rVWF for Von Willebrand Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores a new treatment approach for people with severe von Willebrand Disease, a genetic bleeding disorder. The goal is to test the effectiveness of a medication called rVWF (vonicog alfa) in reducing spontaneous bleeding episodes over a year. Participants will receive this treatment for up to three years, with two groups trying different dosing strategies. This trial suits those managing their condition with von Willebrand factor therapy and who have experienced at least three spontaneous bleeds needing treatment in the past year. 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.
Do I have to stop taking my current medications for the rVWF trial?
The trial information does not specify if you need to stop taking your current medications. However, if you are taking immunomodulatory drugs (medications that modify the immune system), you must stop them at least 30 days before joining the study.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that rVWF (recombinant von Willebrand factor) is generally safe and well-tolerated. In past studies, no new safety issues emerged when rVWF was used to prevent or treat bleeding in individuals with von Willebrand Disease. Another study found that rVWF had a safety profile similar to other treatments for this condition.
Some side effects have been observed, but they are usually mild and not serious. Importantly, no major safety concerns have been reported, which is reassuring for those considering participation in a clinical trial.12345Why are researchers excited about this trial's treatments?
Researchers are excited about rVWF for treating Von Willebrand Disease because it offers a targeted approach by using recombinant von Willebrand factor, which can be administered either as a prophylactic or on-demand treatment. Unlike traditional therapies that often involve plasma-derived factors, rVWF is a synthetic option, reducing the risk of blood-borne infections. Additionally, the on-demand option provides flexibility, as it can be used with or without ADVATE to address acute bleeding episodes, offering a potentially more personalized therapy. This new approach has the potential to improve safety and effectiveness over existing treatments.
What evidence suggests that this trial's treatments could be effective for Von Willebrand Disease?
Research has shown that recombinant von Willebrand factor (rVWF) can help people with von Willebrand Disease. In this trial, participants in the Prophylaxis arm will receive rVWF regularly. Studies have found that this can lead to fewer bleeding episodes each year, with some cases showing a reduction of about 53%. Real-world evidence also supports rVWF's effectiveness in managing bleeding for various types of this condition. This treatment reduces the frequency of unexpected bleeding, making it a promising option for those with severe von Willebrand Disease.678910
Who Is on the Research Team?
Study Director
Principal Investigator
Takeda
Are You a Good Fit for This Trial?
This trial is for pediatric and adult patients with severe Von Willebrand Disease (VWD), specifically types 1, 2A, 2B, 2M, or type 3. Participants must have a history of bleeding episodes requiring treatment and be willing to follow the study protocol. Women who can bear children must test negative for pregnancy and use effective birth control during the study.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive rVWF (vonicog alfa) prophylaxis to assess effectiveness based on annualized bleeding rate (ABR) during the first 12 months
Extended Treatment
Participants continue to receive rVWF treatment for a maximum of 3 years to monitor long-term safety and efficacy
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- rFVIII
- rVWF
Trial Overview
The trial tests rVWF (vonicog alfa) as a preventive treatment over three years to reduce spontaneous bleeding in people with severe VWD. The effectiveness will be measured by counting how many bleeds participants have in the first year on this treatment.
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
Participants will receive recombinant von Willebrand factor (rVWF).
Participants will receive recombinant von Willebrand factor (rVWF) (with or without ADVATE).
Find a Clinic Near You
Who Is Running the Clinical Trial?
Baxalta now part of Shire
Lead Sponsor
Flemming Ornskov
Baxalta now part of Shire
Chief Executive Officer since 2013
MD, MPH
Ludwig N. Hantson
Baxalta now part of Shire
Chief Medical Officer since 2015
PhD
Takeda Development Center Americas, Inc.
Industry Sponsor
Citations
Real-world treatment patterns and outcomes in people with ...
Participantsreceiving routine rVWF prophylaxis in this study experienced reducedABR and HCRU versus the baseline period, indicating that ...
2.
ashpublications.org
ashpublications.org/blood/article/142/Supplement%201/2613/501771/Real-World-Use-of-Recombinant-Von-WillebrandReal-World Use of Recombinant Von Willebrand Factor in ...
Recombinant von Willebrand factor is indicated for use in adults for routine prophylaxis to reduce the frequency of bleeding episodes in patients with severe ...
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.
Outcomes in Patients With von Willebrand Disease ...
rVWF was used to prevent/treat surgery-related bleeds at index in 76 patients. Bleed resolution was achieved in 25/58 rVWF-treated surgeries; bleed resolution ...
5.
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 ...
Safety Data for VONVENDI® [von Willebrand factor ...
Explore the safety profile and the most common adverse reactions observed in VONVENDI® clinical trials for patients with Von Willebrand Disease (VWD).
Real-World Data from an EU Post-Authorization Safety Study
Overall, no new safety signals were identified in this European real-world study when rVWF was used for the prevention or treatment of ...
8.
ashpublications.org
ashpublications.org/blood/article/122/5/648/32057/Pharmacokinetics-and-safety-of-a-novel-recombinantPharmacokinetics and safety of a novel recombinant human ...
Key Points. rVWF is safe, well tolerated, and has a PK profile generally comparable to pdVWF, but promotes enhanced stabilization of endogenous FVIII.
September 5, 2025 Clinical Review Memo - VONVENDI
No significant safety concerns. ATHN 9 is a Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People with ...
Study Details | NCT00816660 | Pharmacokinetic, Safety ...
The objectives of this study are to evaluate the immediate tolerability and safety of rVWF:rFVIII in subjects with Type 3 Von Willebrand Disease after ...
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