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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This study has been withdrawn prior to enrollment.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:
ClinicalTrials.gov Identifier:
NCT00630448
First Posted: March 7, 2008
Last Update Posted: May 25, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Weill Medical College of Cornell University
February 27, 2008
March 7, 2008
May 25, 2017
November 2008
July 2011   (Final data collection date for primary outcome measure)
Blood outgrowth endothelial cells from peripheral blood [ Time Frame: Through study completion, an average of 1 month ]
The number of isolated blood outgrowth endothelial cells from peripheral blood will be calculated
Not Provided
Complete list of historical versions of study NCT00630448 on ClinicalTrials.gov Archive Site
Not Provided
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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.

Aim: The purpose of this protocol is 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 transfer for Von Willebrand disease.

Experimental procedure. This protocol is designed to gather approximately 90 ml (6 tablespoons) of blood from normal individuals without Von Willebrand disease and individuals with known Von Willebrand disease. A total of 20 normal individuals and 20 Von Willebrand disease patients will be enrolled into the study.

Studies to be performed on the blood samples:

Each blood draw will require 10 large blue/black capped (Na Citrate) vacutainer (16x125mm, BD# 362761, Cell Preparation Tubes (CPT) to be filled from each volunteer. All the blood will be used for cell processing.

In initial proof-of-concept experiments, BOEC lines will be established from the peripheral blood of normal individuals and research subjects with Von Willebrand disease. Following culture and phenotypic characterization, they will be transduced with a lentivirus gene transfer vector expressing a reporter gene or wild-type Von Willebrand factor. After demonstrating successful transduction, the next step will be to write an animal protocol that will introduce the modified cells into immunocompromised mice and monitor the mice for stable implantation.

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
Not Provided
  • Group 1
    Control Group. Normal (healthy) individuals without Von Willebrand Disease.
  • Group 2
    Case Group. Individuals with known Von Willebrand Disease.
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Withdrawn
0
July 2011
July 2011   (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)
Sexes Eligible for Study: All
18 Years to 70 Years   (Adult, Senior)
Yes
Contact information is only displayed when the study is recruiting subjects
United States
 
 
NCT00630448
0708009371
Yes
Not Provided
Plan to Share IPD: No
Weill Medical College of Cornell University
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
May 2017