Wilate for Von Willebrand Disease
(EMPOWER Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a treatment called Wilate for women with von Willebrand disease, a bleeding disorder, who experience heavy menstrual bleeding. The goal is to determine if Wilate effectively controls bleeding during menstruation compared to a placebo, an inactive substance. Participants will receive either Wilate or a placebo in a crossover design, allowing each participant to try both at different times to compare effects. Women with von Willebrand disease who have stable heavy menstrual bleeding on their current treatment might be suitable for this trial. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.
Will I have to stop taking my current medications?
The trial requires that you stay on your current treatment for heavy menstrual bleeding and iron deficiency anemia for at least 3 cycles before joining and throughout the study. However, you cannot take desmopressin at the same time as Wilate, unless it's for treating excessive bleeding.
Is there any evidence suggesting that Wilate is likely to be safe for humans?
Studies have shown that Wilate is safe and effective for people with von Willebrand disease (VWD). In everyday use, both children and adults using Wilate have successfully prevented and treated bleeding without major safety issues. Research indicates that Wilate has undergone extensive testing, confirming its safety for people with VWD. While minor side effects may occur, as with any treatment, the overall evidence supports its safe use for managing bleeding episodes.12345
Why do researchers think this study treatment might be promising for von Willebrand disease?
Researchers are excited about Wilate for treating Von Willebrand Disease because it offers a targeted approach by using a specific concentrate of von Willebrand factor and factor VIII. Unlike standard treatments that might involve broader clotting factor concentrates, Wilate delivers a precise dose of 30-60 IU VWF:RCo/kg, tailored for the heaviest days of a patient's menstrual cycle. This specificity not only aims to improve bleeding control but also optimizes the dosing schedule, potentially enhancing patient convenience and treatment adherence.
What evidence suggests that Wilate might be an effective treatment for von Willebrand disease?
Research has shown that Wilate, which participants in this trial may receive, effectively treats von Willebrand disease (VWD). In past studies, Wilate successfully prevented and treated bleeding in VWD patients undergoing surgery. Another study found that regular use of Wilate reduced heavy menstrual bleeding in women with VWD. Additionally, Wilate reduced bleeding in both children and adults with severe VWD when used regularly as a preventive treatment. These findings suggest that Wilate can be a strong option for managing bleeding in people with VWD.46789
Are You a Good Fit for This Trial?
This trial is for adult female outpatients with von Willebrand disease (VWD) who suffer from heavy menstrual bleeding. Participants must have a modified PBAC score over 100, agree to nurse-administered infusions, and use specific feminine hygiene products provided by the sponsor. They should be on stable treatment for their condition and anemia.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment Period 1
Participants receive either pdVWF:FVIII concentrate or placebo for 4 menstrual cycles
Washout
A washout period where no study-based treatment is delivered
Treatment Period 2
Participants crossover to the comparator treatment for 4 menstrual cycles
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Wilate
Find a Clinic Near You
Who Is Running the Clinical Trial?
Unity Health Toronto
Lead Sponsor