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Von Willebrand DiseasesPhase 3EMPOWERNCT06205095

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

A Phase 3 study testing Lyophilized concentrate of human coagulation von Willebrand Factor and factor VIII in people with Von Willebrand Diseases, run by Unity Health Toronto at 2 sites in Canada.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial 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… Read more about this study

Who is it for?
Ages 18 years and older, women only
Conditions the study is for
Von Willebrand Diseases
What is being tested?
Lyophilized concentrate of human coagulation von Willebrand Factor and factor VIII
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
20 participants
Where
2 sitesCanada
Study dates
21 Oct 2024to Sep 2028
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • Women only
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Patient capable of providing informed consent;
  • 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;
  • Modified PBAC score > 100 at screening;
  • Patients with a diagnosis of inherited von Willebrand disease (any type);
  • 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;
  • Patients willing to have an infusion administered by a nurse over the course of the study period;
  • Patients who agree to use only the feminine hygiene products supplied by the sponsor.

Who cannot join

  • Diagnosed with any other known bleeding disorder;
  • Pregnancy or plans to become pregnant within the duration of the study;
  • Breastfeeding or plans to breastfeed within the duration of the study;
  • Known hypersensitivity reactions to human plasma-derived products or any ingredient in the formulation;
  • Known antibodies to VWF or FVIII;
  • Severe liver disease;
Show all 11Show fewer
  • 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;
  • Anticipated elective procedure that is expected to require intensive treatment with VWF or FVIII for >10 days during the study period;
  • 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).
  • Patient concurrently receiving desmopressin (desmopressin cannot be taken concurrently with Wilate®, except for in the context of escalation treatment for excessive bleeding).
  • Anticipated initiation of any new therapies for the treatment of heavy menstrual bleeding 3 weeks prior to enrollment

Trial locations and contacts

2 sites in 1 country. Contact the study team or a site near you to ask about taking part.

St. Michael's Hospital contact

4168646060

empower@unityhealth.to

Canada2 sites, 2 recruiting
  • Kingston Health Sciences Centre

    Kingston, Ontario

    Julie Grabell, +1 613 533 6000 ext. 75223, empower@unityhealth.to

    Recruiting
  • St. Michael's Hospital

    Toronto, Ontario, M5B1W8

    Michelle Sholzberg, 4168646060, empower@unityhealth.to

    Recruiting

Other trials in Canada

About this study

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

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

Read the detailed description

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 explore assay sensitivity of the proposed efficacy clinical outcomes for a definitive randomized controlled trial.

For the first treatment period, patients will be randomized to receive either plasma derived von Willebrand factor:Factor VIII (pdVWF:FVIII) concentrate (plus standard of care) or placebo (plus standard of care) for VWD-associated heavy menstrual bleeding for 4 cycles, crossing over to the comparator treatment during the second treatment period. The first treatment period will be followed by a 1 cycle washout period when no study-based treatment will be delivered.

The main purpose of the pilot will be to evaluate viability and feasibility of the trial design, as well as to explore assay sensitivity to inform determination of the primary efficacy outcome for the definitive randomized trial which will evaluate the effect of prophylaxis with pdVWF:FVIII concentrate compared with placebo on HMB in women with VWD. A secondary objective is to conduct a preliminary assessment of the effect on clinical outcomes of 2-3 doses of prophylaxis with pdVWF:FVIII concentrate when provided on the first 4 days of menstruation compared with placebo.

Being tested
Lyophilized concentrate of human coagulation von Willebrand Factor and factor VIII, Placebo
Medical subject headings
von Willebrand Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Lyophilized concentrate of human coagulation von Willebrand Factor and factor VIIIDrug

    Also called Wilate

    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.

  • PlaceboOther

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

Study groups in NCT06205095
Study groupRoleWhat they receive
pdVWF:FVIII concentrate (Wilate®) Treatment and Standard CareExperimentalWilate® at a dose of 30-60 IU VWF:RCo/kg for the two anticipated heaviest days of bleeding every 24-48 hours within the first 4 days of menstruation will be provided. Additional two optional doses 24-48 hours from the last can be provided. A minimum of 2 doses must be provided.
Receives: Drug: Lyophilized concentrate of human coagulation von Willebrand Factor and factor VIII
Placebo and Standard CarePlacebo comparatorPatients randomized to the placebo arm will receive intravenous placebo (normal saline) at the same approximate volume and frequency as the study drug.
Receives: Other: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Blinding Index (BI) score at the end of cycle 4 of treatment period 1 and 2

    Blinding Index (BI) score at the end of cycle 4 of treatment period 1 and 2

    Measured: 2 years

  2. Proportion of participant drop-out at the end of treatment period 1 and 2

    Proportion of participant drop-out at the end of treatment period 1 and 2

    Measured: 2 years

  3. Proportion of participants with completed for the candidate primary clinical efficacy outcomes at the end of treatment period 1 and 2

    Proportion of patients with completed for the candidate primary clinical efficacy outcomes at the end of treatment period 1 and 2

    Measured: 2 years

  4. Number of participants enrolled in 2 years (i.e. ability to enroll at least 10 participants in 2 years)

    Ability to enroll at least 10 participants in 2 years

    Measured: 2 years

  5. Proportion of participants with carryover effect for the candidate primary clinical efficacy outcomes from period 1 to period 2

    Proportion of participants with carryover effect for the candidate primary clinical efficacy outcomes from period 1 to period 2

    Measured: 2 years

Secondary outcomes (22)
  1. Mean of the 3 highest daily Modified PBAC (mPBAC) scores within each individual participant cycle averaged across 4 individual participant cycles at the end of each treatment period

