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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called Wilate for women with von Willebrand disease, a bleeding disorder, who experience heavy menstrual bleeding. The goal is to determine if Wilate effectively controls bleeding during menstruation compared to a placebo, an inactive substance. Participants will receive either Wilate or a placebo in a crossover design, allowing each participant to try both at different times to compare effects. Women with von Willebrand disease who have stable heavy menstrual bleeding on their current treatment might be suitable for this trial. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

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.

Is there any evidence suggesting that Wilate is likely to be safe for humans?

Studies have shown that Wilate is safe and effective for people with von Willebrand disease (VWD). In everyday use, both children and adults using Wilate have successfully prevented and treated bleeding without major safety issues. Research indicates that Wilate has undergone extensive testing, confirming its safety for people with VWD. While minor side effects may occur, as with any treatment, the overall evidence supports its safe use for managing bleeding episodes. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for von Willebrand disease?

Researchers are excited about Wilate for treating Von Willebrand Disease because it offers a targeted approach by using a specific concentrate of von Willebrand factor and factor VIII. Unlike standard treatments that might involve broader clotting factor concentrates, Wilate delivers a precise dose of 30-60 IU VWF:RCo/kg, tailored for the heaviest days of a patient's menstrual cycle. This specificity not only aims to improve bleeding control but also optimizes the dosing schedule, potentially enhancing patient convenience and treatment adherence.

What evidence suggests that Wilate might be an effective treatment for von Willebrand disease?

Research has shown that Wilate, which participants in this trial may receive, effectively treats von Willebrand disease (VWD). In past studies, Wilate successfully prevented and treated bleeding in VWD patients undergoing surgery. Another study found that regular use of Wilate reduced heavy menstrual bleeding in women with VWD. Additionally, Wilate reduced bleeding in both children and adults with severe VWD when used regularly as a preventive treatment. These findings suggest that Wilate can be a strong option for managing bleeding in people with VWD. ^{4 6 7 8 9}

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

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 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]

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]

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]

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]

Citations

1.

octapharma.com

octapharma.com/news/press-release/2024/publication-of-data-from-wil-31-study

Publication of data from WIL-31 study marks a milestone in ...

WIL-31 is the largest prospective study to date examining the efficacy and safety of regular prophylaxis in patients with VWD.

2.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/28026130/

Efficacy and safety of a VWF/FVIII concentrate (wilate® ) in ...

Wilate demonstrated effective prevention and treatment of bleeding in inherited VWD patients undergoing surgery, with no clinically significant safety concerns.

3.

sciencedirect.com

sciencedirect.com/science/article/pii/S2666577825000620

Efficacy of regular prophylaxis with a plasma-derived von ...

Wilate prophylaxis given 2–3 times a week without menstruation coordination was efficacious in reducing heavy menstrual bleeding in girls/women with VWD ...

4.

ashpublications.org

ashpublications.org/bloodadvances/article/8/6/1405/514639/von-Willebrand-factor-factor-VIII-concentrate

von Willebrand factor/factor VIII concentrate (Wilate ...

Prophylaxis with a VWF/FVIII concentrate (Wilate) reduced bleeding in children/adults with all types of severe VWD vs on-demand treatment.Prophylaxis with.

5.

sciencedirect.com

sciencedirect.com/science/article/pii/S2473952924000582

von Willebrand factor/factor VIII concentrate (Wilate) ...

WIL-31 showed that Wilate prophylaxis was efficacious and well-tolerated in pediatric and adult patients with VWD of all types.

6.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC8255103/

Real-World Data on the Effectiveness and Safety of wilate for ...

Wilate was safe, well tolerated, and effective for the prevention and treatment of bleeding in pediatric and adult VWD patients in a real-world setting.

7.

wilateusa.com

wilateusa.com/pi

highlights of prescribing information

Each vial of WILATE contains the labeled amount in International Units (IU) of von Willebrand factor (VWF) activity as measured with the Ristocetin cofactor.

8.

fda.gov

fda.gov/media/177085/download

January 23, 2024 Clinical Review Memo - Wilate

Safety data are pooled for all 9 Wilate studies in patients with VWD ... von Willebrand disease; VWF = von Willebrand factor. Page 45 ...

9.

journals.sagepub.com

journals.sagepub.com/doi/10.1177/10760296251327593

Safety and Efficacy of the Use of pdVWF/FVIII-C in Patients ...

This real-world evidence study confirmed the efficacy of the pdVWF/FVIII-C as on-demand and/or prophylaxis treatment in patients with bleeding episodes or ...