33 terminated. 33 Phase 2, 15 Phase 1, 3 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 33 | Terminated 33 |
| Phase 1 | 15 | Terminated 15 |
| Phase 3 | 3 | Terminated 3 |
Follow Lysosomal Storage Diseases: new trials by email Literature Review of Lysosomal Storage Diseases trials Research Report on Lysosomal Storage Diseases
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 |
|---|---|---|---|---|---|---|
| Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia Recifercept - Achondroplasia | Phase 2 | Terminated | Pfizer | 35 | 2021-12-24 | 2024-02-08 |
| 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 Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia Recifercept - Achondroplasia | Phase 2 | Terminated | Pfizer | 60 | 2020-12-02 | 2024-02-15 |
| 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 |
| Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease ABO-102 - MPS IIIA, Sanfilippo Syndrome | Phase 1 / Phase 2 | Terminated | Ultragenyx Pharmaceutical Inc | 5 | 2019-09-18 | 2023-07-25 |
| A Phase 2 Study of ELX-02 in Patients With Nephropathic Cystinosis ELX-02 - Genetic Disease, Nonsense Mutation | Phase 2 | Terminated | Eloxx Pharmaceuticals, Inc. | 3 | 2019-08-02 | 2020-05-27 |
| 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 |
| Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB rAAV9.CMV.hNAGLU - Mucopolysaccharidosis Type 3 B | Phase 1 / Phase 2 | Terminated | Abeona Therapeutics, Inc | 11 | 2017-10-16 | 2022-05-05 |
| 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 |
| Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I SB-318 - MPS I | Phase 1 / Phase 2 | Terminated | Sangamo Therapeutics | 3 | 2017-05-24 | 2023-01-26 |
| Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II SB-913 - Mucopolysaccharidosis II, MPS II | Phase 1 / Phase 2 | Terminated | Sangamo Therapeutics | 9 | 2017-05-11 | 2022-10-25 |
| 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 |
| A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously SBC-103 - Mucopolysaccharidosis III, Type B (MPS IIIB), Sanfilippo B | Phase 1 / Phase 2 | Terminated | Alexion Pharmaceuticals, Inc. | 3 | 2015-10-15 | 2018-04-17 |
| An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093. HGT-1410 - Sanfilippo Syndrome, Mucopolysaccharidosis (MPS) | Phase 2 | Terminated | Shire | 17 | 2015-04-08 | 2021-06-11 |
| 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 |
Showing 20 of 33. Sign in to see all of them.
Mucopolysaccharidosis III (6) · Fabry Disease (5) · Mucopolysaccharidosis IV (5) · Mucopolysaccharidosis I (4) · Niemann-Pick Disease, Type C (4) · Glycogen Storage Disease Type II (3) · Leukodystrophy, Metachromatic (3) · Mucopolysaccharidosis II (3) · Sudden Infant Death (3) · Achondroplasia (2) · Adrenoleukodystrophy (2) · Dwarfism (2) · Gaucher Disease (2) · Genetic Diseases, Inborn (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (33) · Metabolic Diseases (33) · Nutritional and Metabolic Diseases (33) · Genetic Diseases, Inborn (31) · Metabolism, Inborn Errors (31) · Nervous System Diseases (21) · Brain Diseases (19) · Brain Diseases, Metabolic (19) · Brain Diseases, Metabolic, Inborn (19) · Central Nervous System Diseases (19) · Lysosomal Storage Diseases, Nervous System (19) · Carbohydrate Metabolism, Inborn Errors (18) · Lipid Metabolism Disorders (16) · Lipid Metabolism, Inborn Errors (16)
GALNS protein, human (3) · 2-Hydroxypropyl-beta-cyclodextrin (2) · Chondroitinases and Chondroitin Lyases (2) · Cyclodextrins (2) · Enzyme Replacement Therapy (2) · Galns protein, mouse (2) · Stem Cell Transplantation (2) · Sulfatases (2) · 1-Deoxynojirimycin (1) · Alemtuzumab (1) · Bone Marrow Transplantation (1) · Busulfan (1) · Cyclophosphamide (1) · ELX-02 (1) · GAA protein, human (1)
BioMarin Pharmaceutical (4) · Shire (3) · AVROBIO (2) · Alexion Pharmaceuticals, Inc. (2) · Amicus Therapeutics (2) · Mandos LLC (2) · Pfizer (2) · Sangamo Therapeutics (2) · Spur Therapeutics (2) · Abeona Therapeutics, Inc (1) · Eloxx Pharmaceuticals, Inc. (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)
United States (25) · United Kingdom (14) · Germany (8) · Australia (7) · France (6) · Italy (5) · Spain (5) · Canada (4) · Denmark (3) · Belgium (2) · Brazil (2) · Netherlands (2) · New Zealand (2) · Portugal (2) · Austria (1)
Aabha Nagral, MD (1) · Adam Shaywitz, MD PhD (1) · Celeste Decker, M.D. (1) · Celeste Decker, MD (1) · Forbes D Porter, M.D. (1) · Gerald V Raymond, M.D. (1) · Hal Landy, MD (1) · Inderpal Panesar, MRPharmS (1) · Lynda E Polgreen, M.D. (1)
PMID 36299240, PMID 21586746, PMID 22547151, PMID 25421091, PMID 29159458, PMID 29526614, PMID 32278382, PMID 32884151, PMID 33332761, PMID 34600820, PMID 36631545, PMID 37624739, PMID 38685585
2015 (2), 2008 (1), 2014 (1), 2018 (1)