16 completed, 6 recruiting, 6 terminated, 4 active not recruiting. 11 Phase 2, 8 Phase 3, 7 Phase 1, 3 Not applicable, 1 Phase 4.
Follow Gangliosidoses, GM2: new trials by email Literature Review of Gangliosidoses, GM2 trials Research Report on Gangliosidoses, GM2
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Long-Term Safety and Tolerability of Nizubaglustat in Participants With Rare Lysosomal Disorders AZ-3102 - Niemann-Pick Disease Type C, GM2 Gangliosidosis | Phase 2 / Phase 3 | Not yet recruiting | Azafaros B.V. | 218 | 2026-12-10 | 2026-10-21 |
| Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol) PLX-200 - Lysosomal Storage Disorders, Sandhoff Disease | Phase 2 | Not yet recruiting | Polaryx Therapeutics, Inc. | 24 | 2026-12-01 | 2026-07-31 |
| A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease AZ-3102 - GM2 Gangliosidosis, Niemann-Pick Type C Disease | Phase 2 | Active not recruiting | Azafaros B.V. | 21 | 2026-02-04 | 2026-10-21 |
| 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 AZ-3102, Placebo - Niemann-Pick Type C Disease, GM1 Gangliosidosis | Phase 3 | Recruiting | Azafaros B.V. | 147 | 2025-06-30 | 2026-09-24 |
| 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 AZ-3102, Placebo - Gangliosidoses, GM2, Gangliosidosis, GM1 | Phase 3 | Active not recruiting | Azafaros B.V. | 75 | 2025-06-30 | 2026-09-22 |
| Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease AXO-AAV-GM2 - GM2 Gangliosidosis, Tay Sachs Disease | Active not recruiting | Terence Flotte | 7 | 2024-09-17 | 2025-10-16 | |
| Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease AZ-3102 (Dose 1), Placebo, AZ-3102 (Dose 2) - GM2 Gangliosidosis, Niemann-Pick Disease, Type C | Phase 2 | Completed | Azafaros B.V. | 13 | 2023-04-24 | 2026-05-14 |
| GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO) GM1 Gangliosidosis, Sandhoff Disease | Completed | Azafaros B.V. | 31 | 2022-02-22 | 2025-09-29 | |
| First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis TSHA-101 - Infantile GM2 Gangliosidosis (Disorder) | Phase 1 / Phase 2 | Active not recruiting | Dr. Anupam Sehgal | 3 | 2021-03-12 | 2023-05-09 |
| A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease AXO-AAV-GM2 Starting Dose, AXO-AAV-GM2 Low Dose, AXO-AAV-GM2 Middle Dose - Tay-Sachs Disease, Sandhoff Disease | Phase 1 | Terminated | Terence Flotte | 9 | 2021-01-15 | 2025-09-02 |
| A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2 venglustat GZ402671, placebo - Tay-Sachs Disease, Sandhoff Disease | Phase 3 | Terminated | Genzyme, a Sanofi Company | 75 | 2020-06-29 | 2026-01-28 |
| Registry Gangliosidoses Gangliosidoses, GM1 Gangliosidosis | Unknown | SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt) | 40 | 2020-06-08 | 2020-11-12 | |
| Impact of Cardiopulmonary Bypass Flow on Renal Oxygenation, Blood Flow and Tubular Injury High CPB flow - Extracorporeal Circulation; Complications, Renal Failure | Not applicable | Completed | Sahlgrenska University Hospital | 100 | 2019-09-27 | 2023-11-22 |
| Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2 GM1 Gangliosidosis, GM2 Gangliosidosis | Completed | Idorsia Pharmaceuticals Ltd. | 226 | 2019-07-31 | 2021-11-08 | |
| N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) IB1001 - GM2 Gangliosidosis, Tay-Sachs Disease | Phase 2 | Completed | IntraBio Inc | 30 | 2019-06-07 | 2024-03-12 |
| Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD) Miglustat - GM2 Gangliosidosis, Supportive Care | Phase 3 | Terminated | Tehran University of Medical Sciences | 30 | 2019-01-14 | 2025-09-17 |
| LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies Leukodystrophy, White Matter Disease | Completed | Children's Hospital of Philadelphia | 236 | 2017-01-06 | 2025-11-10 | |
| The Myelin Disorders Biorepository Project Leukodystrophy, White Matter Disease | Recruiting | Children's Hospital of Philadelphia | 12000 | 2016-12-08 | 2025-10-23 | |
| A Natural History of Late Onset Tay-Sachs Disease GM2 Gangliosidosis | Completed | Massachusetts General Hospital | 10 | 2016-04-01 | 2026-03-02 | |
| Synergistic Enteral Regimen for Treatment of the Gangliosidoses miglustat, Ketogenic Diet - GM1 Gangliosidoses, GM2 Gangliosidoses | Phase 4 | Terminated | University of Minnesota | 16 | 2015-12-22 | 2021-04-14 |
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Sandhoff Disease (24) · Tay-Sachs Disease (24) · Gangliosidosis, GM1 (13) · Leukodystrophy, Globoid Cell (11) · Lysosomal Storage Diseases (9) · Adrenoleukodystrophy (8) · Leukodystrophy, Metachromatic (8) · Gangliosidoses (6) · Mucolipidoses (5) · Mucopolysaccharidosis III (5) · Pelizaeus-Merzbacher Disease (5) · Canavan Disease (4) · Gaucher Disease (4) · Metabolism, Inborn Errors (4)
Brain Diseases (37) · Brain Diseases, Metabolic (37) · Central Nervous System Diseases (37) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (37) · Lipid Metabolism Disorders (37) · Lipid Metabolism, Inborn Errors (37) · Lipidoses (37) · Lysosomal Storage Diseases, Nervous System (37) · Metabolic Diseases (37) · Nutritional and Metabolic Diseases (37) · Sphingolipidoses (37) · Brain Diseases, Metabolic, Inborn (36) · Genetic Diseases, Inborn (36) · Nervous System Diseases (36) · Metabolism, Inborn Errors (33)
miglustat (4) · Alemtuzumab (2) · Clofarabine (2) · Hematopoietic Stem Cell Transplantation (2) · Melphalan (2) · Pyrimethamine (2) · Stem Cell Transplantation (2) · Whole-Body Irradiation (2) · Antilymphocyte Serum (1) · Bone Marrow Transplantation (1) · Busulfan (1) · Cyclophosphamide (1) · Cyclosporine (1) · Diet, Ketogenic (1) · Gemfibrozil (1)
Azafaros B.V. (6) · Masonic Cancer Center, University of Minnesota (3) · University of Minnesota (3) · Children's Hospital of Philadelphia (2) · Joanne Kurtzberg, MD (2) · Terence Flotte (2) · The Hospital for Sick Children (2) · Aldagen (1) · Children's National Research Institute (1) · Dr. Anupam Sehgal (1) · Exsar Corporation (1) · Genzyme, a Sanofi Company (1) · Idorsia Pharmaceuticals Ltd. (1) · IntraBio Inc (1) · Massachusetts General Hospital (1)
United States (26) · Brazil (7) · Canada (7) · Germany (7) · France (5) · Italy (5) · Spain (5) · United Kingdom (5) · Portugal (4) · Argentina (3) · Sweden (3) · Switzerland (3) · Turkey (Türkiye) (3) · Australia (2) · India (2)
Joanne Kurtzberg, MD (3) · Adeline Vanderver, MD (2) · Jeanine R. Jarnes, PharmD (2) · Paul Orchard, MD (2) · Terence Flotte, MD (2) · Anupam Sehgal, MBBS (1) · Bijan Almassian, Ph.D. (1) · Chester B. Whitley, PhD, MD (1) · Cynthia J TIfft, MD, PhD (1) · Cynthia J Tifft, M.D. (1) · Florian Eichler, MD (1) · Guillaume Sillon, MSc (1) · James Hinson, MD (1)
PMID 21586746, PMID 28476546, PMID 40817303, PMID 19595619, PMID 25557439, PMID 28748536, PMID 29352662, PMID 30365961, PMID 32342562, PMID 32749716, PMID 32753397, PMID 33482890, PMID 34349180, PMID 34539759, PMID 36194207, PMID 36456200, PMID 36461157, PMID 36700853, PMID 37624739, PMID 38205442, PMID 38641994, PMID 41838934, PMID 42467089
U54NS065768 (3), 2019 (2), U01NS106845 (1), U54NS115052 (1)