Allogeneic Transplantation For Severe Osteopetrosis
Status:
Completed
Trial end date:
2013-06-01
Target enrollment:
Participant gender:
Summary
The purpose of this research is to explore what we believe may be a safer and more effective
means of performing stem cell transplantation in patients with Osteopetrosis, using
chemotherapy and radiation designed to bring about engraftment and lessen transplant
mortality. Prior multi-institutional data in past studies found that approximately 30% of
Osteopetrosis patients do not engraft. Therefore, in this study, we utilize a reduced
intensity design of pre-transplant drugs to try to make transplants safer for this disease,
as well as to provide a second infusion of stem cells in patients with matched related or
unrelated donors.