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Niemann-Pick Diseases - 25 Studies Found
Estado | Estudiar |
Recruiting |
Nombre del estudio: A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency Condición: Sphingomyelin Lipidosis Fecha: 2013-11-26 Intervenciones: Drug: GZ402665 Pharmaceutical form: Powder for concentrate |
Completed |
Nombre del estudio: Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease Condición: Niemann Pick Diseases Fecha: 2006-04-19 Intervenciones: Drug: OGT918 |
Recruiting |
Nombre del estudio: Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency Condición: Sphingomyelin Lipidosis Fecha: 2013-11-26 Intervenciones:
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Recruiting |
Nombre del estudio: Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency Condición: Sphingomyelin Lipidosis Fecha: 2014-11-07 Intervenciones: Drug: Olipudase alfa Pharmaceutical form: powder for concentrate for solution for infusion Route of admi |
Recruiting |
Nombre del estudio: Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1) Condición: Niemann-Pick Disease, Type C1 Fecha: 2016-09-21 Intervenciones: Drug: Hydroxypropyl-beta-cyclodextrin Used in the treatment of Niemann-Pick Disease C1 ( NPC1) |
Recruiting |
Nombre del estudio: Study of Pharmacokinetics and Preliminary Efficacy in Patients With Niemann-Pick C1 Condición: Niemann-Pick Disease, Type C1 Fecha: 2016-08-19 Intervenciones: Drug: Hydroxypropyl-beta-cyclodextrin Used in the treatment of Niemann-Pick C1 |
Completed |
Nombre del estudio: Application of Miglustat in Patients With Niemann-Pick Type C Condición: Niemann-Pick Disease Type C Fecha: 2013-01-02 Intervenciones: Drug: Miglustat miglustat 200mg tid for adult. For children surface area correction will be made. A lowe |
Completed |
Nombre del estudio: Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease Condición: Fecha: 2024-05-18 |
Terminated |
Nombre del estudio: Screening of Niemann-Pick Disease, Type C in a Psychiatric Population Condición: Psychiatric Adults Patients Fecha: 2016-07-11 Intervenciones:
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Recruiting |
Nombre del estudio: Study of VTS-270 (2-hydroxypropyl-β-cyclodextrin) to Treat Niemann-Pick Type C1 (NPC1) Disease Condición: Niemann-Pick Disease, Type C Fecha: 2015-08-18 Intervenciones:
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