DMD Hub Central Recruitment Project continues after successful first year

The DMD Hub Central Recruitment Database Pilot Project (CRD) was launched with the aim of allowing faster, fairer and more effective recruitment for clinical trials.  One year on, and we now have 176 registered participants who have told us about their preferences for participating in clinical research for DMD.

Over the past year, the CRD has received enquiries from 5 different clinical trial sites asking for potentially suitable patients to be referred to them for 9 different clinical research studies.  This has led to the successful recruitment of boys to gene therapy and exon skipping clinical trials, as well as several natural history studies.  Most critically, we are pairing families desperate to be involved in clinical trials who previously were deemed to live too far away to be eligible, with clinical trial opportunities.

In October 2022, 4-year-old Charlie from Aberdeen started on the EMBARK gene therapy trial, a clinical trial which he wouldn’t have been recruited for without the DMD Hub Central Recruitment Database Project.

Jennifer, Charlie’s mum said: “After realising Charlie may be eligible for some studies, I had contacted the relevant sites throughout the UK but as Charlie received his clinical care here in Aberdeen, our nearest trial site in Glasgow is still 150 miles away, it was unlikely he would be recruited to a site. The DMD Hub’s Central Recruitment project was possibly the only way that Charlie could have had a chance at participating in a trial.  Had it not been for this project, the recruiting site at Newcastle NHS Trust would not have had Charlie’s details. He would not have been recruited to the trial.”

Within a year, the CRD has already demonstrated that it is delivering fairer and more equitable access for patients to research studies in the UK. It is also actively assisting clinical sites in the UK with recruitment to clinical research studies in DMD. Due to this success, Duchenne UK, in partnership with Newcastle University, will continue to develop this resource, with the aim of giving fair and equitable access to clinical trial recruitment, and making the UK a more attractive place for clinical trials to take place.

Dr Michela Guglieri (Senior Clinical Lecturer and Consultant Neurologist at Newcastle University and Newcastle Hospitals NHS Foundation Trust & Primary Investigator for the CRD) said: “The CRD has proven to be a very useful tool for both families and clinicians, in facilitating recruitment to clinical trials and also making the UK a more attractive country for pharmaceutical companies, who are developing new therapeutic approaches for DMD, to run clinical trials. It really demonstrates how clinicians, and the patient community can work effectively together to bring research and hopefully treatments closer to patients.”

The DMD Community and clinical trial centres from across the UK were surveyed to understand their opinions around the DMD Hub Central Recruitment Database pilot project. Results from the survey demonstrate that the DMD community is supportive of the continuation of the DMD Hub CRD pilot project to an open-ended project, to ensure there is an established, fair process for clinical trial recruitment across the UK. You can read the full results here.

If you have any further questions about the CRD itself or about your continued participation, please contact Phillip Cammish (CRD Project Manager) by emailing dmdhub@newcastle.ac.uk  

Please visit DMD Hub Recruits for more details and to register now.