Clinical trial
Safety and Tolerability of Recombinant Von Willebrand Factor Concentrate in Adult ECMO Patients With Major Bleeding: A Phase I Study
Name
HSR230248
Description
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.
Trial arms
Trial start
2024-08-01
Estimated PCD
2026-08-31
Trial end
2026-12-31
Status
Not yet recruiting
Phase
Early phase I
Treatment
Recombinant von Willebrand Factor
Recombinant von Willebrand Factor is a drug that is currently FDA approved to treat patients with certain types of von Willebrand Disease. In the current trial it will be used to treat ECMO patients who have acquired von Willebrand syndrome.
Arms:
Treatment with recombinant vWF
Size
20
Primary endpoint
Serious Adverse Events
30 days after treatment
Area under the plasma concentration curve from zero to infinity (h × U/dL)
96 hours after treatment
Plasma half-life (hours)
96 hours after treatment
Mean residence time (hours)
96 hours after treatment
Clearance (mL/kg per hour)
96 hours after treatment
Volume at a steady state (dL/kg)
96 hours after treatment
Maximum concentration (U/dL)
96 hours after treatment
Time to maximum concentration (hours)
96 hours after treatment
Incremental recovery ([U/dL]/[U VWF: RCo/kg] for VWF)
96 hours after treatment
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
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'Open label non-randomized phase I trial.', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 20, 'type': 'ESTIMATED'}}
Updated at
2024-05-16
1 organization
1 product
1 indication
Organization
University of VirginiaIndication
Bleeding Disorder