A Phase II Trial Evaluating the Safety and Efficacy of Plerixafor and Sargramostim (GM-CSF) for the Mobilization of Peripheral Blood Stem Cells (PBSC) From Normal, HLA-Matched Allogeneic Sibling Donors
The main purpose of this study is to gather information about the combination the drugs
plerixafor with sargramostim in donors of blood-forming cells (stem cells). Stem cells can
be taken from the bone marrow of the pelvic bones or from the blood following treatment with
drugs called growth factors; sargramostim is such a drug. Once stem cells leave the bone
marrow and circulate in the blood, they are called peripheral blood stem cells (PBSCs).
These cells can be collected through a routine procedure called apheresis, which involves
placing two IVs into the arm which are connected to an apheresis machine; the machine then
takes blood from the body, removes the stem cells, and returns the blood to the body.
Normally, a growth factor called filgrastim is given to donors in order to collect the stem
cells used for transplantation. However, when stem cells collected using filgrastim are
transplanted in patients, a possible unpredictable complication is graft versus host
disease. It's thought that using a different growth factor such as sargramostim might
reduce the occurrences of graft versus host disease in patients. However, sargramostim
alone does not provide as many stem cells for transplantation as other growth factors.
Plerixafor is another drug that can increase the number of PBSCs in a donor, but like with
sargramostim, plerixafor alone does not always provide enough stem cells. This is why
sargramostim and plerixafor are being combined in this study: the investigators believe that
the two drugs together will provide enough stem cells for transplantation and may also
reduce the risk of graft versus host disease.
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Reduce the number of donors requiring a second collection
The primary endpoint is to reduce the number of donors treated with GM-CSF who require a second collection to obtain a minimum CD34/Kg (2 x 106) necessary for allogeneic stem cell transplantation when compared to historic controls mobilized with GM-CSF or plerixafor alone. A reduction in failed first leukapheresis from 40% to less than 10% as seen with G-CSF alone would be considered clinically meaningful.
5 days donor and 30 days recipient
Mark Schroeder, M.D.
Washington University School of Medicine
United States: Institutional Review Board
10-1154 / 201108083
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