    Mean of the 3 highest daily Modified PBAC (mPBAC) scores within each individual participant cycle averaged across 4 individual participant cycles at the end of each treatment period

    Measured: At the end of 8 menstrual cycles (approximately 10 days)

  2. The proportion of patients who use of rescue therapy at the end of each treatment period

    The proportion of patients who use of rescue therapy (i.e. more than two days of oral tranexamic acid use, additional treatment with Wilate®, additional hormonal therapy for HMB, urgent/emergent gynecological surgery for HMB, treatment with intravenous iron, red blood cell transfusion, or hospital admission for HMB) at the end of each treatment period

    Measured: At the end of 8 menstrual cycles (approximately 10 days)

  3. Mean of the mPBAC score within each individual participant cycle averaged across 4 individual participant cycles

    Mean of the mPBAC score within each individual participant cycle averaged across 4 individual participant cycles

    Measured: At the end of 8 menstrual cycles (approximately 10 days)

  4. Median of the mPBAC score within each individual participant cycle used to derive the median across 4 individual participant cycles

    Median of the mPBAC score within each individual participant cycle used to derive the median across 4 individual participant cycles

    Measured: At the end of 8 menstrual cycles (approximately 10 days)

  5. Number of days of oral tranexamic acid use

    Number of days of oral tranexamic acid use

    Measured: At the end of 8 menstrual cycles (approximately 10 days)

  6. Number of days of Wilate® treatment received

    Number of days of Wilate® treatment received

    Measured: At the end of 8 menstrual cycles (approximately 10 days)

  7. Duration of menstruation (measured in days)

    Duration of menstruation (measured in days)

    Measured: At the end of 8 menstrual cycles (approximately 10 days)

  8. Major bleed according to the International Society on Thrombosis and Haemostasis (ISTH) definition

    Major bleed according to the International Society on Thrombosis and Haemostasis (ISTH) definition

    Measured: At the end of 8 menstrual cycles (approximately 10 days)

  9. Clinically relevant non-major bleed

    Clinically relevant non-major bleed

    Measured: At the end of 8 menstrual cycles (approximately 10 days)

  10. Hemoglobin levels (g/L)

    Hemoglobin levels (g/L)

    Measured: At the end of 8 menstrual cycles (approximately 10 days)

  11. Ferritin levels (mcg/L)

    Ferritin levels (mcg/L)

    Measured: At the end of 8 menstrual cycles (approximately 10 days)

  12. Use of additional hormonal therapy for heavy menstrual bleeding

    Use of additional hormonal therapy for heavy menstrual bleeding

    Measured: At the end of 8 menstrual cycles (approximately 10 days)

  13. Requirement of urgent/emergent gynecological surgery for heavy menstrual bleeding

    Requirement of urgent/emergent gynecological surgery for heavy menstrual bleeding

    Measured: At the end of 8 menstrual cycles (approximately 10 days)

  14. Fatigue scores (as measured by the FACIT fatigue scale)

    Fatigue scores (as measured by the FACIT fatigue scale)

    Measured: At the end of 8 menstrual cycles (approximately 10 days)

  15. Short Form-12 Scores

    Short Form-12 Scores

    Measured: At the end of 8 menstrual cycles (approximately 10 days)

  16. Scores on the individual components of the Short Form-12

    Scores on the individual components of the Short Form-12

    Measured: At the end of 8 menstrual cycles (approximately 10 days)

  17. Requirement of hemostatic care (tranexamic acid, Wilate®, platelet transfusion)

    Requirement of hemostatic care (tranexamic acid, Wilate®, platelet transfusion)

    Measured: At the end of 8 menstrual cycles (approximately 10 days)

  18. Requirement of anemia focused care (intravenous iron and/or red blood cell transfusion)

    Requirement of anemia focused care (intravenous iron and/or red blood cell transfusion)

    Measured: At the end of 8 menstrual cycles (approximately 10 days)

  19. Number of hypersensitivity infusion reactions

    Number of hypersensitivity infusion reactions

    Measured: At the end of 8 menstrual cycles (approximately 10 days)

  20. Number of thromboembolic events (defined as symptomatic or incidental, suspected or confirmed via diagnostic imaging and/or electrocardiogram where appropriate);

    Number of thromboembolic events (defined as symptomatic or incidental, suspected or confirmed via diagnostic imaging and/or electrocardiogram where appropriate);

    Measured: At the end of 8 menstrual cycles (approximately 10 days)

  21. Proportion of participants who development of VWF inhibitors

    Proportion of participants who development of VWF inhibitors

    Measured: At the end of 8 menstrual cycles (approximately 10 days)

  22. Number of adverse events

    Number of adverse events

    Measured: 8 cycles

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Jan 2024
  2. Study start21 Oct 2024
  3. Primary completionDec 2027 (estimated)
  4. Study completionSep 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Crossover
Masking
Triple blind
Who is masked
Participant, Care provider, Investigator
Enrolment
20 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06205095
Sponsor's protocol ID
2.5
Acronym
EMPOWER
Status last verified
Oct 2026
First submitted
18 Dec 2023
Last updated
14 Oct 2026
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Lyophilized concentrate of human coagulation von Willebrand Factor and factor VIII in people with Von Willebrand Diseases, run by Unity Health Toronto at 2 sites in Canada.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 14 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact St. Michael's Hospital (4168646060, empower@unityhealth.to), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Blinding Index (BI) score at the end of cycle 4 of treatment period 1 and 2 (time frame: 2 years). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Oct 2026.

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