Gene Therapy Recruitment Statement

Gene Therapy is a promising new treatment for DMD and the DMD Hub is committed to making the recruitment process to these trials as clear as possible for the patient community

  • 24 Feb 2021

The DMD Hub is coordinating a 1-year pilot trial to scope and, if possible, implement a nationally agreed process for fair and equitable recruitment to DMD clinical trials in the UK.

However, with sites planning to start screening patients for DMD gene therapy trials in quarter 1 of 2021, it is clear that this process will not be able to be implemented in time.

Therefore, the DMD Hub Principal Investigators (PIs) have agreed an interim statement to update the patient community on the plans for recruiting to gene therapy trials, as follows: 

“Clinical trial sites running gene therapy trials will follow established local processes for recruitment with the aim of also recruiting patients, whose primary care is not provided at one of the recruiting trial sites. However, with the specific challenges of gene therapy trials and travel restrictions imposed by COVID, it is not yet clear when recruitment of patients, whose primary care is not managed by the recruiting site, will be possible. Sites will use their best judgement to minimise risks for patients and optimise patient screening and recruitment”.

Recruitment of patients into clinical trials will ultimately be driven by the objective to give a trial the best chance to succeed. Recruitment is based on strict inclusion and exclusion criteria, a stringent schedule of investigations and a pre-selection of patients that best comply with these requirements.

It is expected that up to 3 clinical trials sites (Newcastle, Great Ormond Street and Alder Hey) will be recruiting a limited number of patients for the first gene therapy trial, Pfizer - DMD C3391003 (please click here to see the eligibility criteria), but additional UK sites may join either in this study or other upcoming gene therapy trials in DMD. Updates will be available on the DMD Clinical Trial Finder.

Patients are encouraged to express their interest in participating in gene therapy clinical trials to their neuromuscular specialist who will collect relevant information and share anonymised details of potentially eligible patients with the recruiting sites. The DMD Hub will work with trial and care sites to help coordinate recruitment and to keep patients informed.

Patients who are registered on the GOSH central database, which includes out of area patients, will be considered for recruitment at GOSH via a locally established procedure.


About the DMD Hub

Duchenne UK’s mission through the DMD Hub is to give everyone diagnosed with DMD access to research opportunities. In just four years, Duchenne UK has funded 32 posts, brought more companies and income to the NHS through pharmaceutical studies, and helped recruit more than 300 boys to take part in DMD trials who otherwise may not have had that opportunity.

To find out more about the DMD Hub, you can read our DMD Hub Impact Report 2020.

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