FOR-DMD

• Research type

  Research Study

• Full title

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

• IRAS ID

  46681

• Contact name

  Kate Bushby

• Contact email

  kate.bushby@ncl.ac.uk

• Sponsor organisation

  University of Rochester

• Eudract number

  2010-023744-33

• ISRCTN Number

  n/a

• Research summary

  The aim of the study is to identify the optimum steroid regimen for boys with Duchenne muscular dystrophy (DMD), with regard to functional outcomes and participant/parent satisfaction. The study population will be boys aged four to eight years old at screening, with confirmed DMD (proximal muscle weakness AND an out of frame deletion OR frame-shifting dystrophin point mutation OR absent/< 3% dystrophin on muscle biopsy), able to rise independently from the floor at that time, and not previously treated with oral corticosteroids. We will compare the three most commonly-used corticosteroid regimens:

  • 0.75mg/kg/day prednisolone
  • 0.75mg/kg/day prednisolone 10 days on/10 days off
  • 0.9mg/kg/day deflazacort

  The study treatment will last for a minimum of 36 months for all participants, and a maximum of 60 months for those recruited into the trial first (since it is anticipated that it will take up to two years to recruit the 300 participants). The longer duration of blinded intervention and follow-up for those subjects recruited early is in anticipation of the continued long-term unblinded follow-up of the full cohort of subjects at the end of this five-year study.

  The primary outcome variable is a multivariate (three-dimensional) measure, comprising two dimensions of function (time to rise from the floor, forced vital capacity) and one of satisfaction (global treatment satisfaction).

  We will also compare the three corticosteroid regimens with regard to tolerability, Adverse Events, secondary functional outcomes, including six-minute walk test and disease milestones, Quality of Life and cardiac function.

• REC name

  South Central - Hampshire B Research Ethics Committee

• REC reference

  11/SC/0543

• Date of REC Opinion

  24 Jan 2012

• REC opinion

  Further Information Favourable Opinion