Pilot Study Comparing Prophylactic Wilate Versus Placebo for Heavy Menstrual Bleeding in Women with Von Willebrand Disease

What this Trial Is About

Researchers are conducting the EMPOWER trial, a pilot multi-center, placebo-controlled, double-blind, crossover randomized trial lasting two years. It focuses on female outpatients with von Willebrand disease VWD who experience heavy menstrual bleeding HMB. The study aims to assess whether this trial design is feasible and viable, and to explore the sensitivity of clinical outcomes to guide a future definitive trial. Participants will be randomly assigned to receive either a plasma-derived von Willebrand factorFactor VIII concentrate called Wilate4 or a placebo normal saline, both given with standard care. Treatment is provided over four menstrual cycles during the first period, followed by a one-cycle washout without study treatment. Then, participants switch to the other treatment in the second period. Wilate4 is administered intravenously at doses of 30-60 IU VWFRCokg on the two heaviest bleeding days within the first four days of menstruation, with optional additional doses. During the study, participants receive infusions by a nurse and use specific feminine hygiene products supplied by the sponsor. Researchers will evaluate trial feasibility by measuring participant retention, blinding success, and data completion. They will also assess menstrual bleeding severity using a modified pictorial blood assessment chart mPBAC and monitor clinical outcomes like bleeding events, hemoglobin and ferritin levels, fatigue, and adverse reactions. The total study participation is two years, including both treatment periods and washout.

A Pilot Crossover Trial of Prophylactic Wilate Compared to Placebo for Heavy Menstrual Bleeding in Patients with VWD