Emicizumab for Von Willebrand Disease
(BCDI-XII Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial examines whether Emicizumab, a medication typically administered as a subcutaneous injection, can safely assist individuals with severe Von Willebrand Disease (VWD) or both VWD and hemophilia. These conditions lead to excessive bleeding, and the trial seeks to determine if Emicizumab can prevent bleeding episodes. It suits those with severe VWD or a combination of VWD and hemophilia who currently require regular treatments to manage bleeding. Participants must not have recently used Emicizumab and must be willing to switch to this treatment if they are using another. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the chance to be among the first to receive this potentially groundbreaking therapy.
Will I have to stop taking my current medications?
If you are currently on a prophylaxis treatment for Von Willebrand Disease or VWD/hemophilia A that is not emicizumab, you may need to stop your current treatment to participate in the trial. The protocol requires that you have not taken emicizumab in the last 18 months and are willing to discontinue your current prophylaxis.
Is there any evidence suggesting that Emicizumab is likely to be safe for humans?
Research has shown that Emicizumab is safe for people with bleeding disorders like hemophilia A. Studies have found that Emicizumab can help prevent bleeding with weekly injections under the skin, a method easier and less invasive than injections into a vein.
In past studies, patients who used Emicizumab reported few side effects, most of which were mild, such as skin reactions at the injection site. No serious side effects were commonly reported, suggesting that most patients tolerate Emicizumab well.
The FDA has approved Emicizumab for hemophilia A, further supporting its safety. While individual experiences may vary, current evidence suggests that Emicizumab could be a safe option for managing bleeding disorders.12345Why do researchers think this study treatment might be promising?
Unlike the standard treatments for von Willebrand Disease, which typically involve replacing missing clotting factors, Emicizumab offers a novel approach by mimicking a protein that helps blood clotting. This gives it a unique mechanism of action that doesn't rely on factor replacement. Researchers are excited because Emicizumab is administered as a subcutaneous injection, which could be more convenient and potentially improve adherence compared to intravenous options. Additionally, Emicizumab has shown promise in other bleeding disorders, raising hopes that it might offer a more effective or convenient option for von Willebrand Disease as well.
What evidence suggests that Emicizumab might be an effective treatment for Von Willebrand Disease?
Research has shown that Emicizumab, also known as Hemlibra, is a promising treatment for bleeding disorders. It acts as a bridge between two important blood proteins, aiding proper clotting. In studies with patients who have hemophilia A, Emicizumab effectively prevented unexpected bleeding when administered as a weekly injection under the skin. This trial will evaluate Emicizumab for Von Willebrand Disease (VWD), which also involves blood clotting issues. Although direct data for VWD is limited, Emicizumab's success in similar conditions provides a strong basis for its potential in treating VWD.12467
Who Is on the Research Team?
Jonathan C Roberts, MD
Principal Investigator
Bleeding and Clotting Disorders Institute
Are You a Good Fit for This Trial?
This trial is for individuals with severe Von Willebrand Disease (VWD) or VWD combined with Hemophilia A. Participants must be at least 2 years old, willing to adhere to the study's protocol and use emicizumab prophylaxis. They should not have used emicizumab in the past 18 months unless switching from a different prophylaxis. Menstruating participants must agree to effective contraception or abstinence.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Retrospective Chart Review
One-year retrospective chart review of annualized bleed rate and hemostatic therapies collected at the time of enrollment
Treatment
Participants receive emicizumab with a 3mg/kg weekly loading dose for 4 weeks, followed by once weekly prophylaxis of 1.5mg/kg for 1 year. Dose up-titration to 3 mg/kg once weekly allowed after 24 weeks if suboptimal efficacy observed.
Follow-up
Participants are monitored for safety and effectiveness after treatment, including collection of adverse events, hypersensitivity reactions, and thrombotic events.
What Are the Treatments Tested in This Trial?
Interventions
- Emicizumab
Emicizumab is already approved in United States, European Union for the following indications:
- Hemophilia A
- Hemophilia A with inhibitors
- Hemophilia A
- Hemophilia A with inhibitors
Find a Clinic Near You
Who Is Running the Clinical Trial?
Bleeding and Clotting Disorders Institute Peoria, Illinois
Lead Sponsor
Genentech, Inc.
Industry Sponsor
Ashley Magargee
Genentech, Inc.
Chief Executive Officer since 2024
MBA from Harvard University, BA from Princeton University
Levi Garraway
Genentech, Inc.
Chief Medical Officer since 2021
MD, PhD