Using recombinant von Willebrand Factor to treat bleeding in ECMO patients

Safety and Tolerability of Recombinant Von Willebrand Factor Concentrate in Adult ECMO Patients With Major Bleeding: A Phase I Study

This study is testing if a new treatment using recombinant von Willebrand Factor can help adults on ECMO who are having serious bleeding problems.

Quick facts

• Phase: PHASE1
• Study type: Interventional
• Enrollment: 12 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: University of Virginia (other)
• Locations: 1 site (Charlottesville, Virginia)
• Trial ID: NCT06118372 on ClinicalTrials.gov

What this trial studies

This phase I study investigates the safety, tolerability, and pharmacokinetics of recombinant von Willebrand Factor (vWF) in adult patients experiencing refractory bleeding while on extracorporeal membrane oxygenation (ECMO). The study aims to address the common complication of coagulopathic bleeding in ECMO patients, which is often linked to acquired von Willebrand syndrome. By administering recombinant vWF, the researchers hope to improve platelet adhesion and reduce bleeding episodes. The study will involve a single treatment of recombinant vWF and will monitor patient responses to evaluate its effectiveness.

Who should consider this trial

Good fit: Ideal candidates for this study are adult patients aged 18 and older who are currently on ECMO and experiencing major bleeding.

Not a fit: Patients with severe thrombocytopenia, active heparin-induced thrombocytopenia, or other significant coagulopathies may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly reduce bleeding complications in ECMO patients, potentially improving their overall outcomes.

How similar studies have performed: Previous studies have shown promising results with plasma-derived vWF concentrate in similar patient populations, suggesting that this approach may be effective.

Eligibility criteria

Inclusion Criteria:

1. Adult patients (18 years or greater)
2. On extracorporeal membrane oxygenation
3. Major bleeding defined by CTCAE class 3 or greater
4. Off systemic anticoagulation for at least 4 hours

Exclusion Criteria:

1. Platelet count less than 40 x 109/L
2. International normalized ratio\> 2.0
3. Fibrinogen less than 150 mg/dL
4. Current participation in another clinical trial (interventional)
5. Heparin induced thrombocytopenia (active)
6. Acute liver failure, as indicated by bilirubin \>20 mg/dL or new onset hepatic encephalopathy
7. Patient or legally authorized representative unable to give informed consent
8. Allergy to recombinant von Willebrand Factor or any component of the product based on prior exposure
9. Of childbearing age and positive pregnancy test during the same hospital admission, a pregnancy test will be mandatory for all women of child-bearing age
10. Known congenital or acquired thrombophilia
11. History of deep venous thrombosis, pulmonary embolism, circuit thrombosis, disseminated intravascular coagulation (DIC), ischemic stroke, ST elevation myocardial infarction (STEMI), or arterial thrombosis in the last 3 months.
12. History of hypersensitivity to vWF concentrate
13. Known history of vWF antibodies

Where this trial is running

Charlottesville, Virginia

• UVA Hospital — Charlottesville, Virginia, United States (RECRUITING)

Study contacts

• Principal investigator: Michael Mazzeffi, MD — UVA
• Study coordinator: Michael Mazzeffi, MD

Conditions: Bleeding Disorder