G.P.116: SCOPE-DMD, an EU FP7 funded consortium for skipping trail across Europe in Duchenne muscular dystrophy

Abstract

The SCOPE-DMD project builds on results from the TREAT-NMD and BIO-NMD EU projects to investigate a personalised-medicinal product through to market for the treatment of Duchenne Muscular Dystrophy (DMD). The initial goal of the project is to develop a therapy that can restore the expression of a functional dystrophin protein in targeted DMD patients that would potentially benefit from skipping of exon 45 of the DMD gene. The project uses an innovative clinical study design and novel outcome measures to reduce development timelines. The SCOPE-DMD study is an exploratory, open-label, dose-escalation phase IIb clinical trial that will assess the efficacy, safety, pharmacodynamics and pharmacokinetics of weekly subcutaneous doses of PRO045 in subjects with DMD. The study is of seamless design that is driven by the need to maximise the data from an exceptionally small patient population and to provide continuity of care in this rapidly progressing disease – a result of direct feedback from parents. It is made up of two phases: a dose escalation phase with 15 patients and an extended pivotal phase with an additional 55 patients. The consortium consists of 2 SMEs and 3 academic institutes coordinated by Newcastle University. Based on extensive experience from clinical trials of two other AONs in DMD patients, the consortium has worked on a highly innovative development plan that if successful, could be applied to future clinical trials in DMD and other rare diseases. Also magnetic resonance imaging, novel biomarkers and functional outcome measures including accelerometry, myotools and the Performance of Upper Limb (PUL) module will be incorporated into the trial protocol in addition to more established ones. Both expertise and scientific knowledge come together in this consortium to launch an innovative drug product but also to provide a regulatory and pathway-to-market precedence to benefit future patients with rare disorders getting earlier access to treatment.