The DMD Hub is a network of trial sites with trained staff which are funded to carry out clinical trials for DMD.
The DMD Hub uses existing UK clinical trial expertise, to provide a central resource offering advice, guidance and training to sites less experienced in running DMD clinical trials.
The DMD Hub engages with key stakeholders at sites and in industry. We facilitate communications between them to accelerate trial readiness. This has already increased the number of UK sites which run DMD trials.
Duchenne muscular dystrophy (DMD) is the most common fatal genetic disease diagnosed in childhood. The disease almost always affect boys, and they tend to be diagnosed before the age of 5.
Duchenne muscular dystrophy is classified as a rare disease. There are around 2,500 patients in the UK and an estimated 300,000 sufferers worldwide.
Before the DMD Hub was launched, clinical trials for DMD were only conducted at Great Ormond Street Hospital (GOSH) in London and the Newcastle-upon-Tyne Hospital.
These sites were working at full capacity and we found out they were turning away trials from drug companies. This meant that only a few clinical trials could be conducted for DMD. So very few patients were able to participate in trials each year.
In 2016, Duchenne UK launched The DMD Hub.
We are now working with staff at the two original sites, or centres of excellence, to develop a network of sites across the UK. GOSH and Newcastle are providing expert DMD advice and training to enable new sites in the UK to begin running trials. This will relieve pressure on the experienced sites.
Within the first year of operation, the DMD Hub has already funded five new trial sites in the UK, which are now running industry-led DMD trials.
Our ongoing training at other sites should open more new opportunities in the future.
Ultimately, the mission of the DMD Hub is to ensure all patients with DMD, both children and adults, have access to clinical research trials.
The DMD Hub uses a staged funding model when providing funding to DMD Hub sites. This ensures any posts introduced to NHS Trusts by the DMD Hub are sustainable once the grant has ended.