NCT06118372

Brief Summary

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 Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
12

participants targeted

Target at below P25 for phase_1

Timeline
10mo left

Started Oct 2024

Typical duration for phase_1

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress66%
Oct 2024Feb 2027

First Submitted

Initial submission to the registry

September 21, 2023

Completed
2 months until next milestone

First Posted

Study publicly available on registry

November 7, 2023

Completed
11 months until next milestone

Study Start

First participant enrolled

October 3, 2024

Completed
2.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2026

Expected
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

February 28, 2027

Last Updated

February 5, 2026

Status Verified

February 1, 2026

Enrollment Period

2.2 years

First QC Date

September 21, 2023

Last Update Submit

February 2, 2026

Conditions

Bleeding Disorder

Outcome Measures

Primary Outcomes (9)

  • Serious Adverse Events

    Events that 1) cause death, 2) are life-threatening, 3) cause permanent damage, 4) required intervention to prevent harm, or 5) are otherwise serious and jeopardize the patient's safety.

    30 days after treatment

  • Area under the plasma concentration curve from zero to infinity (h Ă— U/dL)

    Pharmacokinetic parameter

    96 hours after treatment

  • Plasma half-life (hours)

    Pharmacokinetic parameter

    96 hours after treatment

  • Mean residence time (hours)

    Pharmacokinetic parameter

    96 hours after treatment

  • Clearance (mL/kg per hour)

    Pharmacokinetic parameter

    96 hours after treatment

  • Volume at a steady state (dL/kg)

    Pharmacokinetic parameter

    96 hours after treatment

  • Maximum concentration (U/dL)

    Pharmacokinetic parameter

    96 hours after treatment

  • Time to maximum concentration (hours)

    Pharmacokinetic parameter

    96 hours after treatment

  • Incremental recovery ([U/dL]/[U VWF: RCo/kg] for VWF)

    Pharmacokinetic parameter

    96 hours after treatment

Secondary Outcomes (2)

  • Change in bleeding severity class

    24 hours after treatment

  • Change in bleeding/drain output volume from existing surgical drains

    24 hours after treatment

Study Arms (1)

Treatment with recombinant vWF

EXPERIMENTAL

ECMO patients with major bleeding who are enrolled in the trial will receive treatment with recombinant von Willebrand Factor a single time. The dose will be 50 IU/kg and the drug will be given intravenously.

Drug: Recombinant von Willebrand Factor

Interventions

Recombinant von Willebrand FactorDRUG

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.

Treatment with recombinant vWF

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

  • Platelet count less than 40 x 109/L
  • International normalized ratio\> 2.0
  • Fibrinogen less than 150 mg/dL
  • Current participation in another clinical trial (interventional)
  • Heparin induced thrombocytopenia (active)
  • Acute liver failure, as indicated by bilirubin \>20 mg/dL or new onset hepatic encephalopathy
  • Patient or legally authorized representative unable to give informed consent
  • Allergy to recombinant von Willebrand Factor or any component of the product based on prior exposure
  • 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
  • Known congenital or acquired thrombophilia
  • 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.
  • History of hypersensitivity to vWF concentrate
  • Known history of vWF antibodies

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

UVA Hospital

Charlottesville, Virginia, 22903, United States

RECRUITING

Related Publications (4)

  • Mazzeffi M, Bathula A, Tabatabai A, Menaker J, Kaczorowski D, Madathil R, Galvagno S, Pasrija C, Rector R, Tanaka K, Herr D. Von Willebrand Factor Concentrate Administration for Acquired Von Willebrand Syndrome- Related Bleeding During Adult Extracorporeal Membrane Oxygenation. J Cardiothorac Vasc Anesth. 2021 Mar;35(3):882-887. doi: 10.1053/j.jvca.2020.06.083. Epub 2020 Jul 3.

    PMID: 32758410RESULT

  • Mazzeffi M, Hasan S, Abuelkasem E, Meyer M, Deatrick K, Taylor B, Kon Z, Herr D, Tanaka K. Von Willebrand Factor-GP1balpha Interactions in Venoarterial Extracorporeal Membrane Oxygenation Patients. J Cardiothorac Vasc Anesth. 2019 Aug;33(8):2125-2132. doi: 10.1053/j.jvca.2018.11.031. Epub 2018 Nov 22.

    PMID: 30595484RESULT

  • Mazzeffi M, Henderson R, Krause E, Rabin J, Madathil R, Chow J, Grazioli A, Meyer M, Wu Z, Tanaka K. In Vitro Comparison of Recombinant and Plasma-Derived von Willebrand Factor Concentrate for Treatment of Acquired von Willebrand Syndrome in Adult Extracorporeal Membrane Oxygenation Patients. Anesth Analg. 2022 Feb 1;134(2):312-321. doi: 10.1213/ANE.0000000000005831.

    PMID: 34903705RESULT

  • Mazzeffi M, Gonzalez-Almada A, Wargowsky R, Ting L, Moskowitz K, Hockstein M, Davison D, Levy JH, Tanaka KA. In Vitro Treatment of Extracorporeal Membrane Oxygenation Coagulopathy with Recombinant von Willebrand Factor or Lyophilized Platelets. J Cardiothorac Vasc Anesth. 2023 Apr;37(4):522-527. doi: 10.1053/j.jvca.2022.12.028. Epub 2022 Dec 30.

    PMID: 36690556RESULT

MeSH Terms

Conditions

Hemostatic Disorders

Condition Hierarchy (Ancestors)

Vascular DiseasesCardiovascular DiseasesHemorrhagic DisordersHematologic DiseasesHemic and Lymphatic Diseases

Study Officials

  • Michael Mazzeffi, MD

    UVA

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Michael Mazzeffi, MD

CONTACT

434-924-9520syy4wa@uvahealth.org

Keita Ikeda, PhD

CONTACT

434-924-2283KI2D@uvahealth.org

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Model Details: Open label non-randomized phase I trial.
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor of Anesthesiology

Study Record Dates

First Submitted

September 21, 2023

First Posted

November 7, 2023

Study Start

October 3, 2024

Primary Completion (Estimated)

December 31, 2026

Study Completion (Estimated)

February 28, 2027

Last Updated

February 5, 2026

Record last verified: 2026-02

Data Sharing

IPD Sharing
Will not share

Locations

US(1)