6 terminated. 6 Phase 2, 2 Phase 1, 2 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 6 | Terminated 6 |
| Phase 1 | 2 | Terminated 2 |
| Phase 3 | 2 | Terminated 2 |
Follow Niemann-Pick Diseases: new trials by email Literature Review of Niemann-Pick Diseases trials Research Report on Niemann-Pick 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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| 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 |
Niemann-Pick Disease, Type C (4) · Leukodystrophy, Globoid Cell (2) · Leukodystrophy, Metachromatic (2) · Mucopolysaccharidosis I (2) · Wolman Disease (2) · alpha-Mannosidosis (2) · Adrenoleukodystrophy (1) · Aspartylglucosaminuria (1) · Chanarin-Dorfman Syndrome (1) · Cholesterol Ester Storage Disease (1) · Cirrhosis, Cryptogenic (1) · Cognitive Dysfunction (1) · Deglutition Disorders (1) · Disease (1)
Brain Diseases (6) · Brain Diseases, Metabolic (6) · Brain Diseases, Metabolic, Inborn (6) · Central Nervous System Diseases (6) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (6) · Genetic Diseases, Inborn (6) · Hemic and Lymphatic Diseases (6) · Histiocytosis (6) · Histiocytosis, Non-Langerhans-Cell (6) · Lipid Metabolism Disorders (6) · Lipid Metabolism, Inborn Errors (6) · Lipidoses (6) · Lymphatic Diseases (6) · Lysosomal Storage Diseases (6) · Lysosomal Storage Diseases, Nervous System (6)
2-Hydroxypropyl-beta-cyclodextrin (2) · Cyclodextrins (2) · Stem Cell Transplantation (2) · Alemtuzumab (1) · Bone Marrow Transplantation (1) · Busulfan (1) · Cyclophosphamide (1) · Sebelipase alfa (1)
Mandos LLC (2) · Alexion Pharmaceuticals, Inc. (1) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (1) · Masonic Cancer Center, University of Minnesota (1) · Talaris Therapeutics Inc. (1)
United States (5) · United Kingdom (2) · Australia (1) · Costa Rica (1) · Finland (1) · France (1) · Germany (1) · Italy (1) · New Zealand (1) · Singapore (1) · Switzerland (1) · Turkey (Türkiye) (1)
Forbes D Porter, M.D. (1) · Paul Orchard, MD (1) · Suzanne T Ildstad, MD (1)
2015 (1)