{ "api_version": "1", "request": { "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Gangliosidoses&page=2", "query": { "condition": "Gangliosidoses", "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=Gangliosidoses&page=1&page_size=10" }, "source": "remote", "last_synced_at": "2026-06-26T08:39:32.076Z", "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": "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": "2026-06-26T08:39:32.076Z", "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": "2026-06-26T08:39:32.076Z", "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": "2026-06-26T08:39:32.076Z", "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": "NCT06614569", "title": "Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease", "overall_status": "ACTIVE_NOT_RECRUITING", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "GM2 Gangliosidosis", "Tay Sachs Disease", "Sandhoff Disease" ], "interventions": [ { "name": "AXO-AAV-GM2", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Terence Flotte", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": null, "sex": "ALL", "summary": "Not listed" }, "enrollment_count": 7, "start_date": "2024-09-17", "completion_date": "2028-12", "has_results": false, "last_update_posted_date": "2025-10-16", "last_synced_at": "2026-06-26T08:39:32.076Z", "location_count": 1, "location_summary": "Worcester, Massachusetts", "locations": [ { "city": "Worcester", "state": "Massachusetts" } ], "official_url": "https://clinicaltrials.gov/study/NCT06614569" }, { "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": "2026-06-26T08:39:32.076Z", "location_count": 1, "location_summary": "Boston, Massachusetts", "locations": [ { "city": "Boston", "state": "Massachusetts" } ], "official_url": "https://clinicaltrials.gov/study/NCT02851862" }, { "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": "2026-06-26T08:39:32.076Z", "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": "NCT02254863", "title": "UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells", "overall_status": "RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1" ], "conditions": [ "Adrenoleukodystrophy", "Batten Disease", "Mucopolysaccharidosis II", "Leukodystrophy, Globoid Cell", "Leukodystrophy, Metachromatic", "Neimann Pick Disease", "Pelizaeus-Merzbacher Disease", "Sandhoff Disease", "Tay-Sachs Disease", "Brain Diseases, Metabolic, Inborn", "Alpha-Mannosidosis", "Sanfilippo Mucopolysaccharidoses" ], "interventions": [ { "name": "DUOC-01", "type": "BIOLOGICAL" } ], "intervention_types": [ "BIOLOGICAL" ], "sponsor": "Joanne Kurtzberg, MD", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "1 Week", "maximum_age": "22 Years", "sex": "ALL", "summary": "1 Week to 22 Years" }, "enrollment_count": 40, "start_date": "2014-09", "completion_date": "2026-10", "has_results": false, "last_update_posted_date": "2025-09-08", "last_synced_at": "2026-06-26T08:39:32.076Z", "location_count": 1, "location_summary": "Durham, North Carolina", "locations": [ { "city": "Durham", "state": "North Carolina" } ], "official_url": "https://clinicaltrials.gov/study/NCT02254863" }, { "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": "2026-06-26T08:39:32.076Z", "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": "NCT03047369", "title": "The Myelin Disorders Biorepository Project", "overall_status": "RECRUITING", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Leukodystrophy", "White Matter Disease", "Leukoencephalopathies", "4H Syndrome", "Adrenoleukodystrophy", "AMN", "ALD", "ALD Gene Mutation", "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", "MLD", "Metachromatic Leukodystrophy", "PMD", "Pelizaeus-Merzbacher Disease", "PLP1 Null Syndrome", "PLP1 Gene Duplication | Blood or Tissue | Mutations", "Pelizaeus Merzbacher Like Disease", "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": null, "sex": "ALL", "summary": "Not listed" }, "enrollment_count": 12000, "start_date": "2016-12-08", "completion_date": "2030-12-08", "has_results": false, "last_update_posted_date": "2025-10-23", "last_synced_at": "2026-06-26T08:39:32.076Z", "location_count": 23, "location_summary": "Los Angeles, California • Orange, California • Palo Alto, California + 18 more", "locations": [ { "city": "Los Angeles", "state": "California" }, { "city": "Orange", "state": "California" }, { "city": "Palo Alto", "state": "California" }, { "city": "Sacramento", "state": "California" }, { "city": "San Diego", "state": "California" } ], "official_url": "https://clinicaltrials.gov/study/NCT03047369" }, { "nct_id": "NCT07082543", "title": "A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis", "overall_status": "RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE3" ], "conditions": [ "Gangliosidoses, GM2", "Gangliosidosis, GM1" ], "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": 75, "start_date": "2025-06-30", "completion_date": "2027-11-04", "has_results": false, "last_update_posted_date": "2026-06-18", "last_synced_at": "2026-06-26T08:39:32.076Z", "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/NCT07082543" } ] }