Evaluating the Safety of G-CSF Mobilization in Individuals With Beta Thalassemia Major
Status:
Completed
Trial end date:
2010-08-01
Target enrollment:
Participant gender:
Summary
Beta thalassemia major is a serious genetic disease of the blood. Treatments are limited, and
although a bone marrow transplant from a compatible donor can be curative, only a limited
percentage of individuals with this disease have a matched donor available. A long-term goal
of study researchers is to develop a gene transfer process as a method of curing beta
thalassemia major. Gene transfer involves obtaining blood stem cells from an individual,
adding a normal globin gene to the stem cells, and putting the cells back into the
individual.
Before gene transfer methods can be attempted in individuals with beta thalassemia major, a
safe method of obtaining blood stem cells needs to be developed. The purpose of this study is
to investigate the safety and feasibility of collecting peripheral blood stem cells (PBSC)
from individuals with beta thalassemia major. Research participants will be given G-CSF
(filgrastim) for several days to increase the number of stem cells in the blood, a process
called "mobilization." After mobilization, participants will undergo a procedure called
apheresis to remove the white blood cells. Researchers in the laboratory will purify the stem
cells from the mixture and test methods of putting a normal globin gene into the stem cells.
Half of the participants will receive hydroxyurea (HU) prior to G-CSF mobilization. HU is
used in splenectomized patients to attempt to reduce the risk of clotting during
mobilization. In non-splenectomized patients, HU is given in an attempt to decrease the size
of the spleen.
Phase:
N/A
Details
Lead Sponsor:
University of Washington
Collaborators:
George Papanicolaou Hospital National Heart, Lung, and Blood Institute (NHLBI)