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Abstracts & Posters

DMD HUB: Expanding clinical trial capacity for Duchenne muscular dystrophy in the UK

key information

source: Neuromuscular Disorders

year: 2017

authors: E. Heslop, V. Straub, M. Guglieri, C. Turner, B. Davis, E. Crossley, A. Johnson, K. Bushby


With Duchenne muscular dystrophy (DMD) clinical research at an unprecedented stage in terms of the number of possible therapeutic approaches coming to trials, a workshop was organized to bring together 75 participants from the DMD patient and research community, as well as industry to address concerns regarding the apparent lack of capacity for trials in the UK. Specifically, clinicians in established UK clinical trial centers involved in multiple DMD studies are reaching capacity, while centers that have capacity but lack the expertise need support to develop to achieve the requirements set by industry to run clinical trials. A key outcome of the workshop was the recommendation to establish a UK Hub to expand the clinical trial capacity for DMD. The Hub started in 2016, funded by Duchenne UK, and we are now working in conjunction with staff and facilities available at sites to implement a network of UK sites, providing expert DMD advice, guidance, and training to enable them to take on trials thereby relieving pressure on experienced sites. The Hub is collaborating with existing organizations such as TREAT-NMD, the National Institutes of Health Research, the North Star Network, patient advocacy groups and the DMD patient registry run by action Duchenne to avoid duplication of the significant work already achieved in this area. Within the first year of operation the Hub expects 2–3 new trial sites to be selected for industry led DMD trials in the UK while ongoing training for other sites should open opportunities even further in subsequent years. The mission of the DMD Hub ultimately is to ensure all patients with DMD, including children and adults, have access to clinical research opportunities. Issues of sustainability and expansion of the model to other rare neuromuscular diseases are being explored.

organization: Newcastle University, UK; Duchenne UK, UK

DOI: 10.1016/j.nmd.2017.06.035

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