Phase I/II Clinical Trials Using Gene-Modified Adult Hematopoietic Stem Cells for HIV: Lessons Learnt
Figure 3
(a) The figure shows the prototypic phase II trial design in which CD34+ HSC are obtained from apheresis product that is CD34+ selected. The cells were then transduced with either sham (medium only) or OZ1-containing vector and that population (control or OZ1) infused into the individual. (1) Subjects are injected with a course of G-CSF to mobilize CD34+ HSC from the bone marrow to the peripheral blood. (2) Apheresis product is obtained. (3) The mononuclear cell fraction is applied to a CD34+ isolation system. (4) CD34+ cells are obtained. (5) These are sham transduced (medium alone) or transduced with OZ1 to obtain (6) a control population or OZ1 tranduced cells. (7) This population is infused back into the individual. (b) The figure shows the schedule for the phase II clinical trial. ART, antiretroviral therapy; ATI: analytic treatment interruption. The primary endpoint was viral load at weeks 47/48. Other end-points were area under the viral load curve weeks 40–48 and 40–100.
