Sponsor | PTC therapeutics |
CI | Professor Kate Bushby |
sites | Newcastle and London GOSH |
Contact |
Newcastle: becky.davis@ncl.ac.uk London: c.uzowuru@ucl.ac.uk |
More information |
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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.