Clinical Trial: Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Chronic Granulomatous Disease(CGD)

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

Official Title:

Brief Summary: In this study the investigators plan to use optimal busulfan dose through pharmacokinetic study in stem cell transplantation of CGD patients.

Detailed Summary:

Chronic granulomatous disease is one of the rare congenital immunodeficiency which can be cured by hematopoietic stem cell transplantation. Previous myeloablative conditioning regimen has problems related to the severe toxicities, and non-myeloablative conditioning regimen has the risk of graft failure. Recently, reduced-intensity myeloablative conditioning regimen with busulfan and fludarabine was used usually in leukemia patients.

Busulfan is a highly toxic drug with narrow therapeutic window. In this study we plan to use optimal busulfan dose through pharmacokinetic study in stem cell transplantation of CGD patients.


Sponsor: Seoul National University Hospital

Current Primary Outcome: Engraftment rate [ Time Frame: 1 month after transplantation ]

To evaluate engraftment rate after hematopoietic stem cell transplantation.


Original Primary Outcome: Same as current

Current Secondary Outcome: Transplantation-related mortality and toxicities [ Time Frame: 1, 3, 6 and 12 months after transplantation ]

To evaluate 1-year event free survival after hematopoietic stem cell transplantation, toxicities associated with hematopoietic stem cell transplantation, acute and chronic GVHD, treatment related mortality and relapse rate.


Original Secondary Outcome: Same as current

Information By: Seoul National University Hospital

Dates:
Date Received: March 4, 2011
Date Started: January 2011
Date Completion: December 2013
Last Updated: November 17, 2013
Last Verified: November 2013