6 completed, 2 recruiting, 2 unknown, 1 active not recruiting. 5 Phase 2, 3 Phase 3, 2 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 5 | Completed 2, Active not recruiting 1, Not yet recruiting 1, Terminated 1 |
| Phase 3 | 3 | Completed 1, Not yet recruiting 1, Recruiting 1 |
| Phase 1 | 2 | Active not recruiting 1, Terminated 1 |
Follow GM1 Gangliosidosis: new trials by email Literature Review of GM1 Gangliosidosis trials Research Report on GM1 Gangliosidosis
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 |
| 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 | 2026-10-27 |
| FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases Alpha-Thalassemia, Beta-Thalassemia | Unknown | xCures | 1000 | 2024-06-10 | 2024-11-14 | |
| 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 |
| 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 | |
| Interviews and Video Capture in Patients With GM1 Gangliosidosis Natural history - GM1 Gangliosidosis | Completed | LYSOGENE | 25 | 2020-04-20 | 2023-06-08 | |
| 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 | |
| A Natural History Study of the Gangliosidoses Tay-Sachs Disease, Sandhoff Disease | Recruiting | University of Minnesota | 52 | 2010-12-01 | 2026-03-04 | |
| HSCT for High Risk Inherited Inborn Errors Clofarabine, Total body Irradiation, Melphalan - Adrenoleukodystrophy, Metachromatic Leukodystrophy | Phase 2 | Completed | Masonic Cancer Center, University of Minnesota | 38 | 2006-09-01 | 2019-07-11 |
| Stem Cell Transplant for Inborn Errors of Metabolism Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin - Adrenoleukodystrophy, Metachromatic Leukodystrophy | Phase 2 / Phase 3 | Completed | Masonic Cancer Center, University of Minnesota | 135 | 1995-01-01 | 2017-12-28 |
Gangliosidosis, GM1 (14) · Gangliosidoses, GM2 (6) · Sandhoff Disease (6) · Tay-Sachs Disease (6) · Gaucher Disease (5) · Lysosomal Storage Diseases (5) · Leukodystrophy, Metachromatic (3) · Niemann-Pick Disease, Type C (3) · Rare Diseases (3) · Wolman Disease (3) · Adrenoleukodystrophy (2) · Gangliosidoses (2) · Leukodystrophy, Globoid Cell (2) · Mucolipidoses (2) · Mucopolysaccharidosis III (2)
Brain Diseases (14) · Brain Diseases, Metabolic (14) · Brain Diseases, Metabolic, Inborn (14) · Central Nervous System Diseases (14) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (14) · Lipid Metabolism Disorders (14) · Lipid Metabolism, Inborn Errors (14) · Lipidoses (14) · Lysosomal Storage Diseases, Nervous System (14) · Metabolic Diseases (14) · Nutritional and Metabolic Diseases (14) · Sphingolipidoses (14) · Genetic Diseases, Inborn (13) · Metabolism, Inborn Errors (13) · Nervous System Diseases (13)
Alemtuzumab (1) · Antilymphocyte Serum (1) · Bone Marrow Transplantation (1) · Busulfan (1) · Clofarabine (1) · Cyclophosphamide (1) · Hematopoietic Stem Cell Transplantation (1) · Hydroxyurea (1) · Melphalan (1) · Stem Cell Transplantation (1) · Whole-Body Irradiation (1)
Azafaros B.V. (3) · LYSOGENE (2) · Masonic Cancer Center, University of Minnesota (2) · Albert Einstein College of Medicine (1) · Gemma Biotherapeutics (1) · Idorsia Pharmaceuticals Ltd. (1) · SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt) (1) · University of Minnesota (1) · University of Pennsylvania (1) · xCures (1)
United States (12) · United Kingdom (6) · Brazil (5) · France (4) · Germany (4) · Italy (3) · Turkey (Türkiye) (3) · Canada (2) · Portugal (2) · Spain (2) · Switzerland (2) · Argentina (1) · Australia (1) · Belgium (1) · India (1)
Paul Orchard, MD (2) · Can Ficicioglu, MD, PhD (1) · Jeanine R. Jarnes, PharmD (1) · May Orfali, MD (1) · Melissa Wasserstein, MD (1) · Mindy Leffler, MEd (1)
PMID 21586746, PMID 25557439, PMID 28476546, PMID 29352662, PMID 36194207, PMID 37592153, PMID 37624739
2019 (2), R01HD073292 (1), U54NS065768 (1)