Von Willebrand Factor for Severe Von Willebrand Disease

Not currently recruiting at 25 trial locations

Overseen By Jessica Callis

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: American Thrombosis and Hemostasis Network

Disqualifiers: Platelet-type VWD, Acquired VWD, others

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

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to understand the safety of different treatments for people with severe Von Willebrand Disease (VWD), a condition that impairs blood clotting. Researchers are evaluating the effectiveness of various Von Willebrand Factor (VWF) treatments in managing the condition during situations such as surgery or daily life. It suits individuals with severe congenital VWD who frequently require treatment due to bleeding episodes. As a Phase 3 trial, this study represents the final step before FDA approval, allowing participants to contribute to the development of potentially life-changing treatments.

Will I have to stop taking my current medications?

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 doctor.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that various treatments for Von Willebrand Factor (VWF) have been tested for safety in individuals with bleeding issues due to low levels of this protein. In one study, researchers observed about 1,900 patients with a severe form of this condition, and around 170 of them regularly used VWF to prevent bleeding.

Another study found that two specific VWF treatments, Fanhdi® and Alphanate®, were safe and effective. These treatments helped control bleeding and prevent it during surgeries. Additionally, a treatment called Vonvendi was shown to be safe for people with certain types of this condition, with some cases showing a complete stop in serious bleeding events.

These findings suggest that VWF treatments are generally well-tolerated, with studies confirming their safety and effectiveness in managing the condition. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about the various von Willebrand factor (VWF) regimens because they have the potential to offer more personalized treatment options for severe von Willebrand disease (VWD). Unlike standard treatments that often rely on fixed dosing schedules, these regimens could be tailored to individual patient needs, leading to more effective management of bleeding episodes. Additionally, by studying natural history, researchers aim to better understand the disease's progression and improve treatment strategies over time.

What evidence suggests that these VWF regimens are effective for Von Willebrand Disease?

Research has shown that various treatments for von Willebrand factor (VWF) effectively manage bleeding in individuals with von Willebrand disease (VWD). Studies have found that treatments such as Fanhdi® and Alphanate® effectively stop bleeding during surgeries and in everyday situations. One study showed that a lab-made version of VWF (rVWF) reduced the yearly bleeding rate by 53%. Real-world evidence also supports the effectiveness of rVWF for all types of VWD. These findings suggest that VWF treatments can be a strong option for managing severe VWD symptoms. ^{1 2 3 6 7}

Who Is on the Research Team?

Angela Weyand, MD

Principal Investigator

University of Michigan Hemophilia and Coagulation Disorders

Robert Sidonio, MD

Principal Investigator

Emory University / Children's Healthcare of Atlanta

Are You a Good Fit for This Trial?

Inclusion Criteria

Participants with severe Von Willebrand Disease with Type 3 VWD or VWF:RCo, VWF:GPlbM or VWF:Ag ≤30% of pooled normal control plasma on more than one occasion;

Participants with clinically severe VWD as defined by VWF:RCo, VWF:GPlbM or VWF:Ag ≤40% of normal with severe bleeding phenotype defined as requiring recurrent use of factor concentrates;

Co-enrollment in the ATHNdataset.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Observation

Participants are observed for safety and effectiveness of VWF regimens over a 2-year period

2 years

Data Enrichment and Analysis

Data from participants is enriched and analyzed using standardized diagnostic battery and genetic sequence analysis

3 years

Follow-up

Participants are monitored for safety and effectiveness after the main observation period

1 year

What Are the Treatments Tested in This Trial?

Interventions

• Various VWF Regimens

Who Is Running the Clinical Trial?

American Thrombosis and Hemostasis Network

Lead Sponsor

Trials

11

Recruited

204,000+

Takeda

Industry Sponsor

Trials

1,255

Recruited

4,219,000+

Dr. Naoyoshi Hirota

Takeda

Chief Medical Officer since 2020

MD from University of Tokyo

Christophe Weber

Takeda

Chief Executive Officer since 2015

PhD in Molecular Biology from Université de Montpellier

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11403693/

Real-World Efficacy and Safety of Plasma-Derived Von ... This study confirmed that Fanhdi® and Alphanate® were effective and safe in the management of bleeding episodes, the prevention of bleeding during surgeries ...

2. ashpublications.org ashpublications.org/blood/article/134/Supplement_1/1132/426621/Assessing-the-Safety-of-Various-VWF-Regimens-in

Assessing the Safety of Various VWF Regimens in Patients ... A deficiency of von Willebrand factor (VWF) can be quantitative (type 1 or type 3) or qualitative (type 2) and lead to a bleeding diathesis ...

3. hematologyadvisor.com hematologyadvisor.com/news/recombinant-von-willebrand-factor-prophylaxis-improves-outcomes-treatment/

Recombinant VWF Prophylaxis Improves Outcomes in ... These data suggest that rVWF prophylaxis may result in improved outcomes. The annualized bleeding rate was found to have reduced by 53% among ...

4. tandfonline.com tandfonline.com/doi/full/10.1080/17474086.2025.2504956

Real-world treatment patterns and outcomes in people with ... This retrospective chart review evaluated real-world effectiveness of prophylaxis with recombinant von Willebrand factor (rVWF) in all subtypes of VWD. Research ...

5. sciencedirect.com sciencedirect.com/science/article/pii/S0006497118590492

Assessing the Safety of Various VWF Regimens in Patients ... A deficiency of von Willebrand factor (VWF) can be quantitative (type 1 or type 3) or qualitative (type 2) and lead to a bleeding diathesis ...

6. fda.gov fda.gov/media/188621/download?attachment

September 5, 2025 Clinical Review Memo - VONVENDI Study 071301 demonstrated that Vonvendi is safe and effective in patients with Type 1 and Type 2 VWD with 100% decrease in sABR in prior OD ...

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12499246/

Real-World Data from an EU Post-Authorization Safety Study Medical record data were abstracted for a minimum of 7 days and a maximum of 6 months after the index date, and similarly, after each subsequent ...