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| 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.