Duchenne UK’s ambition through the DMD Hub is that everyone in the UK diagnosed with DMD is given the opportunity to take part in research.
In 2014 Duchenne UK teamed up with Newcastle and Great Ormond Street hospitals to address a chronic lack of clinical trial capacity for Duchenne Muscular Dystrophy (DMD) in the UK. Despite there being a wealth of knowledge and experience at both these sites, they were turning away trials for lack of doctors and nurses.
This was not acceptable to us, so, in a ground-breaking collaboration with the NHS and NIHR, we created the DMD Hub.
To date, we have invested more than £2.7 million into the DMD Hub, supporting patients, sites and industry partners.
NINE sites have now joined the DMD Hub and we are delighted to say that in just THREE years, we have funded 30 posts, and helped recruit more than 250 boys to take part in trials, who otherwise would not have had that opportunity. We have also brought more companies and income to the DMD Hubs sites through pharma studies.
We keep in constant touch with the sites so that we can update our clinical trial tracker where patients can find up to date information on clinical trials in the UK.
Alder Hey was one of the first hospitals to become a DMD Hub site in 2017, whilst Bristol and Birmingham both joined the DMD Hub last year.
At Alder Hey, we were funding a Clinical Research Nurse, Tim Henderson, along with our partner charities, Joining Jack and Alex’s Wish. This post has been key to increasing Alder Hey’s research capacity enabling them to run additional clinical trials. They are currently recruiting patients for the TAM-DMD and Vision-DMD trials, as well as running the Wave Phase 1 and Open Label Extension for exon 51 skipping, Italfarmco and FOR-DMD, amongst others. Between 2018 and 2019, they have conducted 8 clinical trials and have recruited 50 patients.
For more information about Alder Hey, the team there and the trials they are running visit: https://dmdhub.org/hub-sites/where-are-the-dmd-hub-sites/alder-hey/
Bristol’s DMD Hub Trial Coordinator, Mashal Asif, has been in post since April 2019 and has been assisting in the delivery of trials. They are currently running 2 clinical trials (Wave Open Label Extension and Polaris-DMD) and 2 natural history studies (Translarna and Rare Disease Translational Research). Since 2018, they have recruited 16 patients on to these 4 trials.
For more information about Bristol, the team there and the trials they are running visit: https://dmdhub.org/hub-sites/where-are-the-dmd-hub-sites/bristol/
The DMD Hub have supported Birmingham by providing funding for a consultant to spend time dedicated to research and expanding clinical trial capacity. In addition to the FOR-DMD clinical trial and natural history studies, there are several trials in the set-up phase which will be announced soon. Part of the income from these trials will be used to fund an extra neuromuscular research physiotherapist to support the team and help increase trial capacity further locally.
For more information about Birmingham, the team there and the trials they are running visit: https://dmdhub.org/hub-sites/where-are-the-dmd-hub-sites/birmingham/
We responded to the needs of patients who were struggling to find out information on clinical trials and where they were happening. So we launched the DMD HUB WEBSITE DMDHUB.ORG, to provide patients with trustworthy and up to date information about clinical trials in the UK.
The website provides FAQs on what to consider before joining a trial, there is also an interactive map with information on all the DMD Hub sites.
DMDHUB.org also has sections for hospitals and drug development companies to support them to setup and run clinical trials.
We look forward to keeping you updated on the DMD Hub.
Use the clinical trial finder to find every existing and pending clinical trial for Duchenne in the UK: https://dmdhub.org/clinical-trial-finder/
To find out the latest news from the Hub, you can sign up to our newsletter here: https://dmdhub.org/join-the-hub/