Actelion Pharmaceuticals Ltd

act-149071 - miglustat

 

Study ID Status Type Title
AC-056-102 Completed Protocol Summary A single-center, double-blind, randomized, placebo-controlled, two period / two treatment cross-over study to assess the effect of Florastor® (Saccharomyces boulardii lyo) on gastrointestinal tolerability, safety, and pharmacokinetics of Zavesca® (miglustat) in healthy subjects
AC-056-201 Terminated Protocol Summary Single center, double-blind, randomized, placebo-controlled, 2-period/2-treatment crossover study investigating the effect of miglustat on the nasal potential difference in patients with cystic fibrosis homozygous for the ΔF508 mutation
AC-056A202 Completed Protocol Summary Single center, double-blind, randomized, placebo-controlled, two-period/two-treatment crossover study investigating the effect of miglustat on the nasal potential difference in patients with cystic fibrosis homozygous for the F508del mutation
AC-056B404 Completed Protocol Summary An Observational study of the safety and effectiveness of Miglustat / Zavesca in patients with Type 1 Gaucher disease. A multicenter, non-interventional, open-label, prospective Phase 4 study.
OGT 918-011 Completed Protocol Summary Open-label, non comparative, multi-center study to evaluate the long term efficacy, safety and tolerability of oral miglustat as a maintenance therapy after a switch from enzyme replacement therapy in adult patients with stable type 1 Gaucher disease
OGT-918-007 Completed Protocol Summary A Phase II randomized controlled study of miglustat in adult and juvenile patients with Niemann-Pick Type C disease