Queen Square Centre for Neuromuscular Diseases


A phase II, double-blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophy

Sponsor AVI Biopharma
Funder Medical Research Council (MRC) and AVI Biopharma
CI Professor Kate Bushby / Professor Francesco Muntoni
sites Newcastle, London GOSH
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Trial information

This was a Phase IIa, multicentre, double blind, exploratory, parallel clinical trial of GSK2402968 in ambulant DMD boys.

Subcutaneous injections of the drug were given as a continuous OR intermittent regimen over 24 and 48 weeks.

Primary objective

To assess the efficacy of 2 different dosing regimens of GSK2402968,

Secondary objectives

  • To assess the safety and tolerability of 2 different doses of the drug
  • To assess the pharmacokinetics of the 2 different drug doses over 48 weeks
  • To assess long term efficacy of the 2 different drug doses


  • 54 subjects
  • 2 parallel groups
  • Each group included 16 patients on the drug and 8 on matched placebo (2:1 ratio)

This trial recruited in UK at the Great Ormond Street Hospital (GOSH), London and at the Royal Victoria Infirmary, Newcastle.