Wilate for Von Willebrand Disease

(EMPOWER Trial)

Overseen By St. Michael's Hospital

Age: 18+

Sex: Female

Trial Phase: Phase 3

Sponsor: Unity Health Toronto

Must be taking: Wilate

Must not be taking: Desmopressin, Hormonal contraceptives

Disqualifiers: Pregnancy, Breastfeeding, Liver disease, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

20 Participants Needed

Trial Summary

What is the purpose of this trial?

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

Will I have to stop taking my current medications?

The trial requires that you stay on your current treatment for heavy menstrual bleeding and iron deficiency anemia for at least 3 cycles before joining and throughout the study. However, you cannot take desmopressin at the same time as Wilate, unless it's for treating excessive bleeding.

What data supports the effectiveness of the drug Wilate for Von Willebrand Disease?

Research shows that Wilate is effective and safe for managing bleeding in patients with Von Willebrand Disease, with high success rates in both surgical and non-surgical settings. In various studies, Wilate was rated as 'excellent' or 'good' in over 96% of cases, and it effectively stopped bleeding episodes quickly, often within one or two days. ^{1 2 3 4 5}

Is Wilate safe for treating Von Willebrand Disease?

Wilate has been shown to be safe and well-tolerated in both adults and children with Von Willebrand Disease. Studies report no unexpected adverse reactions, no thrombotic events (blood clots), and no development of inhibitory antibodies, indicating a strong safety profile. ^{1 3 4 5 6}

How is the drug Wilate unique in treating von Willebrand disease?

Wilate is unique because it is a plasma-derived concentrate that contains von Willebrand factor and factor VIII in a natural 1:1 ratio, making it effective for both prophylactic and on-demand treatment of bleeding episodes in von Willebrand disease. It is also double virus-inactivated, ensuring a high safety profile, and has shown excellent efficacy in both adults and children, including those undergoing surgery. ^{2 3 4 5 7}

Eligibility Criteria

This trial is for adult female outpatients with von Willebrand disease (VWD) who suffer from heavy menstrual bleeding. Participants must have a modified PBAC score over 100, agree to nurse-administered infusions, and use specific feminine hygiene products provided by the sponsor. They should be on stable treatment for their condition and anemia.

Inclusion Criteria

I have been diagnosed with inherited von Willebrand disease.

You must agree to only use the feminine hygiene products provided by the study sponsor.

I am willing to receive treatments through an IV from a nurse.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Period 1

Participants receive either pdVWF:FVIII concentrate or placebo for 4 menstrual cycles

4 cycles

Washout

A washout period where no study-based treatment is delivered

1 cycle

Treatment Period 2

Participants crossover to the comparator treatment for 4 menstrual cycles

4 cycles

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Wilate

Trial Overview The EMPOWER trial tests a lyophilized concentrate of human coagulation factors against placebo in women with VWD to see if it can reduce heavy menstrual bleeding. It's a double-blind crossover study, meaning patients will receive both the treatment and placebo at different times without knowing which one they're getting.

Participant Groups

Group I: pdVWF:FVIII concentrate (Wilate®) Treatment and Standard Care Experimental Treatment 1 Intervention

Wilate® 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.

Group II: Placebo and Standard Care Placebo Group 1 Intervention

Patients randomized to the placebo arm will receive intravenous placebo (normal saline) at the same approximate volume and frequency as the study drug.

Who Is Running the Clinical Trial?

Unity Health Toronto

Lead Sponsor

Trials

572

Recruited

470,000+

Findings from Research

In a real-world study involving 111 patients with von Willebrand disease (VWD) across 31 centers in 11 countries, wilate was found to be safe and well tolerated, with 96.2% of infusions rated as 'excellent' by patients.

Wilate demonstrated high effectiveness in treating bleeding episodes and providing prophylaxis, with 100% of on-demand treatments rated as 'excellent' or 'good' by patients and investigators, and a median annualized bleeding rate of only 1.9 during prophylaxis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Real-World Data on the Effectiveness and Safety of wilate for the Treatment of von Willebrand Disease. Sholzberg, M., Khair, K., Yaish, H., et al.[2021]

Wilate®, a new plasma-derived concentrate for von Willebrand factor (VWF) and factor VIII (FVIII), demonstrated high efficacy in managing bleeding during surgery in patients with von Willebrand disease (VWD), with 96% of procedures rated as excellent or good.

