Clinical Trial: Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in GRD
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Genetic Rare Disease
Brief Summary: To evaluate the outcome of hematopoietic stem cell transplantation using targeted busulfan, fludarabine conditioning regimen in genetic rare disease
Detailed Summary: Busulfan is a highly toxic drug with narrow therapeutic window used for the conditioning of hematopoietic stem cell transplantation. High exposure is associated with systemic toxicity such as veno-occlusive disease (VOD) and underexposure is associated with graft failure or relapse. In this study the investigators plan to improve the outcome of hematopoietic stem cell transplantation by using optimal busulfan dose through pharmacokinetic study.
Sponsor: Seoul National University Hospital
Current Primary Outcome: To evaluate engraftment rate [ Time Frame: 1 year ]
Original Primary Outcome: Same as current
Current Secondary Outcome: To evaluate treatment related mortality [ Time Frame: 1 year ]
Original Secondary Outcome: Same as current
Information By: Seoul National University Hospital
Dates:
Date Received: January 9, 2014
Date Started: January 2014
Date Completion: December 2018
Last Updated: April 26, 2017
Last Verified: April 2017