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);
Diagnosed with any other known bleeding disorder;
Pregnancy or plans to become pregnant within the duration of the study;
Breastfeeding or plans to breastfeed within the duration of the study;
Known hypersensitivity reactions to human plasma-derived products or any ingredient in the formulation;
Crossover
Lyophilized concentrate of human coagulation von Willebrand Factor and factor VIII
DrugWilate® is a plasma-derived, highly purified concentrate administered through intravenous injection. Wilate® contains an average VWF ristocetin cofactor activity to FVIII activity at ratio of 1:1.
Placebo
Other interventionPatients randomized to the placebo arm will receive intravenous normal saline at the same approximate volume and frequency of Wilate ®.
Blinding Index (BI) score at the end of cycle 4 of treatment period 1 and 2
Blinding Index (BI) score at the end of cycle 4 of treatment period 1 and 2
Proportion of participant drop-out at the end of treatment period 1 and 2
Proportion of participant drop-out at the end of treatment period 1 and 2
Proportion of participants with completed for the candidate primary clinical efficacy outcomes at the end of treatment period 1 and 2
Proportion of patients with completed for the candidate primary clinical efficacy outcomes at the end of treatment period 1 and 2
Number of participants enrolled in 2 years (i.e. ability to enroll at least 10 participants in 2 years)
Ability to enroll at least 10 participants in 2 years
Mean of the 3 highest daily Modified PBAC (mPBAC) scores within each individual participant cycle averaged across 4 individual participant cycles at the end of each treatment period
Mean of the 3 highest daily Modified PBAC (mPBAC) scores within each individual participant cycle averaged across 4 individual participant cycles at the end of each treatment period
The proportion of patients who use of rescue therapy at the end of each treatment period
The proportion of patients who use of rescue therapy (i.e. more than two days of oral tranexamic acid use, additional treatment with Wilate®, additional hormonal therapy for HMB, urgent/emergent gynecological surgery for HMB, treatment with intravenous iron, red blood cell transfusion, or hospital admission for HMB) at the end of each treatment period
Mean of the mPBAC score within each individual participant cycle averaged across 4 individual participant cycles
Mean of the mPBAC score within each individual participant cycle averaged across 4 individual participant cycles
Median of the mPBAC score within each individual participant cycle used to derive the median across 4 individual participant cycles
Median of the mPBAC score within each individual participant cycle used to derive the median across 4 individual participant cycles
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