18 completed, 8 terminated, 7 recruiting, 5 active not recruiting. 14 Phase 2, 11 Phase 1, 9 Phase 3, 3 Not applicable, 1 Phase 4.
Follow Gangliosidoses: new trials by email Literature Review of Gangliosidoses trials Research Report on Gangliosidoses
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 |
|---|---|---|---|---|---|---|
| Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial Gene Transfer with an AAV9 Vector Expressing Human ß-galactosidase - GM1 Gangliosidoses, GM1 Gangliosidosis, Type I | Phase 1 | Not yet recruiting | Tippi Mackenzie | 5 | 2027-01-01 | 2026-08-31 |
| 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 |
| Translational Potential of ex Vivo Gene Therapy in GM2 Gangliosidosis blood sample - Tay-Sachs Disease Ganglioside, Sandhoff Disease Ganglioside | Not yet recruiting | Assistance Publique - Hôpitaux de Paris | 6 | 2026-05-01 | 2026-03-06 | |
| 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 | |
| FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases Alpha-Thalassemia, Beta-Thalassemia | Unknown | xCures | 1000 | 2024-06-10 | 2024-11-14 | |
| 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 | |
| A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis LYS-GM101 - GM1 Gangliosidosis | Phase 1 / Phase 2 | Terminated | LYSOGENE | 5 | 2021-05-11 | 2023-06-09 |
| ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program Confirmatory Testing - Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2 | Enrolling by invitation | Albert Einstein College of Medicine | 100000 | 2021-05-10 | 2026-07-28 | |
| Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis PBGM01 - GM1 Gangliosidosis, GM1 Gangliosidosis, Type I | Phase 1 / Phase 2 | Active not recruiting | Gemma Biotherapeutics | 26 | 2021-03-17 | 2026-06-30 |
| 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 |
| Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients GM1 Gangliosidosis | Completed | University of Pennsylvania | 31 | 2020-06-12 | 2025-10-24 | |
| Registry Gangliosidoses Gangliosidoses, GM1 Gangliosidosis | Unknown | SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt) | 40 | 2020-06-08 | 2020-11-12 | |
| Natural History of Morquio B and Late-Onset of GM1 Gangliosidosis Morquio B Disease, GM1 Gangliosidosis Type III | Unknown | University of British Columbia | 30 | 2020-06-01 | 2020-04-30 |
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Gangliosidoses, GM2 (26) · Sandhoff Disease (24) · Tay-Sachs Disease (24) · Gangliosidosis, GM1 (22) · Lysosomal Storage Diseases (14) · Leukodystrophy, Globoid Cell (11) · Leukodystrophy, Metachromatic (9) · Adrenoleukodystrophy (8) · Gaucher Disease (7) · Mucopolysaccharidosis III (6) · Mucolipidoses (5) · Niemann-Pick Disease, Type C (5) · Pelizaeus-Merzbacher Disease (5) · Canavan Disease (4)
Brain Diseases (50) · Brain Diseases, Metabolic (50) · Central Nervous System Diseases (50) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (50) · Lipid Metabolism Disorders (50) · Lipid Metabolism, Inborn Errors (50) · Lipidoses (50) · Lysosomal Storage Diseases, Nervous System (50) · Metabolic Diseases (50) · Nutritional and Metabolic Diseases (50) · Sphingolipidoses (50) · Brain Diseases, Metabolic, Inborn (49) · Genetic Diseases, Inborn (49) · Nervous System Diseases (49) · Metabolism, Inborn Errors (46)
miglustat (4) · Alemtuzumab (2) · Clofarabine (2) · Hematopoietic Stem Cell Transplantation (2) · Melphalan (2) · Pyrimethamine (2) · Stem Cell Transplantation (2) · Whole-Body Irradiation (2) · Absorptiometry, Photon (1) · Antilymphocyte Serum (1) · Blood Specimen Collection (1) · Bone Marrow Transplantation (1) · Busulfan (1) · Catheterization, Peripheral (1) · Clinical Laboratory Techniques (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) · LYSOGENE (2) · National Human Genome Research Institute (NHGRI) (2) · Terence Flotte (2) · The Hospital for Sick Children (2) · Albert Einstein College of Medicine (1) · Aldagen (1) · Assistance Publique - Hôpitaux de Paris (1) · CENTOGENE GmbH Rostock (1) · Children's National Research Institute (1) · Dr. Anupam Sehgal (1)
United States (34) · Brazil (9) · Canada (9) · Germany (8) · United Kingdom (8) · France (7) · Italy (5) · Spain (5) · Turkey (Türkiye) (5) · Portugal (4) · Argentina (3) · India (3) · Sweden (3) · Switzerland (3) · Australia (2)
Joanne Kurtzberg, MD (3) · Adeline Vanderver, MD (2) · Cynthia J Tifft, M.D. (2) · Jeanine R. Jarnes, PharmD (2) · Paul Orchard, MD (2) · Terence Flotte, MD (2) · Anupam Sehgal, MBBS (1) · Bijan Almassian, Ph.D. (1) · Can Ficicioglu, MD, PhD (1) · Chester B. Whitley, PhD, MD (1) · Cynthia J TIfft, MD, PhD (1) · Florian Eichler, MD (1)
PMID 21586746, PMID 28476546, PMID 37624739, 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 35249485, PMID 36194207, PMID 36456200, PMID 36461157, PMID 36700853, PMID 37592153, PMID 38205442, PMID 38641994, PMID 40766118, PMID 41665410, PMID 41838934, PMID 42467089
2019 (3), U54NS065768 (3), R01HD073292 (1), U01NS106845 (1), U54NS115052 (1)