Prophylactic treatment for heavy menstrual bleeding in women with von Willebrand disease
A Multi-cEnter, Pilot, Crossover Trial of Prophylactic Wilate CoMpared to PlacebO for Heavy Menstrual Bleeding in Patients with Von WillEbRand Disease
This study tests if a new treatment can help women with von Willebrand disease who have heavy menstrual bleeding feel better during their periods.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 18 Years and up |
| Sex | Female |
| Sponsor | Unity Health Toronto Academic / other |
| Locations | 1 site (Toronto, Ontario) |
| Trial ID | NCT06205095 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates are female patients over 18 years old with a diagnosis of inherited von Willebrand disease and a modified PBAC score greater than 100.
Not a fit: Patients with other known bleeding disorders or those not experiencing heavy menstrual bleeding may not benefit from this study.
Why it matters
Potential benefit: If successful, this trial could provide an effective prophylactic treatment option for women with VWD suffering from heavy menstrual bleeding.
How similar studies have performed: While this approach is novel in the context of VWD and heavy menstrual bleeding, similar studies have shown promise in other bleeding disorders.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Toronto, Ontario
- St. Michael's Hospital — Toronto, Ontario, Canada (Recruiting)
Study contacts
- Study coordinator: St. Michael's Hospital
- Email: empower@unityhealth.to
- Phone: 4168646060
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.