Collection of Blood in Normal Subjects and Subjects With Von Willebrand Disease (VWD)

This study has been withdrawn prior to enrollment.
(Prinicipal Investigator initiated study closure.)
Sponsor:
Information provided by:
Weill Medical College of Cornell University
ClinicalTrials.gov Identifier:
NCT00630448
First received: February 27, 2008
Last updated: August 10, 2010
Last verified: August 2010

February 27, 2008
August 10, 2010
November 2008
July 2010   (final data collection date for primary outcome measure)
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Complete list of historical versions of study NCT00630448 on ClinicalTrials.gov Archive Site
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Collection of Blood in Normal Subjects and Subjects With Von Willebrand Disease (VWD)
Collection of Blood in Normal Subjects and Subjects With Von Willebrand Disease (VWD)

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.

Not Provided
Observational
Observational Model: Case Control
Time Perspective: Cross-Sectional
Not Provided
Retention:   Samples With DNA
Description:

Blood

Probability Sample

Source of subjects will be the population of individuals with known Von Willebrand Disease and the normal population from 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"

Von Willebrand Disease
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  • 1
    Control Group. Normal (healthy) individuals without Von Willebrand Disease.
  • 2
    Case Group. Individuals with known Von Willebrand Disease.
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*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Withdrawn
40
October 2010
July 2010   (final data collection date for primary outcome measure)

Inclusion Criteria:

  1. 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"

  2. 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

Exclusion Criteria:

  1. 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)
  2. 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)
Both
18 Years to 70 Years
Yes
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00630448
0708009371
Yes
Ronald G. Crystal, MD, Weill Cornell Medical College; Department of Genetic Medicine
Weill Medical College of Cornell University
Not Provided
Principal Investigator: Ronald G Crystal Weill Medical College of Cornell University
Weill Medical College of Cornell University
August 2010

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP