Actively Recruiting
A Multi-center Pilot Crossover Trial of Prophylactic Wilate Compared to Placebo for Heavy Menstrual Bleeding in Patients with Von Willebrand Disease
Led by Unity Health Toronto · Updated on 2024-12-24
20
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are conducting the EMPOWER trial, a pilot multi-center, placebo-controlled, double-blind, crossover randomized trial lasting two years. It focuses on female outpatients with von Willebrand disease (VWD) who experience heavy menstrual bleeding (HMB). The study aims to assess whether this trial design is feasible and viable, and to explore the sensitivity of clinical outcomes to guide a future definitive trial. Participants will be randomly assigned to receive either a plasma-derived von Willebrand factor:Factor VIII concentrate called Wilate4 or a placebo (normal saline), both given with standard care. Treatment is provided over four menstrual cycles during the first period, followed by a one-cycle washout without study treatment. Then, participants switch to the other treatment in the second period. Wilate4 is administered intravenously at doses of 30-60 IU VWF:RCo/kg on the two heaviest bleeding days within the first four days of menstruation, with optional additional doses. During the study, participants receive infusions by a nurse and use specific feminine hygiene products supplied by the sponsor. Researchers will evaluate trial feasibility by measuring participant retention, blinding success, and data completion. They will also assess menstrual bleeding severity using a modified pictorial blood assessment chart (mPBAC) and monitor clinical outcomes like bleeding events, hemoglobin and ferritin levels, fatigue, and adverse reactions. The total study participation is two years, including both treatment periods and washout.
CONDITIONS
Brief Title
A Pilot Crossover Trial of Prophylactic Wilate Compared to Placebo for Heavy Menstrual Bleeding in Patients with VWD
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patient capable of providing informed consent
- Female patients over 18 years old with heavy menstrual bleeding
- Modified PBAC score greater than 100 at screening
- Diagnosis of inherited von Willebrand disease (any type)
- Stable treatment for heavy menstrual bleeding and iron deficiency anemia for 3 cycles prior to study entry and expected to remain stable
- Willingness to have infusions administered by a nurse during the study
- Agreement to use only feminine hygiene products supplied by the sponsor
You will not qualify if you...
- Diagnosis of any other known bleeding disorder
- Pregnancy or plans to become pregnant during the study
- Breastfeeding or plans to breastfeed during the study
- Known hypersensitivity to human plasma-derived products or ingredients
- Known antibodies to von Willebrand factor or factor VIII
- Severe liver disease
- Planned initiation of hormonal contraceptives, GnRH analogues, or hormonal IUD during the study
- Planned elective procedure requiring intensive VWF or FVIII treatment for more than 10 days
- More than 2 risk factors for venous thromboembolism or recent thrombosis within the past year
- Concurrent use of desmopressin except for bleeding escalation
- Planned start of new therapies for heavy menstrual bleeding within 3 weeks before enrollment or during the study period
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 4 menstrual cycles
Participants receive either Wilate® (pdVWF:FVIII concentrate) plus standard care or placebo plus standard care during the first 4 menstrual cycles. Wilate® is administered intravenously on the first 4 days of menstruation with 2 to 3 doses per cycle.
4 treatment cycles with dosing during menstruation
Duration - 1 menstrual cycle
Participants do not receive any study-based treatment for 1 menstrual cycle to clear any effects from the first treatment period.
1 cycle without treatment
Duration - 4 menstrual cycles
Participants cross over to receive the alternate treatment (Wilate® or placebo) plus standard care for the next 4 menstrual cycles with similar dosing as in Treatment Period 1.
4 treatment cycles with dosing during menstruation
Trial Site Locations
Total: 1 location
1
St. Michael's Hospital
Toronto, Ontario, Canada, M5B1W8
Actively Recruiting
Research Team
S
St. Michael's Hospital
How is the study designed?
Study Type
INTERVENTIONAL
Masking
TRIPLE
Allocation
RANDOMIZED
Model
CROSSOVER
Primary Purpose
TREATMENT
Number of Arms
2
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