FOR- DMD- double blind randomized trial to find the optimum steroid regime for DMD

FOR- DMD- double blind randomized trial to find the optimum steroid regime for DMD MSG/ TREAT-NMD collaboration Funded by NINDS

The story of the trial started in 2003……

• Inconsistency in corticosteroid dosage regimes for DMD identified as top priority for further work • Multiple steroid regimens in use is a major cause of confusion and stops some children being offered steroids at all or at suboptimal doses • International steering group with patient organisation support and several working groups for trial design • NIH planning grant 2005, full grant submission 2007, resubmissions June 2008, 2009, final funding decision September 2009, money to sites??

105 US DMD clinics were surveyed in Jan 2008 • 27 different corticosteroid regimens were reported • The 5 most frequently used regimens are      

0.75 mg/kg/d prednisone, 61 centers 75 mg/kg/d prednisone 10 days on/10 days off, 36 centers 0.9 mg /kg/d deflazacort, 32 centers; 5mg/kg/d prednisone weekends, 10 centers 0.75 mg/kg/d prednisone, alternate days, 10 sites ~10% of centers do not routinely prescribe corticosteroids

• Survey of TREAT-NMD registries in 23 countries  In 4-7 year old age group 57% not on steroids with wide variation country- country

Inconsistency in use of steroids remains a major clinical problem and may be a threat to ongoing and planned trials of innovative therapies The question here is not if steroids work in DMD but rather how best to use them

Trial organisation • MSG data management and statistics, NIH communication • Newcastle CTU master files etc • TREAT-NMD trials co-ordination centre training, dissemination of information, site initiation, monitoring  The co-ordination of the trial will combine two networks working together to cover sites in the US and Europe

Interaction with new treatments?

• Further phase I/II/III trials will be initiated in the course of the trial  We understand that it is really important for people to have the opportunity to take part in these trials

• As steroid use is the current “gold standard” participation in this trial should not prevent recruitment to other studies • The best way to use steroids in DMD remains an important question which is why this trial is also an important study • The way forward is likely to be with additive therapies for the foreseeable future- the appropriate use of steroids is a key part of that