Niemann-Pick Disease, Type C - 25 Studies Found
Recruiting |
: Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency : Sphingomyelin Lipidosis : 2013-11-26 :
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Recruiting |
: Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency : Sphingomyelin Lipidosis : 2014-11-07 : Drug: Olipudase alfa Pharmaceutical form: powder for concentrate for solution for infusion Route of admi |
Terminated |
: Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) :
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Recruiting |
: Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1) : Niemann-Pick Disease, Type C1 : 2016-09-21 : Drug: Hydroxypropyl-beta-cyclodextrin Used in the treatment of Niemann-Pick Disease C1 ( NPC1) |
Recruiting |
: Study of Pharmacokinetics and Preliminary Efficacy in Patients With Niemann-Pick C1 : Niemann-Pick Disease, Type C1 : 2016-08-19 : Drug: Hydroxypropyl-beta-cyclodextrin Used in the treatment of Niemann-Pick C1 |
Completed |
: Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease : Niemann-Pick Disease, Type C1 : 2012-12-08 : Drug: VTS-270 Other Name: 2-hydroxypropyl-beta-cyclodextrin |
Recruiting |
: Study of VTS-270 (2-hydroxypropyl-β-cyclodextrin) to Treat Niemann-Pick Type C1 (NPC1) Disease : Niemann-Pick Disease, Type C : 2015-08-18 :
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Completed |
: Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease : Niemann Pick Diseases : 2006-04-19 : Drug: OGT918 |
Terminated |
: Screening of Niemann-Pick Disease, Type C in a Psychiatric Population : Psychiatric Adults Patients : 2016-07-11 :
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Completed |
: Application of Miglustat in Patients With Niemann-Pick Type C : Niemann-Pick Disease Type C : 2013-01-02 : Drug: Miglustat miglustat 200mg tid for adult. For children surface area correction will be made. A lowe |