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Niemann-Pick Disease Type A - 12 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
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
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: Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease
Condition: Niemann Pick Diseases
Date: 2006-04-19
Interventions: Drug: OGT918
Recruiting Study Name: Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)
Condition: Niemann-Pick Disease, Type C1
Date: 2016-09-21
Interventions: Drug: Hydroxypropyl-beta-cyclodextrin Used in the treatment of Niemann-Pick Disease C1 ( NPC1)
Recruiting Study Name: Study of Pharmacokinetics and Preliminary Efficacy in Patients With Niemann-Pick C1
Condition: Niemann-Pick Disease, Type C1
Date: 2016-08-19
Interventions: Drug: Hydroxypropyl-beta-cyclodextrin Used in the treatment of Niemann-Pick C1
Completed Study Name: Application of Miglustat in Patients With Niemann-Pick Type C
Condition: Niemann-Pick Disease Type C
Date: 2013-01-02
Interventions: Drug: Miglustat miglustat 200mg tid for adult. For children surface area correction will be made. A lowe
Completed Study Name: Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease
Condition: Niemann-Pick Disease, Type C1
Date: 2012-12-08
Interventions: Drug: VTS-270 Other Name: 2-hydroxypropyl-beta-cyclodextrin
Terminated Study Name: Screening of Niemann-Pick Disease, Type C in a Psychiatric Population
Condition: Psychiatric Adults Patients
Date: 2016-07-11
Interventions:
  • Biological: Blood sampling
  • Biological: Bio
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