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

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 - VWD characterization and correction in BOECs

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

NL-OMON

Registry ID

NL-OMON47740

Enrollment

200

Registered

2015-12-17

Start date

2016-05-18

Completion date

Unknown

Last updated

2024-04-23

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Von Willebrand Disease

Interventions

None listed

Eligibility

Age

18 Years to 99 Years

Inclusion criteria

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

Exclusion criteria:

Design outcomes

Primary

Measure	Time frame
As this study is a basic science, experimental in vitro, non-therapeutic study, there are no efficacy parameters. However the outcome of the study will be: optimized isolation and characterization procedures of BOECs, iPSC-ECs, and iPSC-MKs; identification of new pathophysiologic mechanisms of VWD; establishment of the feasibility of silencing of dominant-negative mutant VWF alleles using RNA-targeted silencing or CRISPR/Cas9-editing in BOECs and iPSC-ECs.	—

Measure

Time frame

As this study is a basic science, experimental in vitro, non-therapeutic study, there are no efficacy parameters. However the outcome of the study will be: optimized isolation and characterization procedures of BOECs, iPSC-ECs, and iPSC-MKs; identification of new pathophysiologic mechanisms of VWD; establishment of the feasibility of silencing of dominant-negative mutant VWF alleles using RNA-targeted silencing or CRISPR/Cas9-editing in BOECs and iPSC-ECs.

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Countries

Netherlands

Outcome results