Granulomatous Disease, Chronic Clinical Trial
Official title:
Phase I/II Gene Therapy Study for X-Linked Chronic Granulomatous Disease
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.
n/a
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label
Status | Clinical Trial | Phase | |
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Not yet recruiting |
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