Recombinant vWF Concentrate for Bleeding
What You Need to Know Before You Apply
What is the purpose of this trial?
Adult patients on extracoporeal membrane oxygenation (ECMO) frequently experience bleeding, which is in part caused by acquired von Willebrand syndrome (vWS). Prior in vitro studies have shown that the addition of recombinant von Willebrand Factor (vWF) to ECMO patient blood samples, normalizes platelet adhesion and thrombus formation. This study is a phase I study, where adult ECMO patients with refractory bleeding will be treated with recombinant vWF a single time. The primary objectives are to evaluate the safety, tolerability, and pharmacokinetics of recombinant vWF in adult ECMO patients.
Will I have to stop taking my current medications?
The trial requires that participants stop taking systemic anticoagulation medications for at least 4 hours before joining. Other medications are not specifically mentioned, so it's best to discuss with the trial team.
Is Recombinant von Willebrand Factor (rVWF) safe for humans?
Recombinant von Willebrand Factor (rVWF) has been shown to be generally safe in humans, with studies reporting it was well-tolerated in patients with von Willebrand disease undergoing surgery. Some patients experienced treatment-emergent adverse events, but serious allergic reactions or inhibitory antibodies were not reported.12345
How is the drug Recombinant von Willebrand Factor (rVWF) unique compared to other treatments for bleeding in von Willebrand disease?
Recombinant von Willebrand Factor (rVWF) is unique because it is a purified concentrate produced without exposure to the VWF-cleaving enzyme ADAMTS13, preventing degradation of large VWF multimers, and it does not contain factor VIII, unlike plasma-derived products. This makes it effective for patients who need treatment without the risk of factor VIII accumulation.36789
What data supports the effectiveness of the drug Recombinant von Willebrand Factor (rVWF) for bleeding?
Research shows that rVWF is effective in treating and preventing bleeding episodes in patients with von Willebrand disease, including during surgery. In a study, patients using rVWF had excellent or good control of bleeding, and it was well-tolerated with no severe allergic reactions reported.134910
Who Is on the Research Team?
Michael Mazzeffi, MD
Principal Investigator
UVA
Are You a Good Fit for This Trial?
This trial is for adults on ECMO with major bleeding who aren't on blood thinners for at least 4 hours. It's not for those with recent serious clots, vWF antibodies, low platelets or fibrinogen levels, high INR, liver failure, current trial participation, heparin allergy, inability to consent or pregnancy.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
ECMO patients with major bleeding receive a single intravenous dose of recombinant von Willebrand Factor
Follow-up
Participants are monitored for safety and effectiveness after treatment, including pharmacokinetic assessments and adverse event monitoring
What Are the Treatments Tested in This Trial?
Interventions
- Recombinant vWF Concentrate
Recombinant vWF Concentrate is already approved in United States, European Union for the following indications:
Find a Clinic Near You
Who Is Running the Clinical Trial?
University of Virginia
Lead Sponsor