Status | Study |
Recruiting |
Study Name: Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1) Condition: Niemann-Pick Disease, Type C1 Date: 2016-09-21 Interventions: Drug: Hydroxypropyl-beta-cyclodextrin Used in the treatment of Niemann-Pick Disease C1 ( NPC1) |
Recruiting |
Study Name: Study of Pharmacokinetics and Preliminary Efficacy in Patients With Niemann-Pick C1 Condition: Niemann-Pick Disease, Type C1 Date: 2016-08-19 Interventions: Drug: Hydroxypropyl-beta-cyclodextrin Used in the treatment of Niemann-Pick C1 |
Terminated |
Study Name: Screening of Niemann-Pick Disease, Type C in a Psychiatric Population Condition: Psychiatric Adults Patients Date: 2016-07-11 Interventions: Biological: Blood sampling Biological: Bio |
Active, not recruiting |
Study Name: Arimoclomol Prospective Study in Patients Diagnosed With NiemannPick Disease Type C Condition: Niemann-Pick Disease, Type C Date: 2015-11-12 Interventions: Drug: arimoclomol Drug: Placebo |
Recruiting |
Study Name: Study of VTS-270 (2-hydroxypropyl-β-cyclodextrin) to Treat Niemann-Pick Type C1 (NPC1) Disease Condition: Niemann-Pick Disease, Type C Date: 2015-08-18 Interventions: Drug: VTS-270 Lumber intrathec |
Completed |
Study Name: A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C Condition: Niemann-Pick Disease, Type C Date: 2015-04-22 |
Recruiting |
Study Name: Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency Condition: Sphingomyelin Lipidosis Date: 2014-11-07 Interventions: Drug: Olipudase alfa Pharmaceutical form: powder for concentrate for solution for infusion Route of admi |
Completed |
Study Name: Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1 Condition: Neimann-Pick Disease Date: 2014-04-25 Interventions: Drug: Vorinostat Histone deactylase inhibitor |
Recruiting |
Study Name: Investigating Lysosomal Storage Diseases in Minority Groups Condition: Lysosomal Storage Disorders Gaucher Disease Fabry Disease Date: 2014-04-17 |
Recruiting |
Study Name: A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency Condition: Sphingomyelin Lipidosis Date: 2013-11-26 Interventions: Drug: GZ402665 Pharmaceutical form: Powder for concentrate |