Niemann-Pick Diseases - 25 Studies Found
Completed |
: Longitudinal Study of Cognition With Niemann-Pick Disease, Type C : Niemann-Pick Disease, Type C : 2013-07-04 |
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 |
Active, not recruiting |
: Arimoclomol Prospective Study in Patients Diagnosed With NiemannPick Disease Type C : Niemann-Pick Disease, Type C : 2015-11-12 :
|
Completed |
: A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C : Niemann-Pick Disease, Type C : 2015-04-22 |
Terminated |
: Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) :
|
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 |
: Biomarker for Niemann Pick Type C Disease :
|
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 |
Recruiting |
: Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C : Neimann-Pick Disease, Type C : 2006-06-23 |