The purpose of this study is to determine whether GSK2402968 given as a continuous dose and as an intermittent dose is effective and safe in the treatment of Duchenne muscular dystrophy.
This is a phase II, double-blind, exploratory, parallel-group, placebo-controlled clinical study in ambulant subjects with DMD resulting from a mutation that can be corrected by exon skipping induced by GSK2402968. The study aims to randomise 54 subjects. There will be 2 parallel cohorts. Each cohort will include subjects on GSK2402968 and matched placebo in a 2:1 ratio.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
53
Subcutaneous injection
Subcutaneous injection
GSK Investigational Site
Westmead, New South Wales, Australia
GSK Investigational Site
Parkville, Victoria, Australia
GSK Investigational Site
Ghent, Belgium
To assess the efficacy of 2 different dosing regimens of subcutaneous GSK2402968 administered over 24 weeks in ambulant subjects with DMD
Time frame: 48 weeks
To assess the safety and tolerability of 2 different dosing regimens of subcutaneous GSK2402968 administered over 48 weeks in ambulant subjects
Time frame: one year
To assess the PK of 2 different dosing regimens of subcutaneous GSK2402968 administered over 48 weeks in ambulant subjects with DMD
Time frame: 48 weeks
To assess long term efficacy of 2 different dosing regimens of subcutaneous GSK2402968 administered over 48 weeks in ambulant subjects with DMD
Time frame: one year
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GSK Investigational Site
Paris, France
GSK Investigational Site
Freiburg im Breisgau, Baden-Wurttemberg, Germany
GSK Investigational Site
Essen, North Rhine-Westphalia, Germany
GSK Investigational Site
Jerusalem, Israel
GSK Investigational Site
Nijmegen, Netherlands
GSK Investigational Site
Esplugues (Barcelona), Spain
GSK Investigational Site
Valencia, Spain
...and 3 more locations