Status | Study |
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 |
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) |
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: 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 |
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 |
Recruiting |
Study Name: Biomarker for Niemann Pick Type C Disease Condition: Niemann-Pick Disease Niemann-Pick Disease, Type C 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 |
Terminated |
Study Name: Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism Condition: Hurler's Syndrome Maroteaux-Lamy Syndrome Sly Syndrome Date: 2008-04-25 Interventions: Procedure: Stem Cell Transplantation |
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 |