{ "api_version": "1", "request": { "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Gangliosidosis&page=2", "query": { "condition": "Gangliosidosis", "page": 2 }, "page_size": 10 }, "pagination": { "page": 2, "page_size": 10, "total_count": 10, "total_pages": 1, "next_page_url": null, "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Gangliosidosis&page=1&page_size=10" }, "source": "remote", "last_synced_at": "2026-06-26T12:58:38.257Z", "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": "2026-06-26T12:58:38.257Z", "location_count": 1, "location_summary": "Bethesda, Maryland", "locations": [ { "city": "Bethesda", "state": "Maryland" } ], "official_url": "https://clinicaltrials.gov/study/NCT03952637" }, { "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": "2026-06-26T12:58:38.257Z", "location_count": 1, "location_summary": "Minneapolis, Minnesota", "locations": [ { "city": "Minneapolis", "state": "Minnesota" } ], "official_url": "https://clinicaltrials.gov/study/NCT00668187" }, { "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": "2026-06-26T12:58:38.257Z", "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": "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": "2026-06-26T12:58:38.257Z", "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": "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": "2026-06-26T12:58:38.257Z", "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": "NCT02699190", "title": "LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies", "overall_status": "COMPLETED", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Leukodystrophy", "White Matter Disease", "4H Syndrome", "Adrenoleukodystrophy", "AMN", "ALD", "ALD (Adrenoleukodystrophy)", "X-linked Adrenoleukodystrophy", "X-ALD", "Adrenomyeloneuropathy", "Aicardi Goutieres Syndrome", "AGS", "Alexander Disease", "Alexanders Leukodystrophy", "AxD", "ADLD", "Canavan Disease", "CTX", "Cerebrotendinous Xanthomatoses", "Krabbe Disease", "GALC Deficiency", "Globoid Leukodystrophy", "TUBB4A-Related Leukodystrophy", "H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum", "HBSL", "HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity", "LBSL", "Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder)", "Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation", "ALSP", "CSF1R Gene Mutation", "HCC - Hypomyelination and Congenital Cataract", "MLC1", "Megalencephalic Leukoencephalopathy With Subcortical Cysts 1", "MLD", "Metachromatic Leukodystrophy", "PMD", "Pelizaeus-Merzbacher Disease", "PLP1 Null Syndrome", "PLP1 Gene Duplication | Blood or Tissue | Mutations", "Pelizaeus-Merzbacher-Like Disease, 1", "Peroxisomal Biogenesis Disorder", "Zellweger Syndrome", "Refsum Disease", "Salla Disease", "Sialic Storage Disease", "Sjögren", "Sjogren-Larsson Syndrome", "Van Der Knapp Disease", "Vanishing White Matter Disease", "Charcot-Marie-Tooth", "CMT", "Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency", "Allan-Herndon-Dudley Syndrome", "Cadasil", "Cockayne Syndrome", "Multiple Sulfatase Deficiency", "Gangliosidoses", "GM2 Gangliosidosis", "BPAN", "Labrune Syndrome", "LCC", "Mucopolysaccharidoses", "TBCK-Related Intellectual Disability Syndrome" ], "interventions": [], "intervention_types": [], "sponsor": "Children's Hospital of Philadelphia", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": "18 Years", "sex": "ALL", "summary": "Up to 18 Years" }, "enrollment_count": 236, "start_date": "2017-01-06", "completion_date": "2024-10-31", "has_results": true, "last_update_posted_date": "2025-11-10", "last_synced_at": "2026-06-26T12:58:38.257Z", "location_count": 1, "location_summary": "Philadelphia, Pennsylvania", "locations": [ { "city": "Philadelphia", "state": "Pennsylvania" } ], "official_url": "https://clinicaltrials.gov/study/NCT02699190" }, { "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": "2026-06-26T12:58:38.257Z", "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": "NCT02851862", "title": "A Natural History of Late Onset Tay-Sachs Disease", "overall_status": "COMPLETED", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "GM2 Gangliosidosis" ], "interventions": [], "intervention_types": [], "sponsor": "Massachusetts General Hospital", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "7 Years", "maximum_age": null, "sex": "ALL", "summary": "7 Years and older" }, "enrollment_count": 10, "start_date": "2016-04", "completion_date": "2025-12", "has_results": false, "last_update_posted_date": "2026-03-02", "last_synced_at": "2026-06-26T12:58:38.257Z", "location_count": 1, "location_summary": "Boston, Massachusetts", "locations": [ { "city": "Boston", "state": "Massachusetts" } ], "official_url": "https://clinicaltrials.gov/study/NCT02851862" }, { "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": "2026-06-26T12:58:38.257Z", "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": "NCT04669535", "title": "A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease", "overall_status": "TERMINATED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1" ], "conditions": [ "Tay-Sachs Disease", "Sandhoff Disease" ], "interventions": [ { "name": "AXO-AAV-GM2 Starting Dose", "type": "BIOLOGICAL" }, { "name": "AXO-AAV-GM2 Low Dose", "type": "BIOLOGICAL" }, { "name": "AXO-AAV-GM2 Middle Dose", "type": "BIOLOGICAL" }, { "name": "AXO-AAV-GM2 High Dose", "type": "BIOLOGICAL" } ], "intervention_types": [ "BIOLOGICAL" ], "sponsor": "Terence Flotte", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "6 Months", "maximum_age": "12 Years", "sex": "ALL", "summary": "6 Months to 12 Years" }, "enrollment_count": 9, "start_date": "2021-01-15", "completion_date": "2024-12-16", "has_results": true, "last_update_posted_date": "2025-09-02", "last_synced_at": "2026-06-26T12:58:38.257Z", "location_count": 2, "location_summary": "Boston, Massachusetts • Worcester, Massachusetts", "locations": [ { "city": "Boston", "state": "Massachusetts" }, { "city": "Worcester", "state": "Massachusetts" } ], "official_url": "https://clinicaltrials.gov/study/NCT04669535" } ] }