Total Body Irradiation Dose De-escalation Study in Patients With Fanconi Anemia Undergoing Alternate Donor Hematopoietic Cell Transplantation
Study Treatment: Patients will receive voriconazole (antifungal therapy) by mouth beginning
1 month prior to conditioning therapy, if possible. 1) The subject is to receive total body
irradiation (300 cGy) with thymic shielding; it will be given six days before the stem cells
are given (day -6). 2) Day -5 through Day -2, subjects will receive a chemotherapy regimen
of Fludarabine and Cyclophosphamide via central line (i.e. Hickman or Broviac). Starting
Day -3, patients will receive sirolimus therapy with a taper commencing on day +180 and also
mycophenolate mofetil (MMF) through day +30 or for 7 days after engraftment, whichever day
is later, if no acute graft-versus-host disease (GVHD). 4) If the subject is receiving bone
marrow or "peripheral" stem cells (cells collected from the donor's arm via a cell
separator), on the day of transplantation, the stem cells taken from the donor will be put
into a machine which will separate the lymphocytes (the cells that cause graft-versus-host
disease [GVHD]) from the stem cells. If the subject is receiving an umbilical cord blood,
the lymphocytes will not be removed because the risk of GVHD is not as high. Otherwise all
patients will receive the same treatment. The stem cells are given as an infusion into the
subject's existing catheter over 1-2 hours on day 0.5. On the day after transplant (day +1)
subjects will be given G-CSF to stimulate the growth of the transplanted cells. 6. While
receiving treatment and until the subject's blood counts recover he/she will have daily
blood tests, and several bone marrow biopsies and aspirates. After recovery, subjects will
be seen once a month for a health assessment and blood tests until at least 3 months after
the cells have been infused. Additional blood tests or assessments may be done as medically
indicated.
Interventional
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Incidence of neutrophil recovery (absolute neutrophil count ≥500/µL for three consecutive days) .
by day 42
Yes
Margaret L MacMillan, M.D.
Principal Investigator
University of Minnesota Medical Center
United States: Food and Drug Administration
MT2006-05
NCT00352976
May 2006
May 2016
Name | Location |
---|---|
University of Minnesota Medical Center | Minneapolis, Minnesota 55455 |