Collection of Blood in Normal Subjects and Subjects With Von Willebrand Disease (VWD)
Withdrawn before enrolling
Conditions studied: Von Willebrand Disease
In brief
Von Willebrand disease is an inherited bleeding disorder that impacts the blood's ability to clot properly. Von Willebrand disease is cause by the lack or not working substance in the blood known as Von Willebrand factor. Current therapy for Von Willebrand disease includes desmopressin acetate (DDAVP) and /or VWF/FVIII concentrates. Patients with severe Von Willebrand disease face a lifetime of weekly treatments and mounting medical bills. Gene therapy could help these patients improve their quality of life by providing the missing factors necessary for the blood's ability to clot properly. The gene transfer options being studied include naked DNA, viral gene transfer vectors encoding Von Willebrand factor transgenes, and ex vivo cell therapy. The latter involves transplantation of the patient's own cells modified with a corrected copy of the defective gene. Human blood outgrowth endothelial cells (BOEC) display all the properties needed for successful ex vivo cell therapy. We plan to obtain blood samples from normal research subjects and patients with Von Willebrand Disease in order to isolate blood outgrowth endothelial cells (BOEC) from peripheral blood, and develop a ex vivo gene therapy for Von Willebrand Disease.
Key facts
- Study ID
- NCT00630448
- Run by
- Weill Medical College of Cornell University
- People needed
- 0
- Starts
- 2008-11-01
- Expected to finish
- 2011-07-01
- Last updated by the study team
- 2017-05-25
Who can join
Age: 18 and older, up to 70. Sex: any. Healthy volunteers: accepted.
You may qualify if…
- Normal subjects:
- study individuals will be taken from those enrolled in Weill-IRB protocol #0005004439 entitled "Evaluation of the Lungs of Normal (Smokers, Ex-smokers, Non-smokers) Individuals with Segmental Bronchopulmonary Lung Lavage, Bronchial Brushing, and Bronchial Wall Biopsy"
- Subjects with von Willebrand Disease
- a definitive diagnosis on VWD from a patient's physician
- all subjects should be able to provide informed consent
- males or females 18-70 years of age
You may not qualify if…
- Normal individuals
- individuals with a history of bleeding disorders
- individuals with anemia (defined as females with an Hgb concentration less than 12 and males with an HgB concentration less than 12.5)
- Subjects with VWD
- females who are pregnant will not be accepted into the study
- individuals with anemia (defined as females with an Hgb concentration less than 12 and males with an Hgb concentration less than 12.5)
Where it is running
- Weill Cornell Medical College — New York, New York, United States
Full record on ClinicalTrials.gov
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