19 terminated. 19 Phase 2, 8 Phase 1, 2 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 19 | Terminated 19 |
| Phase 1 | 8 | Terminated 8 |
| Phase 3 | 2 | Terminated 2 |
Follow Lysosomal Storage Diseases, Nervous System: new trials by email Literature Review of Lysosomal Storage Diseases, Nervous System trials Research Report on Lysosomal Storage Diseases, Nervous System
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 |
|---|---|---|---|---|---|---|
| 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 |
| A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190 FLT190 - Fabry Disease, Lysosomal Storage Diseases | Phase 1 / Phase 2 | Terminated | Spur Therapeutics | 3 | 2020-09-08 | 2026-09-03 |
| Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1 VTS-270 - Niemann-Pick Disease, Type C1 | Phase 1 / Phase 2 | Terminated | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 2 | 2020-01-06 | 2022-08-30 |
| Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1 VTS-270 - Niemann-Pick Disease, Type C | Phase 2 / Phase 3 | Terminated | Mandos LLC | 2 | 2019-12-02 | 2023-12-26 |
| A Fabry Disease Gene Therapy Study FLT190 - Fabry Disease, Lysosomal Storage Diseases | Phase 1 / Phase 2 | Terminated | Spur Therapeutics | 3 | 2019-07-08 | 2026-09-03 |
| Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease AVR-RD-02 - Gaucher Disease | Phase 1 / Phase 2 | Terminated | AVROBIO | 8 | 2019-05-30 | 2024-01-18 |
| Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3 Arimoclomol, Placebo oral capsule - Gaucher Disease, Type 1, Gaucher Disease, Type 3 | Phase 2 | Terminated | ZevraDenmark | 39 | 2018-06-06 | 2023-01-11 |
| Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease AVR-RD-01 - Fabry Disease | Phase 1 / Phase 2 | Terminated | AVROBIO | 15 | 2018-02-21 | 2024-01-05 |
| VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease VAL-1221, RhGAA - Pompe Disease | Phase 1 / Phase 2 | Terminated | Valerion Therapeutics, LLC | 12 | 2017-06-21 | 2020-06-02 |
| Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease Adrabetadex - Niemann-Pick Disease, Type C | Phase 2 / Phase 3 | Terminated | Mandos LLC | 66 | 2015-12-23 | 2023-08-29 |
| Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency Sebelipase Alfa - Lysosomal Acid Lipase Deficiency | Phase 2 | Terminated | Alexion Pharmaceuticals, Inc. | 10 | 2014-06-06 | 2019-11-18 |
| Extension Study for Patients Who Have Participated in a BMN 701 Study BMN 701 - Pompe Disease | Phase 2 | Terminated | BioMarin Pharmaceutical | 21 | 2011-08-15 | 2018-05-22 |
| Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders hematopoietic stem cell infusion - Hurler Syndrome (MPS I), Hurler-Scheie Syndrome | Phase 1 / Phase 2 | Terminated | Talaris Therapeutics Inc. | 3 | 2011-04-01 | 2023-04-12 |
| Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease Duvoglustat - Pompe Disease | Phase 2 | Terminated | Amicus Therapeutics | 3 | 2008-12-08 | 2025-09-11 |
| Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism Stem Cell Transplantation, Cyclophosphamide, Campath-1H - Hurler's Syndrome, Maroteaux-Lamy Syndrome | Phase 2 | Terminated | Masonic Cancer Center, University of Minnesota | 18 | 2008-03-01 | 2017-12-28 |
| Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD HGT-1111 - Late Infantile Metachromatic Leukodystrophy | Phase 2 | Terminated | Shire | 11 | 2008-02-20 | 2021-06-08 |
| Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study migalastat HCl - Fabry Disease | Phase 2 | Terminated | Amicus Therapeutics | 23 | 2007-09-17 | 2018-10-03 |
| Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme Fabrazyme (agalsidase beta) - Fabry Disease, Chronic Kidney Disease, Stage IV (Severe) | Phase 2 | Terminated | Genzyme, a Sanofi Company | 20 | 2002-12-01 | 2015-04-07 |
| Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy glyceryl trierucate/glyceryl trioleate - Adrenoleukodystrophy | Phase 2 | Terminated | Hugo W. Moser Research Institute at Kennedy Krieger, Inc. | 126 | 1998-04-01 | 2020-10-23 |
Fabry Disease (5) · Lysosomal Storage Diseases (4) · Niemann-Pick Disease, Type C (4) · Glycogen Storage Disease Type II (3) · Leukodystrophy, Metachromatic (3) · Adrenoleukodystrophy (2) · Gaucher Disease (2) · Leukodystrophy, Globoid Cell (2) · Metabolism, Inborn Errors (2) · Mucopolysaccharidosis I (2) · Niemann-Pick Diseases (2) · Sphingolipidoses (2) · Wolman Disease (2) · alpha-Mannosidosis (2) · Aspartylglucosaminuria (1)
Brain Diseases (19) · Brain Diseases, Metabolic (19) · Brain Diseases, Metabolic, Inborn (19) · Central Nervous System Diseases (19) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (19) · Genetic Diseases, Inborn (19) · Metabolic Diseases (19) · Nervous System Diseases (19) · Nutritional and Metabolic Diseases (19) · Metabolism, Inborn Errors (17) · Lipid Metabolism Disorders (16) · Lipid Metabolism, Inborn Errors (16) · Lipidoses (16) · Lysosomal Storage Diseases (15)
2-Hydroxypropyl-beta-cyclodextrin (2) · Cyclodextrins (2) · Stem Cell Transplantation (2) · 1-Deoxynojirimycin (1) · Alemtuzumab (1) · Bone Marrow Transplantation (1) · Busulfan (1) · Cyclophosphamide (1) · GAA protein, human (1) · Lorenzo's oil (1) · Sebelipase alfa (1) · Triolein (1) · agalsidase beta (1) · arimoclomol (1) · larazotide acetate (1)
AVROBIO (2) · Amicus Therapeutics (2) · Mandos LLC (2) · Spur Therapeutics (2) · Alexion Pharmaceuticals, Inc. (1) · BioMarin Pharmaceutical (1) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (1) · Genzyme, a Sanofi Company (1) · Hugo W. Moser Research Institute at Kennedy Krieger, Inc. (1) · LYSOGENE (1) · Masonic Cancer Center, University of Minnesota (1) · Shire (1) · Talaris Therapeutics Inc. (1) · Valerion Therapeutics, LLC (1) · ZevraDenmark (1)
United States (15) · United Kingdom (8) · Australia (4) · France (4) · Germany (4) · Brazil (2) · Canada (2) · Italy (2) · New Zealand (2) · Austria (1) · Costa Rica (1) · Denmark (1) · Finland (1) · India (1) · Norway (1)
Aabha Nagral, MD (1) · Forbes D Porter, M.D. (1) · Gerald V Raymond, M.D. (1) · Hal Landy, MD (1) · Inderpal Panesar, MRPharmS (1) · Milena Veselinovic, MD (1) · Paul Orchard, MD (1) · Suzanne T Ildstad, MD (1)
PMID 21586746, PMID 33332761, PMID 36631545, PMID 37624739
2008 (1), 2015 (1)