Accelerating drug development

New staff join the DMD Hub to run clinical trials for DMD treatments

The DMD Hub is one of Duchenne UK’s flagship projects and has successfully expanded capacity for Duchenne muscular dystrophy (DMD) clinical trials in the UK. By funding key posts at hospital sites across the UK, many more boys are now able to take part in trials for potentially life-changing treatments.

We were delighted to welcome the following new staff to the DMD Hub network. Each team member will play an important role in running effective trials for potential DMD treatments: 

Charlotte Lillien, Physiotherapist, Oxford University Hospitals NHS Foundation Trust  

Charlotte Lilien joined the team at the DMD Hub site, at Oxford University Hospitals NHS Foundation Trust (OUH), as senior research physiotherapist to actively support the development of DMD clinical trials. 

Charlotte’s role will also be embedded in the Specialised Translational Research Oxford Neuromuscular Group (STRONG), within the MDUK Oxford Neuromuscular Centre. 

OUH joined the DMD Hub in 2019 and has previously run the Santhera SIDEROS and WAVE Life Sciences’ DYSTANCE 51 trials, and is currently recruiting for the Sarepta ESSENCE trial. 

Charlotte is qualified as a Master trainer and consultant for the scales and outcome measures used in neuromuscular clinics and clinical trials.  

As well as helping run DMD clinical trials, her role at Oxford involves developing procedures and resources for assessing patients during clinical trials and helping to define the role of research physiotherapists. She also trains paediatric physiotherapists at the John Radcliffe Hospital on the outcome measures and scales used in DMD care and trials. 

Professor Laurent Servais, Professor of Paediatric Neuromuscular Diseases at the MDUK Oxford Neuromuscular Centre, says: 

“Charlotte has surpassed my expectations, becoming not only a specialist of the evaluation and standards of care of people living with neuromuscular diseases, but also a trainer for all outcome measures. She has plenty of ideas that will be developed here in Oxford as soon as we can.” 

Naomi Barkby, Research Nurse, and Andrew Cameron, Clinical Trials Administrator, Leeds Teaching Hospital 

Two new team members have joined Leeds Teaching Hospital to expand their capacity for clinical trials.  

Leeds Teaching Hospital joined the Hub in 2018 and to date has run 11 clinical trials for DMD. This includes TAM-DMD and TAM-DMD Open Label Extension, which are investigating the use of existing drug Tamoxifen to treat DMD. They have also run the VISION-DMD study for Vamorolone, and the long-term analysis of Translarna, the only treatment currently approved for boys whose DMD is caused by a nonsense mutation.  

The Principal Investigator (PI) leading trials at the site, Dr Anne-Marie Childs, has worked tirelessly to bring more trials and patients to Leeds over the past three years. The additional research nurse and clinical trials administrator has allowed the team to recruit a further eight boys over and above their original target numbers, including patients from outside Yorkshire who otherwise would not have the opportunity to take part in trials. Three further interventional studies (Mi51ion, FibroGen 093 and 094) are scheduled to open before the end of the year. 

Duchenne UK initially funded two key posts at Leeds: a neuromuscular coordinator and a clinical research fellow. Thanks to our funding model, which encourages posts to be sustained by the NHS Trust once our grant has ended, both posts are now funded by the Trust. The clinical research fellow has since become a neuromuscular consultant, taking a leading role in conducting clinical trials. 

Amy Wyatt, Research Physiotherapist and Chloe Perry, Clinical Trial Coordinator, Robert Jones and Agnes Hunt Hospital (RJAHH), Oswestry 

Two new team members were appointed at Oswestry, one of the newest sites to join the DMD Hub, in May 2021. The neuromuscular team is led by Tracey Wills, who also chairs the West Midlands Neuromuscular Network and is a member of the North West Neuromuscular Network.  

Amy, as a research physiotherapist, will undertake the tests and measurements required as part of clinical trials. Chloe, as a clinical trial coordinator, will work with patients and their families, as well as pharmaceutical companies, to help set up clinical trials and help them run smoothly.  

Funding has also been agreed for an Advanced Nurse Practitioner (ANP) role. ANPs are highly qualified nurses, able to take on some clinical trial tasks traditionally taken on by doctorsThis role will therefore relieve some tasks from the Principal Investigator (the lead doctor on trials) and provide much-needed additional support for trial teams.  

The Oswestry site is currently running Italfarmaco’s Givinostat trial, and was part of Santhera’s SIDEROS and Open Label Extension trial programme.  

Professor Tracey Willis, Consultant Paediatric Neurologist at the RJAHH says “With funding for the extra posts, this means that we can consider more clinical trials in Oswestry and have flexibility within the team, due to the increased numbers, to arrange scheduling as required and more innovative ways of running them to accommodate more children and young people who are eligible for the trials.” 

Amy Wyatt says: “I am looking forwards to getting to know the boys and families involved in the Givinostat trial. It is an exciting time for research within the neuromuscular world with lots of progress being made and it is exciting to be involved in looking at treatments that can slow or improve this condition.” 

To find out more about clinical trials and their important role in discovering new treatments for DMD, visit the DMD Hub website. 

Thank you to the following Family & Friends Funds for contributing to the funding of these posts: Help Harry, Archie’s March, Jacobi’s Wish, Project GO, Moving Muscles for Marcus, Backing Jack, Standing with Jack, Chasing Connor’s Cure 

Published on 13 August 2021

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