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.
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