(NU/1257) Clinical Trail Unit or Contracted Research Organisation to Set up and Manage Vision DMD Phase 2b and extension study

Tender description

Building on positive preclinical and Phase 1 results funded by government grants and international patient groups and based on FDA and EMA advice, VISION-DMD proposes a Phase 2 registration directed clinical programme aimed at an affordable therapy: Phase 2a will study the safety and tolerability of ascending doses of Vamorolone in ambulant DMD boys; Phase 2b will demonstrate the efficacy and safety of two doses of Vamorolone in young ambulant DMD boys. Keywords: Clinical Trial, Research Manager