A Multi-cEnter, Pilot, Crossover Trial of Prophylactic Wilate CoMpared to PlacebO for Heavy Menstrual Bleeding in Patients with Von WillEbRand Disease
The EMPOWER trial is a pilot multi-center, placebo-controlled (normal saline), double-blind (patient and outcome assessor), crossover, 2-year randomized trial in female outpatients with von Willebrand disease (VWD) and heavy menstrual bleeding to determine trial feasibility and viability, and to explore assay sensitivity of the proposed efficacy clinical outcomes for a definitive randomized controlled trial
• Patient capable of providing informed consent;
• Female patients with HMB over the age of 18 years, for whom prophylactic treatment with Wilate® is deemed clinically appropriate according to the medical discretion (based on their expert opinion given consideration of the patient's bleeding history and responsiveness to treatment) of the treating hemostasis-focused physician practicing at a Hemophilia Treatment Center;
• Modified PBAC score \> 100 at screening;
• Patients with a diagnosis of inherited von Willebrand disease (any type);
• Stable treatment for HMB and iron deficiency anemia for 3 cycles before entering the study and anticipated to remain unchanged for the duration of the study;
• Patients willing to have an infusion administered by a nurse over the course of the study period;
• Patients who agree to use only the feminine hygiene products supplied by the sponsor.