On 15th October 2021, the Duchenne Muscular Dystrophy (DMD) Hub held an interactive workshop in Newcastle aimed at nurses and clinical trial coordinators involved or interested in the set-up, management and delivery of Duchenne muscular dystrophy (DMD) gene therapy trials.
The workshop facilitated the exchange of ideas, experiences, and common challenges in delivering DMD gene therapy (GT) trials. There was also the opportunity for people in different roles, such as a Principle Investigator, Clinical Trial Coordinator and Nurse, to share their experiences and one key session enabled the parent and patient perspective of GT trial recruitment to be discussed.
The DMD Hub works collaboratively with NA-ATTC to address the need for training and education to support implementation of GT clinical studies and subsequent rollout. Sarah Hanson, Senior Paediatric Research Nurse (Leeds Teaching Hospitals Trust) presented the NA-ATTC roadmap for nurses’ training and Kelsie Thomas, NA-ATTC Project Manager (The Newcastle upon Tyne Hospitals NHS Foundation Trust), shared the practical tools to facilitate delivery of gene therapy clinical trials highlighting the NA-ATTC Advanced Therapy Clinical Trials Toolbox.
This workshop is a result of the DMD Hub’s objective to accelerate NHS site readiness for DMD trials. Indeed, its work enabled Newcastle to be the first trial site in the UK to recruit into the CIFFREO trial earlier this year. The trial, sponsored by Pfizer, is evaluating the safety and efficacy of PF-06939926 for the treatment of Duchenne muscular dystrophy. This was an exciting achievement as it was the first GT trial in Duchenne muscular dystrophy to open in the UK.
Further information on the DMD Hub can be found on the DMD website.