Clinical Trial: Reduced Intensity AlloTransplant For Osteopetrosis

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

Official Title: Reduced Intensity Allogeneic Transplantation For Severe Osteopetrosis Incorporating A Second Cd34 Selected Graft

Brief Summary: We believe that hematopoietic stem cell transplantation (HSCT) will help subjects with Osteopetrosis generate functioning osteoclasts, and by so doing assist in the resolution of the abnormal bone architecture, and the anemia and bone marrow failure that is also characteristic of this disease. However, we have found in past studies that approximately 30% of Osteopetrosis patients do not engraft. Therefore, in this study, we plan to use a different combination 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. The purpose of this research is to find 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.

Detailed Summary: This transplant protocol will test the following: 1) the ability to achieve engraftment with the reduced intensity protocol, 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, Campath levels just prior to the administration of the graft, and establishment of mesenchymal stem cell lines. In older patients, studies to evaluation osteoclast differentiation and function will also be offered.
Sponsor: Masonic Cancer Center, University of Minnesota

Current Primary Outcome: Number of Patients Achieving Donor Cell Engraftment [ Time Frame: Day 100 ]

Number of patients with persistent presence of donor-derived cells at Day 100


Original Primary Outcome: Engraftment, as defined by the persistent presence of donor-derived cells. [ Time Frame: 100 days to 5 year ]

Current Secondary Outcome:

  • Number of Patients With Transplant Related Death [ Time Frame: Day 100 ]
    Number of participants died during study by Day 100 and reason for death was related to transplant.
  • Number of Patients With Transplant Related Toxicity [ Time Frame: Day 100 ]
    Number of patients experiencing adverse effects due to transplant categorized by body system using Common Terminology Criteria for Adverse Events coding from the National Cancer Institute, Version 3.0.
  • Differential Imaging and Biologic Evaluations [ Time Frame: Day 100, 6 months, 1, 2 and 5 years ]
    These outcome measures were not assessed due to early study termination.


Original Secondary Outcome:

  • The mortality associated with transplant [ Time Frame: by 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: March 11, 2008
Date Started: September 2007
Date Completion:
Last Updated: February 22, 2011
Last Verified: February 2011