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Niemann-Pick Disease, Type C - 25 Studies Found

Status Study
Completed Study Name: Longitudinal Study of Cognition With Niemann-Pick Disease, Type C
Condition: Niemann-Pick Disease, Type C
Date: 2013-07-04
Completed Study Name: Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine
Condition: Niemann-Pick Disease, Type C
Date: 2009-09-10
Interventions: Drug: N-Acetyl Cysteine 900mg effervescent tablet; Dosed as 15 mg/kg/day (maximum dose 900 mg per day) f
Completed Study Name: Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1
Condition: Neimann-Pick Disease
Date: 2014-04-25
Interventions: Drug: Vorinostat Histone deactylase inhibitor
Active, not recruiting Study Name: Arimoclomol Prospective Study in Patients Diagnosed With NiemannPick Disease Type C
Condition: Niemann-Pick Disease, Type C
Date: 2015-11-12
Interventions:
  • Drug: arimoclomol
  • Drug: Placebo
Completed Study Name: A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C
Condition: Niemann-Pick Disease, Type C
Date: 2015-04-22
Recruiting Study Name: Biomarker for Niemann Pick Type C Disease
Condition:
  • Niemann-Pick Disease
  • Niemann-Pick Disease
    Date: 2011-03-01
Completed Study Name: Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients
Condition: Human Acid Sphingomyelinase Deficiency
Date: 2012-11-05
Interventions: Drug: Recombinant human acid sphingomyelinase Administered intravenously every 2 weeks for 26 weeks
Completed Study Name: Miglustat in Niemann-Pick Type C Disease
Condition: Niemann-Pick Type C Disease
Date: 2007-08-15
Interventions: Drug: miglustat Patients aged 12 years or more randomised to the treatment group will receive 200 mg OGT
Recruiting Study Name: Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
Condition: Neimann-Pick Disease, Type C
Date: 2006-06-23
Recruiting Study Name: A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
Condition: Sphingomyelin Lipidosis
Date: 2013-11-26
Interventions: Drug: GZ402665 Pharmaceutical form: Powder for concentrate
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