Reduced Intensity AlloTransplant For Osteopetrosis

Status Terminated

Clinical Trial 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.

Clinical Trial Description

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. ;

Study Design

Related Conditions & MeSH terms

Osteopetrosis

Clinical Trial Details

NCT number	NCT00638820
Study type	Interventional
Source	Masonic Cancer Center, University of Minnesota
Contact
Status	Terminated
Phase	Phase 2
Start date	September 2007
Completion date	May 2008

View Details

See also
Status	Clinical Trial	Phase
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Terminated	`NCT00145587` - Stem Cell Transplantation for Children Affected With Osteopetrosis	N/A
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