Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A

Recruiting Phase 1 Interventional Study
Von Willebrand Disease, Type 3 Concomitant VWD and Hemophilia
No Placebo Group Every participant receives an active treatment — no one gets a placebo.
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At a Glance
Age
0 – 90
Sex
Any
Trial phase
Phase 1
Study type
Interventional
Purpose
Treatment
Participants needed
40 (estimated)
Sponsor
Bleeding and Clotting Disorders Institute Peoria, Illinois · Other
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About This Trial
Von Willebrand Disease (VWD) is the most common inherited bleeding disorder affecting up to 0.1% of the population, is usually characterized by mucocutaneous bleeding, HMB, surgical bleeding or other hemostatic challenges. Severe bleeding events require VWF concentrates administered solely through intravenous access. Emicizumab (Hemlibra) is a monoclonal bispecific antibody developed to bind activ…
Trial Locations
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Eligibility Criteria
Inclusion Criteria: * Signed informed consent * Age 0 and older (infants weighing ≥3 kg) * ability to comply with protocol in investigators judgement * diagnosis of: severe VWD type 3, or VWD with VWF antigen, activity or collagen binding \</= 20 U/dl or variant VWD confirmed by genetic mutation an…
Contacts

Megan L Woodbury, PhD

309-692-5337

meganw@ilbcdi.org

CONTACT

Dayna Lenski, BS

309-692-5337

dayna@ilbcdi.org

CONTACT