Niemann-Pick Disease Type A - 12 Studies Found
| Completed |
: Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients : Human Acid Sphingomyelinase Deficiency : 2012-11-05 : Drug: Recombinant human acid sphingomyelinase Administered intravenously every 2 weeks for 26 weeks |
| Completed |
: Miglustat in Niemann-Pick Type C Disease : Niemann-Pick Type C Disease : 2007-08-15 : Drug: miglustat Patients aged 12 years or more randomised to the treatment group will receive 200 mg OGT |
| Terminated |
: Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) :
|
| Active, not recruiting |
: Arimoclomol Prospective Study in Patients Diagnosed With NiemannPick Disease Type C : Niemann-Pick Disease, Type C : 2015-11-12 :
|
| Completed |
: Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease : Niemann Pick Diseases : 2006-04-19 : Drug: OGT918 |
| 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 |
: 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 |
| 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 |
| Terminated |
: Screening of Niemann-Pick Disease, Type C in a Psychiatric Population : Psychiatric Adults Patients : 2016-07-11 :
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