We are spearheading for the start-up phase of a Duchenne Muscular Dystrophy Trials Acceleration Programme (TAP). This will involve creating a Hub of expertise on clinical trials for DMD in the UK for both patients (ie young people with DMD and their families) and industry (ie those people undertaking clinical trials – scientists or larger biotech companies from around the world). There will be one central Hub, (provisionally sited at Newcastle) with links out to sites of clinical excellence in DMD in the UK. 

The UK is at the forefront of clinical trials in DMD and we have some of the world’s most respected doctors/professors practising here. However there is a big problem: it is a very complex and time-consuming process to get these therapies from the lab to the point of a clinical trial that will benefit patients.

Clinical trials typically take between 1 year and 18 months to set up - which is torture for parents. Most trials in DMD require boys to walk, so a delay of 18 months could mean that a boy who wants to take part in a trial can no longer do so. Instead of a trial taking up to two years it is our goal for the process to take less than 12 months from the point of contact with our hub. 

We will use the Hub to coordinate the existing network of DMD resources and also enable it to set up its own unique networks between DMD sites.

The aim would be that if a biotech company had a project that they wished to take to the clinical trial stage, instead of going to four or five sites the UK and waiting for responses, they would go straight to the Hub, who would assess their needs and be able to tell them where best to place the trial, and help with regulatory matters.

Importantly the Hub would also be able to monitor the trial and be a collection point for all information about current UK trials and their results, which would be invaluable for the science of DMD, and for patients.

 

Another way the Hub would work for the benefit of patients would be to enable sites, which are currently under-used to be Trial-ready.