Clinical Trial: Allogeneic Transplantation For Severe Osteopetrosis

Study Status: Terminated
Recruit Status: Unknown status
Study Type: Interventional

Official Title: Allogeneic Hematopoietic Stem Cell Transplantation For Severe Osteopetrosis

Brief 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.

Detailed Summary: 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.
Sponsor: Masonic Cancer Center, University of Minnesota

Current Primary Outcome: Estimate the rate of donor engraftment for patients treated by hematopoietic stem cell transplantation [ Time Frame: Day 100 ]

Original Primary Outcome: the ability to achieve engraftment with the reduced intensity protocol and a second infusion of stem cells on day 42 [ Time Frame: Day 100 ]

Current Secondary Outcome:

  • Estimate Peri-transplant mortality (deaths) [ Time Frame: day 100 ]
  • Transplant related toxicity [ Time Frame: Day 100 post transplant ]
  • Graft-versus-host disease (incidence and severity) [ Time Frame: after transplantation ]
  • Tolerance of Campath-1H administration [ Time Frame: During study ]
  • Clinical Disease Monitoring [ Time Frame: post transplant ]


Original Secondary Outcome:

  • the mortality associated with transplant [ Time Frame: day 100 ]
  • patient outcomes, based on differential imaging and biologic evaluations prior to transplantation and at designated points after transplantation [ Time Frame: day 100, 6 months, 1, 2 and 5 years ]


Information By: Masonic Cancer Center, University of Minnesota

Dates:
Date Received: October 16, 2008
Date Started: August 2008
Date Completion: October 2015
Last Updated: April 22, 2015
Last Verified: April 2015