Queen Square Centre for Neuromuscular Diseases


An open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy (DMD)

Sponsor GlaxoSmithKline
Funder GlaxoSmithKline
CI Professor Volker Straub
sites Newcastle and London GOSH
More information

Trial information

A Phase III, multicentre, open-label extension, study in male outpatients with DMD. Patient will have to have participated in one of the previous GSK studies of the same drug.

All patients will receive 6mg/kg of the drug weekly or an intermittent dosing frequency. Patients will receive the drug for at least two years.

Primary objective

To assess the long term safety, tolerability and efficacy of subcutaneous 6mg/kg/week of the drug in DMD patients.

Secondary objectives

To assess:

  • the long-term pharmacokinetics (PK) of the drug
  • the long-term impact on health-related quality of life (HRQoL) and functional outcomes of continued treatment with GSK2402968
  • DMD disease progression and outcomes (clinical, HRQoL and functional) in subjects who stop active treatment during the study
  • the long-term safety, efficacy and PK of an intermittent dose of the drug in patients unable to tolerate weekly doses


Approximately 200 subjects.