DMD Hub Central Recruitment Pilot Project
The DMD Hub Central Recruitment Pilot Project is looking to create a national contact list of children and adults with DMD who are interested in participating research studies.
Over the past 10 years, there has been an increase in the number of DMD clinical trials and research studies evaluating the effect of new interventions and treatments, better understanding the natural course of the disease, and exploring new markers to measure disease progression or treatment effect. Due to the complexity of the disease and of setting up clinical sites to conduct them, these studies are often run in a limited number of centres in the UK. Inevitably this creates some challenges to both clinicians and patients and families.
The objective of the DMD Hub Central Recruitment Pilot Project is to assess the benefit to patients and trial sites of maintaining a centrally coordinated national recruitment contact list of children and adults with DMD who are interested in participating in DMD research studies.
This pilot study will establish a national contact list of national children and adults with DMD, which will be a computer record containing information about people diagnosed with DMD, their condition and their preferences relating to participation in DMD research studies. The database will contain information that will support clinical sites to identify potentially eligible candidates for research studies (including participant genetic diagnosis and other key details of their disease) as well as the contact details for their main neuromuscular centre and clinician.
Participants will be asked to consent to take part in the pilot study, and to consent to the responsible member of the DMD Hub staff contacting their main neuromuscular clinician to confirm some of the information provided, if required.
This pilot study is funded by Duchenne UK and an initiative led by the DMD Hub. Launched in 2016, the DMD Hub is a collaboration between the two neuromuscular centres of excellence (The John Walton Muscular Dystrophy Research Centre (Newcastle University) and Great Ormond Street Hospital (London) and Duchenne UK). The DMD Hub is a network of clinical trial sites with dedicated, trained staff funded to carry out research studies for DMD. It uses existing UK clinical trial expertise, to also provide a central resource offering advice, guidance and training to sites less experienced in running DMD research studies. Ultimately, the mission of the DMD Hub is to ensure all patients with DMD, both children and adults, have access to clinical research trials.
It is anticipated that the pilot study will be active for 12 months.