Prophylactic treatment for heavy menstrual bleeding in women with von Willebrand disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

The EMPOWER trial is a pilot, multi-center, placebo-controlled, double-blind crossover trial designed to evaluate the feasibility and viability of using prophylactic Wilate in female patients with von Willebrand disease (VWD) experiencing heavy menstrual bleeding. Over a two-year period, participants will receive either the plasma-derived von Willebrand factor:Factor VIII concentrate or a placebo during their menstrual cycles, followed by a washout period. The study aims to assess the sensitivity of clinical outcomes to inform a future definitive randomized controlled trial.

Why it matters

Who can join: the full rules

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

Where it's running

• Kingston Health Sciences Centre
  Kingston, Ontario, Canada Recruiting
• St. Michael's Hospital
  Toronto, Ontario, Canada Recruiting

Study contacts

• Study coordinator: St. Michael's Hospital
• Email: empower@unityhealth.to
• Phone: 4168646060

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Managing von Willebrand Disease in Pregnant Women

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.