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Niemann-Pick Disease, Type A - 25 Studies Found

Status Study
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
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
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
Completed Study Name: Longitudinal Study of Cognition With Niemann-Pick Disease, Type C
Condition: Niemann-Pick Disease, Type C
Date: 2013-07-04
Recruiting Study Name: Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
Condition: Sphingomyelin Lipidosis
Date: 2013-11-26
Interventions:
  • Drug: placebo (saline)

Recruiting Study Name: Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency
Condition: Sphingomyelin Lipidosis
Date: 2014-11-07
Interventions: Drug: Olipudase alfa Pharmaceutical form: powder for concentrate for solution for infusion Route of admi
Terminated Study Name: Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)
Condition:
  • Acid Sphingomyelinase Deficiency
  • Niemann-
    Date: 2006-12-11
    Interventions:
    • Drug: rhASM Single dose of 0.0
Recruiting Study Name: Biomarker for Niemann Pick Type C Disease
Condition:
  • Niemann-Pick Disease
  • Niemann-Pick Disease
    Date: 2011-03-01
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
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