An open-label, fixed dose, exploratory study to assess the efficacy and safety of P-188 NF on left ventricular volume changes in patients with Duchenne muscular dystrophy (DMD).

Dr Symons
Phrixus Pharmaceuticals Inc, USA

Saturday 5th June 2010

In summary, TACT recommended that in order for P-188 to enter clinical trials in DMD additional pre-clinical studies were required using the route, dose and frequency proposed for human acute and chronic studies. TACT considered it important to assess the action of P-188 in conjunction with corticosteroids and compared to ACE inhibitors, as these are currently the standard of care in DMD. TACT concluded that, following the suggested pre-clinical studies and toxicity studies, a dose escalation study was essential to guide further development, to ensure that an effective dose could be determined in DMD and to make a go/no-go decision regarding further development of P-188. Based on the initial safety evaluation and dose selection, the next step would be a randomised placebo controlled dose escalation trial to establish the potential benefit over sufficient time to observe cardiac remodelling.

12 Apr 2017