TBI Dose De-escalation for Fanconi Anemia

    Not Recruiting
  • End date
    Dec 12, 2022
  • participants needed
  • sponsor
    Masonic Cancer Center, University of Minnesota
Updated on 25 January 2021
platelet count
stem cell transplantation
total body irradiation
mycophenolate mofetil
bone marrow transplant
acute leukemia
ejection fraction
karnofsky performance status
growth factors
fanconi syndrome


This is a single arm, total body irradiation (TBI) trial. All patients will be prescribed TBI 300 cGy with the goal of evaluating secondary endpoints.


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.

Condition Fanconi's Anemia, Fanconi Anemia
Treatment cyclophosphamide, mycophenolate mofetil, Fludarabine, Total body irradiation, Sirolimus, Bone Marrow Transplantation, Bone Marrow Transplantation
Clinical Study IdentifierNCT00352976
SponsorMasonic Cancer Center, University of Minnesota
Last Modified on25 January 2021

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