Queen Square Centre for Neuromuscular Diseases


Duchenne muscular dystrophy (DMD): double-blind randomized trial to find optimum steroid regimen

Sponsor University of Rochester
Funder NIH
CI Professor Kate Bushby
UK sites Newcastle, London GOSH
Contact details Newcastle: Gillian.Kenyon@newcastle.ac.uk; London: c.uzowuru@ucl.ac.uk
More information

Trial information

This is a multi-centre, double-blind, parallel group, 36-60 month study. It will compare three corticosteroid regimens in wide use in DMD:

  • daily prednisone (0.75 mg/kg/day)
  • intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
  • daily deflazacort (0.9 mg/kg/day)

Primary objective

 Test if daily corticosteroids will be better for function and subject/parent satisfaction than intermittent corticosteroids

A second hypothesis is that daily deflazacort will show fewer side effects than daily prednisone.

The study protocol includes:

  • Standardized regimens for prevention/ treatment of predictable side effects of corticosteroid medication,
  • Standards of care for the general management of DMD.

The trial directly addresses the current chaos in prescribed treatment schedules. The results will have a direct impact on the management of boys with DMD throughout the world. It will also provide the evidence base:

  • for clear and specific guidelines for patient treatment
  • for rational clinical practice.