Emicizumab study for severe von Willebrand disease or overlap disorder
Treatments studied
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This Phase 1 trial studies how well and safely emicizumab works for people with severe von Willebrand disease (VWD) or VWD plus hemophilia A. Emicizumab is a preventive medicine given regularly to reduce bleeding risk.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You (or your child) must be age 0 or older, and infants must weigh at least 3 kg
- Your past test results must show severe VWD, or a VWD/hemophilia A overlap, with low clotting protein levels
- Your bleeding has to be serious enough that your clinician recommends regular (prophylaxis) treatment
- You must be able to follow the study plan and be willing to use emicizumab as your preventive treatment during the study
- If you are not already on emicizumab, you must be able to stop your current prophylaxis (timing rules apply)
- If you can become pregnant, you must use very reliable birth control or abstain, and you must not be pregnant
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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