VWF Replacement Therapy for Von Willebrand Disease

(VIP Trial)

Recruiting at 11 trial locations

Overseen By Central Study Contact

Age: 18+

Sex: Female

Trial Phase: Academic

Sponsor: University of Washington

Must be taking: VWF replacement

Disqualifiers: Liver disease, Renal disease, Preeclampsia, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 5-10 days postpartum

110 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the best management of delivery for pregnant women with von Willebrand disease, a bleeding disorder, by using Wilate. This treatment helps maintain certain blood factor levels to reduce bleeding during and after childbirth. The main goal is to assess how effectively this approach prevents primary postpartum hemorrhage, or heavy bleeding after giving birth. Women with von Willebrand disease who struggle to maintain specific blood factor levels in late pregnancy might be suitable candidates for this study. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important medical advancements.

Do I need to stop my current medications for this trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your healthcare provider.

Is there any evidence suggesting that this trial's treatment is likely to be safe?

Research has shown that Wilate is safe and effective for treating bleeding in people with von Willebrand disease (VWD). In studies, both children and adults with VWD tolerated Wilate well. Importantly, no reports of blood clots or harmful antibodies, which are proteins that might attack the body’s own cells, were found. This suggests that Wilate generally does not cause serious side effects. The FDA reviewed the treatment and found no major safety concerns. Overall, evidence indicates that Wilate is a reliable option for managing bleeding in people with VWD. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Wilate is unique because it is a plasma-derived treatment specifically designed for von Willebrand disease (VWD), which aims to provide a more tailored approach to managing the condition during pregnancy. Unlike other treatments that may not offer the same precision, Wilate contains both von Willebrand factor and Factor VIII, crucial components for blood clotting, potentially offering better control over bleeding episodes. Researchers are excited because Wilate's dual-action mechanism could offer more effective and consistent management of VWD in pregnant women, potentially reducing complications for both the mother and the baby.

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

Research has shown that Wilate effectively treats von Willebrand disease (VWD). Studies indicate that regular use of Wilate reduced bleeding episodes by 87% compared to treating them as they occurred. Approximately 84% of bleeding treatments with Wilate succeeded across all ages, with an 88% success rate in children and a 93% success rate overall. Wilate also reduces heavy menstrual bleeding in girls and women with VWD. It has proven safe and well-tolerated in both children and adults.

In this trial, participants will be categorized into two groups: "Correctors" and "Non-Correctors," based on their von Willebrand factor parameter levels. ^{1 3 6 7 8}

Who Is on the Research Team?

Jill M Johnsen, M.D.

Principal Investigator

University of Washington

Barbara A Konkle, M.D.

Principal Investigator

Washington Center for Bleeding Disorders

Peter A Kouides, M.D.

Principal Investigator

Mary M. Gooley Hemophilia Center

Are You a Good Fit for This Trial?

Inclusion Criteria

von Willebrand Disease (VWD) patients defined prepartum as Type 1 per National Heart, Lung, and Blood Institute (NHLBI) criterion of von Willebrand Factor (VWF) level less than 30 percent, or Type 2, or Type 3

Patients with gestational week 34-38 VWF:Ag, VWF:Act (or VWF:RCo), or FVIII:Act less than 100 percent will be enrolled in the non-corrector group. In patients with an isolated VWF:CB type 2 defect, VWF:CB less than 100 percent can also be determined as a non-corrector Patients with VWF parameter levels greater than or equal to 100 percent self-corrected at gestational weeks 34-38 will be enrolled in the corrector group

Written informed consent from the patient prepartum, before gestational week 39

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Wilate VWF replacement therapy to maintain VWF levels of 100-150% for delivery and immediate postpartum period, followed by 50-100% for 5-10 days postpartum

5-10 days postpartum

In-person visits for delivery and postpartum monitoring

Postpartum Monitoring

Tranexamic acid administration for 14 days postpartum and monitoring for primary and secondary postpartum hemorrhage

14 days

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessment of thrombus occurrence and infusion-related reactions

up to 42 days postpartum

What Are the Treatments Tested in This Trial?

Interventions

• Wilate

How Is the Trial Designed?

Group I: Non-Corrector Experimental Treatment 3 Interventions

Patients with gestational week 34-38 von Willebrand factor activity, or von Willebrand factor ristocetin cofactor, or Factor VIII procoagulant activity less than 100 percent will be termed non-correctors. When laboratory monitoring can be performed, patients with an isolated von Willebrand factor collagen binding type 2 defect, von Willebrand factor collagen binding less than 100 percent can also be enrolled and determined as a non-corrector.

Group II: Corrector Experimental Treatment 2 Interventions

Patients with von Willebrand factor parameter levels greater than or equal to 100 percent self-corrected at gestational weeks 34-38 will be termed correctors.

Who Is Running the Clinical Trial?

University of Washington

Lead Sponsor

Trials

1,858

Recruited

2,023,000+

Bloodworks

Lead Sponsor

Trials

12

Recruited

630+

Mary M. Gooley Hemophilia Center

Collaborator

Trials

2

Recruited

120+

Octapharma

Industry Sponsor

Trials

86

Recruited

11,300+

Wolfgang Marguerre

Octapharma

Chief Executive Officer since 1983

MBA from INSEAD

Wolfgang Frenzel

Octapharma

Chief Medical Officer since 2010

MD from University of Vienna

Ergomed

Industry Sponsor

Trials

11

Recruited

3,300+

Citations

1. 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 ...

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

Real-World Data on the Effectiveness and Safety of wilate for ... Conclusion wilate was safe, well tolerated, and effective for the prevention and treatment of bleeding in pediatric and adult VWD patients in a ...

3. 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 ... Mean spontaneous ABR decreased by 87% during wilate® prophylaxis compared with on-demand treatment. Reductions in total and spontaneous bleeding ...

4. wilateusa.com wilateusa.com/vwd/learn-about-wilate/clinical-evidence-safety/

wilate® Clinical Evidence and Safety for VWD Treatment For all patients studied, 84% of bleeding treatments were successful · For pediatric patients studied, 88% of bleeding treatments were successful · 93% of the ...

5. fda.gov fda.gov/media/177085/download

January 23, 2024 Clinical Review Memo - Wilate This study investigated the efficacy and safety of Wilate in previously treated patients with Type 3, Type 2 (except 2N), or severe Type 1 VWD ≥ ...

6. wilateusa.com wilateusa.com/vwd/safety-tolerability/prophylaxis/

Wilate® Prophylaxis Safety for von Willebrand Disease Learn how Wilate® reduces bleeding in VWD patients. See safety data from clinical trials supporting prophylaxis in children and adults.

7. wilate.com wilate.com/m-en/tolerability/

Safety and tolerability of vwd treatment - wilate Treatment with von Willebrand factor concentrates is generally well tolerated. No thromboembolic events or inhibitory antibodies haven been reported.

8. fda.gov fda.gov/media/161029/download

2022 Wilate PAC Review Memo This memorandum documents FDA's complete evaluation, including review of adverse event reports in passive surveillance data, periodic safety reports from the.