Duchenne muscular dystrophy steroid trial launched

Published Date
Neil Bennett
Drugs 72dpi

Researchers led by Professor Katie Bushby in Newcastle have started recruiting participants to a clinical trial that aims to identify the best way for doctors to give corticosteroids to boys with Duchenne muscular dystrophy and is being supported by the Muscular Dystrophy Campaign-funded clinical trials coordinator.

Officially titled the “Duchenne Muscular Dystrophy: Double-blind Randomized Trial to Find Optimum Steroid Regimen”, researchers aim to recruit young boys (4-7 years old) with Duchenne muscular dystrophy who have not yet started taking steroids. The trial aims to identify the best way for doctors to give corticosteroids to boys with Duchenne muscular dystrophy.

The trial is being organised by Professor Katie Bushby at Newcastle University and Professor Robert Griggs at the University of Rochester in the USA. Ten locations in the UK are now recruiting participants – including two hospitals in London, and one each in Leeds, Glasgow, Cardiff, Birmingham, Liverpool, Manchester, Newcastle and Oxford. Other trials sites are located in Italy, Germany, Canada and the USA. 

Professor Francesco Muntoni, a clinician involved in the study said:

After many years of discussions regarding the best way to give corticosteroids to boys with DMD, this is the first study which will systematically compare efficacy and side effects of different regimens of steroids. This study will also provide additional information on how to best manage steroids induced side effects in DMD, so it is a very important study that will have long lasting repercussion in the way steorids are prescribed worldwide.

To find out more about the For-DMD trial you can visit the study’s website. On the website, you will also find the full list and contact details of the UK sites participating in the trial and will be able to recruit boys. People who are interested in participating in the trial and would like more detailed information or to discuss participation, can contact the study team directly at michela.guglieri@ncl.ac.uk or by phoning +44 (0)191 2227623

Background information

Treatment with corticosteroids is a part of the most recent standards of care, and the drugs have been given to boys with Duchenne muscular dystrophy for over 20 years. They are currently the only treatment that keeps boys walking for longer and they may also help to prevent some heart problems.

No proper clinical trial has ever investigated the best way to use steroids in boys with Duchenne muscular dystrophy over a long period of time. This means that different clinicians prescribe steroids differently – with the type and dose regime of steroid often varying between clinicans, hospitals and countries. In the UK there is a more or less 50:50 split between boys on daily or intermittent regimes of prednisolone, but in the USA and Canada many doctors favour a different steroid, deflazacort. Which regime is used is more dependent on the clinician prescribing it than on the results of any unbiased studies into their use. A trial is needed to study this so that any bias in choice of steroid and any bias in the reporting of benefits and side effects can be taken out of the equation. The end result of this trial should be to find out which steroid and dose is most effective in boys with Duchenne muscular dystrophy and to examine the side effects caused by different treatments and how to minimise them. All boys in the trial will be given steroids, with one third receiving deflazacort and others taking prednisolone either every day, or intermittently (ten days on, ten days off).

The researchers aim to recruit 300 boys aged between four and seven who have not taken steroids before. The participants will visit a trial site every six months for between three and five years; at each visit clinicians will perform a number of tests and measurements. These will include physical examination and checking vital signs (height, weight, waist circumference, blood pressure, pulse, and lung capacity). There will also be motor skills tests (jumping, hopping, time to stand from lying etc) and a six-minute walk test.

Links and further information

To read more about Duchenne muscular dystrophy

See the research we are funding into Duchenne muscular dystrophy

To look at our clinical trials FAQ

It is only through your contributions that we can continue to fund the vital work that takes us closer to finding treatments and cures for muscle-wasting conditions. Donate now and help change the lives of thousands of people living with muscular dystrophy and related neuromuscular conditions.


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