Duchenne UK and University Children’s Hospital Basel: Support for repurposing Tamoxifen for Duchenne Muscular Dystrophy: a phase 3 RCT (TAM DMD)


Clinical Trials

Poster Number: 39


David Bull, PhD, Fiona Lawrence, Emily Crossley, Alex Johnson, Dirk Fischer


1. Duchenne UK, 2. Duchenne UK, 3. Duchenne UK, 4. Duchenne UK, 5. University Children's Hospital Basel, University of Basel, Basel, Switzerland

DUK is the leading UK funder of Duchenne research, working with industry, academics and other stakeholders (NICE/NHS) to help accelerate research and tackle drug development barriers. Prof Dirk Fisher, UKBB, was awarded an ERA-Net (E-Rare) grant to support a multi-centre, phase 3, randomised, placebo controlled clinical trial of tamoxifen (TAMDMD). This grant followed the generation of promising pre-clinical data by Dr Olivier Dorchies in a DMD mouse model in the laboratory of Prof Scapozza at University of Geneva.

DUK became involved with this re-purposing project in order to support the development of tamoxifen for DMD by providing funding and regulatory knowledge in order to facilitate early access in Europe

DUK’s role included:
•Application for Orphan Drug Designation (ODD) for tamoxifen
•Providing regulatory support for scientific advice from EMA to agree the design of a phase 3 European multicentre trial (TAMDMD)
•Extending TAMDMD to include UK Investigator sites, boosting number of trial sites and speeding up recruitment
•Funding a project manager post, full-time, at UKBB to coordinate the trial

•ODD for tamoxifen granted by EMA
•Agreement with EMA of primary endpoint and number of participants to be recruited to TAMDMD
•The TAMDMD trial was started 06/2018, and 05/2019 in the UK
•Recruitment at 3 UK investigator sites
•Currently (Oct 2019), there have been 67 patients screened and 53 randomised. A further 8 screenings are planned. In the UK, there have been 9 patients screened and all randomised. A further 5 screenings are planned in Glasgow in November

•Patient organisations can be powerful partners in the development of repurposed drugs
•The addition of 3 UK sites was critical to the rapid completion of recruitment
•Funding of the project manager allowed continuity and efficiency for the whole project
•Engaging early with EMA increased confidence in the regulatory validity of the trial design