Special Protocol Assessment (SPA) approval has been given to Ark Therapeutics Group plc of London for a phase III pivotal trial of Trinam, a novel gene-based medicine to prevent blood vessel blockage in kidney dialysis patients who have undergone vascular access graft surgery.
Results of a phase II open-label, non-randomized trial showed that access grafts of patients who received the planned dose of Trinam exhibited almost three times the primary unassisted patency compared with controls.
The phase III study will be a U.S. multicenter, randomized, controlled trial in which Trinam’s efficacy and safety will be investigated in patients with end-stage renal disease requiring vascular access for hemodialysis. While the SPA was reviewed, Ark conducted a preclinical study and provided extra biodistribution data on an “end-to-side” surgical procedure for placement of the graft.
The resulting data were in line with previous findings, allowing the phase III trial to include this procedure alongside the existing “end-to-end” placement procedure used in phase II.
In addition to beginning work on the phase III trial, the company intends to apply to the FDA for fast-track designation.