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

Launched by UNITY HEALTH TORONTO · Jan 3, 2024

Keywords

ClinConnect Summary

The EMPOWER trial is a research study looking at the use of a treatment called Wilate® for women who have von Willebrand disease (VWD) and experience heavy menstrual bleeding (HMB). This trial will compare Wilate® to a placebo (a substance with no active treatment) to see if it helps reduce heavy bleeding. It is designed for women over the age of 18 who have been diagnosed with VWD and have a history of heavy menstrual bleeding, as determined by their doctor. To be eligible, participants must be stable in their current treatment for HMB and iron deficiency anemia for at least three cycles before joining the study.

If you decide to participate, you will receive infusions of either Wilate® or the placebo, and you will be closely monitored throughout the trial, which lasts for two years. Participants will also need to use specific feminine hygiene products provided by the study. This trial is currently recruiting participants and aims to gather important information that could help develop better treatments for women with VWD and heavy menstrual bleeding in the future.

Gender

FEMALE

Eligibility criteria

• Inclusion Criteria:
• • 1. Patient capable of providing informed consent;
• • 2. 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;
• • 3. Modified PBAC score \> 100 at screening;
• • 4. Patients with a diagnosis of inherited von Willebrand disease (any type);
• • 5. 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;
• • 6. Patients willing to have an infusion administered by a nurse over the course of the study period;
• • 7. Patients who agree to use only the feminine hygiene products supplied by the sponsor.
• Exclusion Criteria:
• • 1. Diagnosed with any other known bleeding disorder;
• • 2. Pregnancy or plans to become pregnant within the duration of the study;
• • 3. Breastfeeding or plans to breastfeed within the duration of the study;
• • 4. Known hypersensitivity reactions to human plasma-derived products or any ingredient in the formulation;
• • 5. Known antibodies to VWF or FVIII;
• • 6. Severe liver disease;
• • 7. Anticipated initiation of the following: oral, transdermal, injectable, and vaginal ring hormonal contraceptives; GnRH analogues; or a hormonal intrauterine device (IUD) within the study period;
• • 8. Anticipated elective procedure that is expected to require intensive treatment with VWF or FVIII for \>10 days during the study period;
• • 9. Patients with \>2 risk factors for VTE (risk factors are determined at discretion of treating physician) or recent history of thrombosis (i.e. within the last year).
• • 10. Patient concurrently receiving desmopressin (desmopressin cannot be taken concurrently with Wilate®, except for in the context of escalation treatment for excessive bleeding).
• • 11. Anticipated initiation of any new therapies for the treatment of heavy menstrual bleeding 3 weeks prior to enrollment