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Niemann-Pick Disease, Type A - 25 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 |
Completed |
: Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1 : Neimann-Pick Disease : 2014-04-25 : Drug: Vorinostat Histone deactylase inhibitor |
Recruiting |
: A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency : Sphingomyelin Lipidosis : 2013-11-26 : Drug: GZ402665 Pharmaceutical form: Powder for concentrate |
Completed |
: Longitudinal Study of Cognition With Niemann-Pick Disease, Type C : Niemann-Pick Disease, Type C : 2013-07-04 |
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 |
: Biomarker for Niemann Pick Type C Disease :
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Completed |
: Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine : Niemann-Pick Disease, Type C : 2009-09-10 : Drug: N-Acetyl Cysteine 900mg effervescent tablet; Dosed as 15 mg/kg/day (maximum dose 900 mg per day) f |