Clinical Trial: Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) in Children
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: Phase I/II Gene Therapy Study for X-linked Chronic Granulomatous Disease in Children
Brief Summary: The aim of the study is to evaluate the side effects and risks after infusion of retroviral gene corrected autologous CD34+ cells of the peripheral blood of chemotherapy conditioned (busulphan) children with chronic granulomatous disease (CGD). Also gene corrected and functional active granulocytes in the peripheral blood and the engraftment in the bone marrow of the patients will be monitored an documented.
Detailed Summary:
Sponsor: University of Zurich
Current Primary Outcome: eradication of pre-existing therapy refractory bacterial and/or fungal infections [ Time Frame: 6 months ]
Original Primary Outcome: Same as current
Current Secondary Outcome: Reconstitution of ROS production by peripheral blood cells [ Time Frame: 1 month ]
Original Secondary Outcome: Same as current
Information By: University of Zurich
Dates:
Date Received: June 22, 2009
Date Started: June 2004
Date Completion:
Last Updated: September 26, 2011
Last Verified: September 2011