10 completed, 3 terminated, 1 recruiting, 1 unknown. 15 Phase 1, 8 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 15 | Completed 10, Terminated 3, Recruiting 1, Unknown 1 |
| Phase 2 | 8 | Completed 6, 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 Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C Hydroxypropyl-β-cyclodextrin - Niemann-Pick Disease, Type C1 | Phase 1 | Unknown | Cyclo Therapeutics, Inc. | 12 | 2019-05-23 | 2021-06-18 |
| Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C VTS-270 - Niemann-Pick Disease, Type C | Phase 1 / Phase 2 | Completed | Washington University School of Medicine | 4 | 2019-02-22 | 2026-01-05 |
| Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1) Hydroxypropyl-beta-cyclodextrin - Niemann-Pick Disease, Type C1 | Phase 1 | Completed | Cyclo Therapeutics, Inc. | 13 | 2017-10-11 | 2021-02-21 |
| Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients Hydroxypropyl-beta-cyclodextrin - Niemann-Pick Disease, Type C1 | Phase 1 / Phase 2 | Completed | Cyclo Therapeutics, Inc. | 12 | 2017-03-20 | 2021-08-10 |
| Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency Olipudase alfa - Sphingomyelin Lipidosis | Phase 1 / Phase 2 | Completed | Genzyme, a Sanofi Company | 20 | 2015-05-01 | 2022-03-23 |
| UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells DUOC-01 - Adrenoleukodystrophy, Batten Disease | Phase 1 | Recruiting | Joanne Kurtzberg, MD | 40 | 2014-09-01 | 2026-09-03 |
| Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1 Vorinostat - Neimann-Pick Disease | Phase 1 / Phase 2 | Completed | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 12 | 2014-04-25 | 2018-02-22 |
| Human Placental-Derived Stem Cell Transplantation Human Placental Derived Stem Cell - Mucopolysaccharidosis I, Mucopolysaccharidosis VI | Phase 1 | Completed | New York Medical College | 43 | 2013-04-01 | 2022-10-25 |
| Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease VTS-270 - Niemann-Pick Disease, Type C1 | Phase 1 | Completed | Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company | 14 | 2013-01-01 | 2021-07-23 |
| 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 |
| Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine N-Acetyl Cysteine - Niemann-Pick Disease, Type C | Phase 1 / Phase 2 | Completed | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 35 | 2009-08-01 | 2013-05-06 |
| Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells Hematopoietic stem cell transplantation - Sickle Cell Disease, Thalassemia | Phase 1 / Phase 2 | Completed | Children's Hospital Los Angeles | 25 | 2008-08-01 | 2016-06-23 |
| Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) rhASM - Acid Sphingomyelinase Deficiency, Niemann-Pick Disease | Phase 1 | Terminated | Genzyme, a Sanofi Company | 11 | 2006-12-01 | 2015-03-19 |
| Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease OGT918 - Niemann Pick Diseases | Phase 1 | Completed | National Eye Institute (NEI) | 30 | 2002-10-17 | 2017-07-02 |
Niemann-Pick Disease, Type C (8) · Adrenoleukodystrophy (3) · Leukodystrophy, Globoid Cell (3) · Leukodystrophy, Metachromatic (3) · Mucopolysaccharidosis I (3) · Niemann-Pick Disease, Type A (3) · Fucosidosis (2) · Mucopolysaccharidosis II (2) · Nerve Degeneration (2) · Neuronal Ceroid-Lipofuscinoses (2) · Sandhoff Disease (2) · Tay-Sachs Disease (2) · alpha-Mannosidosis (2) · Anemia (1)
Brain Diseases (15) · Brain Diseases, Metabolic (15) · Central Nervous System Diseases (15) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (15) · Hemic and Lymphatic Diseases (15) · Histiocytosis (15) · Histiocytosis, Non-Langerhans-Cell (15) · Lipid Metabolism Disorders (15) · Lipid Metabolism, Inborn Errors (15) · Lipidoses (15) · Lymphatic Diseases (15) · Lysosomal Storage Diseases, Nervous System (15) · Metabolic Diseases (15) · Nervous System Diseases (15) · Nutritional and Metabolic Diseases (15)
2-Hydroxypropyl-beta-cyclodextrin (4) · Acetylcysteine (1) · Hematopoietic Stem Cell Transplantation (1) · Stem Cell Transplantation (1) · Vorinostat (1) · miglustat (1) · olipudase alfa (1)
Cyclo Therapeutics, Inc. (3) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (3) · Genzyme, a Sanofi Company (2) · Children's Hospital Los Angeles (1) · Joanne Kurtzberg, MD (1) · National Eye Institute (NEI) (1) · New York Medical College (1) · Talaris Therapeutics Inc. (1) · Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company (1) · Washington University School of Medicine (1)
United States (14) · United Kingdom (3) · Brazil (1) · France (1) · Germany (1) · Israel (1) · Italy (1) · Sweden (1)
Forbes D Porter, M.D. (3) · Caroline Hastings, MD (2) · Hisham Abdel-Azim, MD (1) · Joanne Kurtzberg, MD (1) · Mitchell S Cairo, MD (1) · Patricia I Dickson, MD (1) · Reena Sharma, MB BS (1) · Suzanne T Ildstad, MD (1)
PMID 36279795, PMID 31187454, PMID 33875845, PMID 34389161, PMID 34810067, PMID 37670901, PMID 40525490
U01HD090845 (1)