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 ]

To evaluate engraftment rate


Original Primary Outcome: Same as current

Current Secondary Outcome: To evaluate treatment related mortality [ Time Frame: 1 year ]

To evaluate treatment related mortality


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