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Niemann-Pick Diseases - 25 Studies Found
Estado | Estudiar |
Completed |
Nombre del estudio: Longitudinal Study of Cognition With Niemann-Pick Disease, Type C Condición: Niemann-Pick Disease, Type C Fecha: 2013-07-04 |
Completed |
Nombre del estudio: Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine Condición: Niemann-Pick Disease, Type C Fecha: 2009-09-10 Intervenciones: Drug: N-Acetyl Cysteine 900mg effervescent tablet; Dosed as 15 mg/kg/day (maximum dose 900 mg per day) f |
Active, not recruiting |
Nombre del estudio: Arimoclomol Prospective Study in Patients Diagnosed With NiemannPick Disease Type C Condición: Niemann-Pick Disease, Type C Fecha: 2015-11-12 Intervenciones:
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Completed |
Nombre del estudio: A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C Condición: Niemann-Pick Disease, Type C Fecha: 2015-04-22 |
Terminated |
Nombre del estudio: Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Condición:
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Completed |
Nombre del estudio: Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1 Condición: Neimann-Pick Disease Fecha: 2014-04-25 Intervenciones: Drug: Vorinostat Histone deactylase inhibitor |
Recruiting |
Nombre del estudio: Biomarker for Niemann Pick Type C Disease Condición:
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Completed |
Nombre del estudio: Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients Condición: Human Acid Sphingomyelinase Deficiency Fecha: 2012-11-05 Intervenciones: Drug: Recombinant human acid sphingomyelinase Administered intravenously every 2 weeks for 26 weeks |
Completed |
Nombre del estudio: Miglustat in Niemann-Pick Type C Disease Condición: Niemann-Pick Type C Disease Fecha: 2007-08-15 Intervenciones: Drug: miglustat Patients aged 12 years or more randomised to the treatment group will receive 200 mg OGT |
Recruiting |
Nombre del estudio: Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C Condición: Neimann-Pick Disease, Type C Fecha: 2006-06-23 |