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RECRUITING

NCT06205095

PHASE3

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

Sponsor: Unity Health Toronto

View on ClinicalTrials.gov

Summary

The EMPOWER trial is a pilot multi-center, placebo-controlled (normal saline), double-blind (patient and outcome assessor), crossover, 2-year randomized trial in female outpatients with von Willebrand disease (VWD) and heavy menstrual bleeding to determine trial feasibility and viability, and to explore assay sensitivity of the proposed efficacy clinical outcomes for a definitive randomized controlled trial

Official title: A Multi-cEnter, Pilot, Crossover Trial of Prophylactic Wilate CoMpared to PlacebO for Heavy Menstrual Bleeding in Patients with Von WillEbRand Disease

Key Details

Gender

FEMALE

Age Range

18 Years - Any

Study Type

INTERVENTIONAL

Enrollment

Start Date

2024-10-21

Completion Date

2026-09

Last Updated

2024-12-24

Healthy Volunteers

Conditions

Von Willebrand Diseases

Interventions

DRUG

Lyophilized concentrate of human coagulation von Willebrand Factor and factor VIII

Wilate® is a plasma-derived, highly purified concentrate administered through intravenous injection. Wilate® contains an average VWF ristocetin cofactor activity to FVIII activity at ratio of 1:1.

OTHER

Placebo

Patients randomized to the placebo arm will receive intravenous normal saline at the same approximate volume and frequency of Wilate ®.

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

Locations (1)

St. Michael's Hospital

Toronto, Ontario, Canada