Megadose CD34 Selected Progenitor Cells for Transplantation in Patients With Advanced Hematological Malignant Diseases
Before taking part in this study, donors will have an evaluation that is the standard for
any bone marrow or blood stem cell donor. These include standard blood tests, an
electrocardiogram (EKG), and a chest X-ray. Donors will also be given a general health
questionnaire that is given to all blood donors in the United States. It should take no
more than 10 minutes to complete the questionnaire.
Donors in this study will receive standard mobilization therapy with daily G-CSF every 12
hours. The Granulocyte colony-stimulating factor (G-CSF) will be given as an injection under
the skin. The mobilization phase starts on the first day that donors receive G-CSF and
continues until the final day of the stem cell collection process (leukapheresis).
Donors in this study will receive Neupogen (white blood cell growth factor) to stimulate the
immature blood cells. They will receive two injections, twice a day for four days. On the
fourth day, assuming they have enough immature white blood cells, researchers will start the
stem cell collection process (leukapheresis).
The stem cell collection will go from 1 to 4 days until enough immature cells have been
collected, but will not be done on any day the donor's platelet count falls below 75,000.
The stem cell is called a CD34(+) cell. These cells will then be processed over a
cell-processing machine to try to purify the immature fraction of cells and remove the
T-lymphocytes that are part of the fatal graft versus host disease. The T-cell is called a
Leukapheresis, with later CD34(+) cell selection, will start on the day when circulating
CD34(+) count is at a high enough level. Leukapheresis will continue until the appropriate
count is reached. If the CD3(+) count is too high, adjustments will be made.
For those donors who cannot reach the collection goal in one series of collection attempts,
researchers will wait until the donor recovers from the first stem cell collection and try
again. If the donor is unable to reach the collection goal again, another attempt will be
made with a different donor.
The blood thinner used for the procedure will be acid citrate dextrose (ACD). Heparin may
be substituted when clinically needed. No additional blood thinners or additives should be
added beyond those normally used during leukapheresis. A unique identification and labeling
system shall be used to track the leukapheresis product from collection to infusion.
Samples will be taken from each leukapheresis product pre- and post-selection for quality
This is an investigational study. No more than 90 donors will take part in this study. All
will be enrolled at M. D. Anderson.
Before taking part in this study, recipients will have standard evaluations to determine the
stage of their disease. These may include bone marrow aspirations and biopsies and if
necessary, CT scans and chest x-rays. All recipients will go through cardiopulmonary
The recipients will have an allogeneic bone marrow transplant with pre-treatment of
thiotepa, fludarabine, melphalan, and antithymocyte globulin. This will be followed by
infusion of the peripheral blood progenitor cells.
Recipients will have daily follow-up exams in the hospital. Recipients will be evaluated at
least one to five times per week after they leave until Day 100. After that, they will have
evaluations at least once every three months until about one year and then once every six
The CliniMACS device is being provided by used of an investigational device exemption for
the FDA. Without the CliniMACS device, this procedure would not be possible.
This is an investigational study. A total of 40 patients will take part in this study. All
will be enrolled at M. D. Anderson.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Number of Participants With Absolute Neutrophil Count Engraftment
Absolute neutrophil engraftment defined as first of 3 consecutive days with Absolute neutrophil count (ANC) equal to or more than 0.5 * 10^9/L. Baseline to Day 30 post transplant.
Day 0 up to Day 30
Richard E. Champlin, MD
M.D. Anderson Cancer Center
United States: Food and Drug Administration
|MD Anderson Cancer Center||Houston, Texas 77030-4096|