Clinical Trial: Gene Therapy for Chronic Granulomatous Disease

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

Official Title: Autologous Transplantation of Genetically Modified Cells for the Treatment of X-Linked Chronic Granulomatous Disease

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)patients 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: Johann Wolfgang Goethe University Hospital

Current Primary Outcome: safety, toxicity and feasibility [ Time Frame: 2 years ]

Original Primary Outcome: Same as current

Current Secondary Outcome: Engraftment of gene corrected stem cells, functional reconstitution of respiratory burst, clinical benefit [ Time Frame: 2 years ]

Original Secondary Outcome: 1) To evaluate further the safety of gene therapy@@@2) To monitor long-term results of gene therapy with conditioning

Information By: Johann Wolfgang Goethe University Hospital

Dates:
Date Received: October 31, 2006
Date Started: October 30, 2006
Date Completion: December 2008
Last Updated: November 27, 2007
Last Verified: November 2007