The treatment was well-tolerated, with 100% of patients reporting good tolerability during both major and minor surgeries, and it allows for administration at similar FVIII doses but with lower VWF doses compared to older products.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of a new generation von Willebrand factor/factor VIII concentrate (Wilate®) in the management of perioperative haemostasis in von Willebrand disease patients undergoing surgery. Windyga, J., von Depka-Prondzinski, M.[2012]

In a study involving 15 pediatric patients under 6 years old with von Willebrand disease, treatment with Wilate demonstrated excellent efficacy, with 99.7% of prophylactic infusions and 100% of treatments for bleeding episodes or surgical procedures rated as effective.

Wilate was found to be safe, with no thromboembolic events or development of anti-VWF antibodies, and a dosage of 20-50 IU/kg was sufficient to achieve hemostasis in 97% of bleeding episodes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of Wilate in paediatric VWD patients under 6 years of age - results of a prospective multicentre clinical study including recovery information. Nowak-Göttl, U., Krümpel, A., Russo, A., et al.[2013]

Wilate, an albumin-free von Willebrand factor/factor VIII concentrate, demonstrated excellent or good efficacy in treating 1,095 bleeding episodes in 44 patients with von Willebrand disease, achieving a success rate of 96%.

The treatment was well-tolerated with rare mild to moderate adverse reactions, and prophylactic use significantly reduced bleeding frequency from an average of 4.5 to 1.4 bleeds per month, indicating its effectiveness for both treatment and prevention.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Treatment and prevention of acute bleedings in von Willebrand disease--efficacy and safety of Wilate, a new generation von Willebrand factor/factor VIII concentrate. Berntorp, E., Windyga, J.[2009]

Wilate®, a plasma-derived concentrate for treating von Willebrand disease (VWD), demonstrated excellent or good efficacy in 94% of surgical episodes and 98% of bleeding episodes among 54 patients treated over a 5-year period.

The treatment was found to be safe, with only 6 adverse events reported (0.44% of infusions), and no significant accumulation of FVIII observed, indicating a low risk of complications during prolonged use.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and efficacy of a von Willebrand factor/factor VIII concentrate (Wilate®): a single centre experience. Batty, P., Chen, YH., Bowles, L., et al.[2014]

A study involving 47 children with von Willebrand disease showed that Wilate(®), a VWF:FVIII concentrate, is effective for treating bleeds, surgical procedures, and for prophylaxis, with most bleeds responding to a single treatment.

The treatment was found to be safe, with no thromboembolic events or accumulation of FVIII or VWF, and 95% of surgical procedures reported excellent or good efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Wilate use in 47 children with von Willebrand disease: the North London paediatric haemophilia network experience. Khair, K., Batty, P., Riat, R., et al.[2014]

Switching to the pdVWF:pdFVIII concentrate (Wilate) for long-term prophylaxis in a 12.6-year-old boy with type 3 von Willebrand disease significantly reduced his bleeding rates, demonstrating its efficacy in managing this condition.

The treatment was safe and led to a marked improvement in the patient's quality of life, with annualized bleeding rates decreasing from 7.5 to 2 over two years, indicating a successful management strategy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Switch to pdVWF:pdFVIII concentrate for prophylaxis in a paediatric patient with Type 3 von Willebrand disease: a case report. Berger, C., Thouvenin, S., Montmartin, A., et al.[2023]

References

1. Germany pubmed.ncbi.nlm.nih.gov

Real-World Data on the Effectiveness and Safety of wilate for the Treatment of von Willebrand Disease. [2021]

2. Germany pubmed.ncbi.nlm.nih.gov

Efficacy and safety of a new generation von Willebrand factor/factor VIII concentrate (Wilate®) in the management of perioperative haemostasis in von Willebrand disease patients undergoing surgery. [2012]

3. England pubmed.ncbi.nlm.nih.gov

Efficacy and safety of Wilate in paediatric VWD patients under 6 years of age - results of a prospective multicentre clinical study including recovery information. [2013]

4. England pubmed.ncbi.nlm.nih.gov

Treatment and prevention of acute bleedings in von Willebrand disease--efficacy and safety of Wilate, a new generation von Willebrand factor/factor VIII concentrate. [2009]

5. England pubmed.ncbi.nlm.nih.gov

Safety and efficacy of a von Willebrand factor/factor VIII concentrate (Wilate®): a single centre experience. [2014]

6. England pubmed.ncbi.nlm.nih.gov

Wilate use in 47 children with von Willebrand disease: the North London paediatric haemophilia network experience. [2014]

7. England pubmed.ncbi.nlm.nih.gov

Switch to pdVWF:pdFVIII concentrate for prophylaxis in a paediatric patient with Type 3 von Willebrand disease: a case report. [2023]