Enzyme replacement therapy for mucopolysaccharidosis VI: A phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study

2006 ◽  
Vol 148 (4) ◽  
pp. 533-539.e6 ◽  
Author(s):  
Paul Harmatz ◽  
Roberto Giugliani ◽  
Ida Schwartz ◽  
Nathalie Guffon ◽  
Elisa Leão Teles ◽  
...  
Keyword(s):  
Multinational Study ◽  
Open Label ◽  
Phase 3 ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Enzyme Replacement ◽  
Arylsulfatase B ◽  
Open Label Extension ◽  
Mucopolysaccharidosis Vi ◽  
Human Arylsulfatase

Safety and efficacy of incobotulinumtoxinA for the treatment of upper facial lines: A randomized, double-blind, placebo-controlled, phase 3 study with open-label extension

Toxicon ◽  
2018 ◽  
Vol 156 ◽  
pp. S110
Author(s):  
Patrick Trevidic ◽  
Simon A. Connolly ◽  
Bernard Biwer ◽  
Petra Weissenberger ◽  
Philippe Kestemont ◽  
...  
Keyword(s):  
Open Label ◽  
Phase 3 ◽  
Double Blind ◽  
Safety And Efficacy ◽  
Double Blind Placebo ◽  
Open Label Extension

scholarly journals Eculizumab monotherapy for NMOSD: Data from PREVENT and its open-label extension

2021 ◽  
pp. 135245852110382
Author(s):  
Sean J Pittock ◽  
Kazuo Fujihara ◽  
Jacqueline Palace ◽  
Achim Berthele ◽  
Ho Jin Kim ◽  
...  
Keyword(s):  
Relapse Prevention ◽  
Interim Analysis ◽  
Time To Event ◽  
Open Label ◽  
Phase 3 ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Kaplan Meier ◽  
Open Label Extension

During PREVENT (a phase 3, randomized, double-blind, placebo-controlled, time-to-event study) and its open-label extension (interim analysis), 33 adults with aquaporin-4 immunoglobulin G-positive neuromyelitis optica spectrum disorder (AQP4-IgG + NMOSD) received eculizumab monotherapy for a median of 2.8 years (range, 14 weeks–5.2 years). At 192 weeks (~4 years), 96% of these patients were free from adjudicated relapses (Kaplan–Meier analysis; 95% confidence interval, 75.7–99.4). During PREVENT, 95% (20/21) of patients receiving eculizumab monotherapy had no disability worsening. Eculizumab monotherapy provides effective long-term relapse prevention, relieving the chronic immunosuppression burden in patients with AQP4-IgG + NMOSD. ClinicalTrials.gov; PREVENT: NCT01892345; open-label extension: NCT02003144.

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