The FOR-DMD trial

As part of an ancillary study running alongside the FOR-DMD trial all participating boys will be asked if they are willing to provide blood samples that would be stored in the EuroBioBank. The blood samples which will be collected at baseline and annually thereafter will be used to obtain serum and extract DNA and RNA and then stored in the MRC Neuromuscular Centre BioBank in Newcastle upon Tyne, UK for use in approved research projects in the future.

Proteomics and genomics studies are planned and samples will be available for further future researches.

Other researchers wishing to use samples from the EuroBioBank must obtain approval from the MRC BioBank Committee.

The results obtained are expected to improve knowledge of Duchenne muscular dystrophy and may result in useful benefits for the community as a whole. The FOR DMD study provides a unique opportunity to collect samples from boys affected by DMD. Samples will be of partcisulr value as they will be collected at baseline (young children, pre-steroid treatment) and longitudinally thereafter (up to 5 years, as per the duration of the study).

The study received great feedback from the Data and Safety Monitoring Board - DSMB who commented: "Although not directly within the purview of the FOR DMD study, this unique opportunity to collect genetic material prospectively from DMD subjects who are thoroughly characterized in a 3 year longitudinal study will provide an extremely valuable resource to investigate genetic modifiers of disease course or response to steroids. Similarly, a unique collection of serum from each subject before and during treatment would be of great value for additional investigations".

12 Apr 2017