Evaluating the Safety and Effectiveness of Mozobil Mobilization in Adults With Beta-Thalassemia Major
Status:
Completed
Trial end date:
2014-12-01
Target enrollment:
Participant gender:
Summary
Thalassemia is considered the most common genetic disorder worldwide, occurring with high
frequency in Mediterranean areas, the Middle East, Southeast Asia, and the Pacific Islands.
Currently, the only cure for thalassemia is bone marrow transplantation from a related,
compatible donor. Gene transfer, achieved by transplantation of the patient's own blood stem
cells that have been genetically-modified with the corrected gene, could potentially cure
thalassemia.
The first step in developing gene transfer for treatment of thalassemia is to develop a safe
and effective method to obtain blood stem cells from thalassemia patients. Eventually, high
numbers of genetically modified cells will need to be infused into the patient for clinical
gene transfer to be effective. The blood stem cells are obtained by giving a "mobilization"
agent to the patients. This causes the stem cells to leave the bone marrow and go into the
blood. The purpose of this study is to test the safety and effectiveness of the new
mobilization agent, Mozobil, in causing mobilization of blood stem cells for patients with
beta-thalassemia major.
Phase:
N/A
Details
Lead Sponsor:
University of Washington
Collaborators:
Genzyme, a Sanofi Company George Papanicolaou Hospital National Heart, Lung, and Blood Institute (NHLBI)