Blood Outgrowth Endothelial Cells (BOECs) to study the pathophysiology of von Willebrand disease and the feasibility of in vitro correction of von Willebrand factor defects
Completed
- Conditions
- Von Willebrand Disease1006447710005330
- Registration Number
- NL-OMON47740
- Lead Sponsor
- eids Universitair Medisch Centrum
- Brief Summary
Trial is onging in other countries
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 80
Inclusion Criteria
Patients with von Willebrand disease (any subtype), age 18 years or older.
Family members of patients with von Willebrand disease (may or may not be
affected themselves), age 18 years or older.
Healthy control (not known with von Willebrand disease or other hemostatic
disorders), age 18 years or older.
Exclusion Criteria
< 18 years of age
Unable to visit the LUMC or Erasmus MC
Unable to give written informed consent
Study & Design
- Study Type
- Observational invasive
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>As this study is a basic science, experimental in vitro, non-therapeutic study,<br /><br>there are no efficacy parameters.<br /><br>However the outcome of the study will be: optimized isolation and<br /><br>characterization procedures of BOECs, iPSC-ECs, and iPSC-MKs; identification of<br /><br>new pathophysiologic mechanisms of VWD; establishment of the feasibility of<br /><br>silencing of dominant-negative mutant VWF alleles using RNA-targeted silencing<br /><br>or CRISPR/Cas9-editing in BOECs and iPSC-ECs.</p><br>
- Secondary Outcome Measures
Name Time Method <p>Not applicable</p><br>