Queen Square Centre for Neuromuscular Diseases


A phase III study of Ataluren (PTC124) in patients with nonsense mutation dystrophinopathy

Sponsor PTC therapeutics
CI Professor Kate Bushby
sites Newcastle and London GOSH

Newcastle: becky.davis@ncl.ac.uk

London: c.uzowuru@ucl.ac.uk

More information

Trial information

The primary objective: to determine the ability of ataluren to slow disease progression. This will be assessed by ambulatory decline (decrease in 6 Minute Walking Distance)

Secondary endpoints: to evaluate changes in skeletal muscle function through assessment of proximal muscle function by:

  • the time to run/walk 10 meters
  • time to ascend 4 stairs
  • time to descend 4 stairs
  • patient or parent/caregiver perception of physical functioning.

Additional secondary endpoints have been selected to enhance understanding of the primary and secondary treatment effects.

For example, a beneficial effect in physical function relative to placebo, as assessed by the North Star Ambulatory Assessment (NSAA), would complement positive changes in ambulation proximal muscle function.

Collection of patient and/or parent reported changes in disease status provides an opportunity to expand the implications of a drug effect on the patient's disease symptoms and daily activities.