIOLOGICAL"
        },
        {
          "name": "Abdominal ultrasound",
          "type": "PROCEDURE"
        },
        {
          "name": "Rituximab",
          "type": "DRUG"
        },
        {
          "name": "Sirolimus",
          "type": "DRUG"
        },
        {
          "name": "Methylprednisolone",
          "type": "DRUG"
        },
        {
          "name": "Prednisone",
          "type": "DRUG"
        },
        {
          "name": "Audiology assessment with ABR",
          "type": "DIAGNOSTIC_TEST"
        },
        {
          "name": "Bone density scan (DEXA)",
          "type": "DIAGNOSTIC_TEST"
        },
        {
          "name": "Electrocardiogram (EKG)",
          "type": "DIAGNOSTIC_TEST"
        },
        {
          "name": "Echocardiogram",
          "type": "DIAGNOSTIC_TEST"
        },
        {
          "name": "Electroencephalogram (EEG) awake and extended overnight",
          "type": "OTHER"
        },
        {
          "name": "Laboratory tests",
          "type": "DIAGNOSTIC_TEST"
        },
        {
          "name": "Lumbar puncture",
          "type": "PROCEDURE"
        },
        {
          "name": "Brain MRI/MRS/fMRI",
          "type": "PROCEDURE"
        },
        {
          "name": "Neurocognitive testing",
          "type": "BEHAVIORAL"
        },
        {
          "name": "Neurology exam",
          "type": "OTHER"
        },
        {
          "name": "PICC or other Central line placement",
          "type": "BEHAVIORAL"
        },
        {
          "name": "Skeletal survey",
          "type": "PROCEDURE"
        },
        {
          "name": "Skin biopsy",
          "type": "PROCEDURE"
        },
        {
          "name": "Speech and modified barium swallow study",
          "type": "PROCEDURE"
        },
        {
          "name": "Ophthalmology exam",
          "type": "PROCEDURE"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL",
        "PROCEDURE",
        "DRUG",
        "DIAGNOSTIC_TEST",
        "OTHER",
        "BEHAVIORAL"
      ],
      "sponsor": "National Human Genome Research Institute (NHGRI)",
      "sponsor_class": "NIH",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Months",
        "maximum_age": "12 Years",
        "sex": "ALL",
        "summary": "6 Months to 12 Years"
      },
      "enrollment_count": 54,
      "start_date": "2019-08-19",
      "completion_date": "2028-01-01",
      "has_results": false,
      "last_update_posted_date": "2025-12-11",
      "last_synced_at": "2026-07-23T03:07:22.703Z",
      "location_count": 1,
      "location_summary": "Bethesda, Maryland",
      "locations": [
        {
          "city": "Bethesda",
          "state": "Maryland"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03952637"
    },
    {
      "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": "2026-07-23T03:07:22.703Z",
      "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"
    },
    {
      "nct_id": "NCT04310163",
      "title": "Interviews and Video Capture in Patients With GM1 Gangliosidosis",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "GM1 Gangliosidosis"
      ],
      "interventions": [
        {
          "name": "Natural history",
          "type": "OTHER"
        }
      ],
      "intervention_types": [
        "OTHER"
      ],
      "sponsor": "LYSOGENE",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 25,
      "start_date": "2020-04-20",
      "completion_date": "2023-05-05",
      "has_results": false,
      "last_update_posted_date": "2023-06-08",
      "last_synced_at": "2026-07-23T03:07:22.703Z",
      "location_count": 1,
      "location_summary": "Plymouth, Massachusetts",
      "locations": [
        {
          "city": "Plymouth",
          "state": "Massachusetts"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04310163"
    },
    {
      "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": "2026-07-23T03:07:22.703Z",
      "location_count": 1,
      "location_summary": "Minneapolis, Minnesota",
      "locations": [
        {
          "city": "Minneapolis",
          "state": "Minnesota"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02030015"
    },
    {
      "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": "2026-06-30",
      "last_synced_at": "2026-07-23T03:07:22.703Z",
      "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": "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": "2026-07-23T03:07:22.703Z",
      "location_count": 1,
      "location_summary": "Los Altos, California",
      "locations": [
        {
          "city": "Los Altos",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06539169"
    },
    {
      "nct_id": "NCT05368038",
      "title": "ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program",
      "overall_status": "ENROLLING_BY_INVITATION",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Acid Sphingomyelinase Deficiency",
        "Ceroid Lipofuscinosis, Neuronal, 2",
        "Cerebrotendinous Xanthomatosis",
        "Fabry Disease",
        "GM1 Gangliosidosis",
        "Gaucher Disease",
        "Lysosomal Acid Lipase Deficiency",
        "Metachromatic Leukodystrophy",
        "Mucopolysaccharidosis II",
        "Mucopolysaccharidosis III-B",
        "Mucopolysaccharidosis IV A",
        "Mucopolysaccharidosis VI",
        "Mucopolysaccharidosis VII",
        "Niemann-Pick Disease, Type C"
      ],
      "interventions": [
        {
          "name": "Confirmatory Testing",
          "type": "DIAGNOSTIC_TEST"
        }
      ],
      "intervention_types": [
        "DIAGNOSTIC_TEST"
      ],
      "sponsor": "Albert Einstein College of Medicine",
      "sponsor_class": "OTHER",
      "healthy_volunteers": true,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "4 Weeks",
        "sex": "ALL",
        "summary": "Up to 4 Weeks"
      },
      "enrollment_count": 100000,
      "start_date": "2021-05-10",
      "completion_date": "2029-08-31",
      "has_results": false,
      "last_update_posted_date": "2025-09-12",
      "last_synced_at": "2026-07-23T03:07:22.703Z",
      "location_count": 9,
      "location_summary": "Brooklyn, New York • Manhasset, New York • New York, New York + 2 more",
      "locations": [
        {
          "city": "Brooklyn",
          "state": "New York"
        },
        {
          "city": "Brooklyn",
          "state": "New York"
        },
        {
          "city": "Manhasset",
          "state": "New York"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "New York",
          "state": "New York"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05368038"
    },
    {
      "nct_id": "NCT04041102",
      "title": "Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "GM1 Gangliosidosis"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "University of Pennsylvania",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 31,
      "start_date": "2020-06-12",
      "completion_date": "2024-05-01",
      "has_results": false,
      "last_update_posted_date": "2025-10-24",
      "last_synced_at": "2026-07-23T03:07:22.703Z",
      "location_count": 2,
      "location_summary": "Oakland, California • Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "Oakland",
          "state": "California"
        },
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT04041102"
    }
  ]
}