| A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (NCT05163288) | IntraBio Inc | 2022-06-30 |
| ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program (NCT05368038) | Albert Einstein College of Medicine | 2021-05-10 |
| Complement Activation in the Lysosomal Storage Disorders (NCT04189601) | Melbourne Health | 2020-09-30 |
| Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) Disease (NCT03687476) | Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company | 2020-05-01 |
| Safety and Efficacy of Miglustat in Chinese NPC Patients (NCT03910621) | Actelion | 2020-04-02 |
| Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1 (NCT03879655) | Mandos LLC | 2019-12-02 |
| Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C (NCT03471143) | Washington University School of Medicine | 2019-02-22 |
| Biomarker for Niemann Pick Type C Disease (BioNPC) (NCT01306604) | CENTOGENE GmbH Rostock | 2018-08-20 |
| Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease (NCT04958642) | Mandos LLC | 2015-12-23 |
| VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease (NCT02534844) | Mandos LLC | 2015-10-01 |
| A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C (NCT02435030) | ZevraDenmark | 2015-09-01 |
| Longitudinal Study of Cognition With Niemann-Pick Disease, Type C (NCT01899950) | Mayo Clinic | 2011-03-01 |
| Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine (NCT00975689) | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 2009-08-01 |
| Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism (NCT00668564) | Masonic Cancer Center, University of Minnesota | 2008-03-01 |
| Application of Miglustat in Patients With Niemann-Pick Type C (NCT01760564) | National Taiwan University Hospital | 2008-01-01 |
| Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C (NCT00344331) | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 2006-08-14 |
| Early Access Program With Arimoclomol in US Patients With NPC (NCT04316637) | ZevraDenmark | |