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)
Phrixus Pharmaceuticals Inc, USA Non-confidential report
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.
Name of applicant: Dr Symons Reviewed: June, 2010 in Barcelona, Spain
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.
