Allogeneic Hematopoietic Stem Cell Transplantation For Severe Osteopetrosis
This revised transplant protocol will test the following: 1) the ability to achieve
engraftment with the reduced intensity protocol and a second infusion of stem cells on day
42, 2) the mortality associated with transplant by day 100, 3) patient outcomes, based on
differential imaging and biologic evaluations prior to transplantation and at designated
points after transplantation (day 100, 6 months, 1, 2 and 5 years). Additional biologic
studies will include microarray analysis, and evaluation of blood parameters and genes that
may be important in the disease process. In older patients, studies to evaluation
osteoclast differentiation and function will also be offered.
Interventional
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Estimate the rate of donor engraftment for patients treated by hematopoietic stem cell transplantation
Day 100
No
Paul Orchard, MD
Principal Investigator
Masonic Cancer Center, University of Minnesota
United States: Institutional Review Board
MT2008-20
NCT00775931
October 2008
October 2015
Name | Location |
---|---|
University of Minnesota, Fairview | Minneapolis, Minnesota 55455 |