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

Von Willebrand Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT00630448
• Other study ID #: 0708009371
• Status: Withdrawn
• Phase: N/A
• First received: February 27, 2008
• Last updated: May 23, 2017
• Start date: November 2008
• Est. completion date: July 2011

Study information

• Verified date: May 2017
• Source: Weill Medical College of Cornell University
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

Description:

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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: July 2011
• Est. primary completion date: July 2011
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 70 Years

Eligibility

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)

Related Conditions & MeSH terms

• Von Willebrand Disease
• Von Willebrand Diseases

Locations

Country	Name	City	State
United States	Weill Cornell Medical College	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
Weill Medical College of Cornell University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Blood outgrowth endothelial cells from peripheral blood	The number of isolated blood outgrowth endothelial cells from peripheral blood will be calculated	Through study completion, an average of 1 month