1 not yet recruiting, 1 recruiting, 1 terminated. 1 Phase 1, 1 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 1 | Not yet recruiting 1 |
| Phase 4 | 1 | Terminated 1 |
Follow GM1 Gangliosidoses: new trials by email Literature Review of GM1 Gangliosidoses trials Research Report on GM1 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 |
| 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 |
| Longitudinal Study of Neurodegenerative Disorders Palliative Care, Hematopoetic Stem Cell Transplantation - MLD, Krabbe Disease | Recruiting | University of Pittsburgh | 1500 | 2012-01-11 | 2026-02-09 |
Gangliosidosis, GM1 (2) · Sandhoff Disease (2) · Tay-Sachs Disease (2) · Gangliosidoses (1) · Gangliosidoses, GM2 (1) · Gaucher Disease (1) · Hypermethioninemia (1) · Language (1) · Leukodystrophy, Globoid Cell (1) · Leukoencephalopathies (1) · Lysosomal Storage Diseases (1) · Mucopolysaccharidosis III (1) · Mucopolysaccharidosis IV (1) · Multiple Sulfatase Deficiency Disease (1) · Neuronal Ceroid-Lipofuscinoses (1)
Brain Diseases (3) · Brain Diseases, Metabolic (3) · Brain Diseases, Metabolic, Inborn (3) · Central Nervous System Diseases (3) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · Genetic Diseases, Inborn (3) · Lipid Metabolism Disorders (3) · Lipid Metabolism, Inborn Errors (3) · Lipidoses (3) · Lysosomal Storage Diseases, Nervous System (3) · Metabolic Diseases (3) · Metabolism, Inborn Errors (3) · Nervous System Diseases (3) · Nutritional and Metabolic Diseases (3) · Sphingolipidoses (3)
Diet, Ketogenic (1) · Genetic Engineering (1) · Palliative Care (1) · miglustat (1)
Tippi Mackenzie (1) · University of Minnesota (1) · University of Pittsburgh (1)
United States (2)
U54NS065768 (1)