A Phase I/II, Non Randomized, Multicenter, Open-Label Study of G1XCGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease
X-linked Chronic Granulomatous Disease (X-CGD) prevents white blood cells from killing foreign invaders. Patients have persistent, untreatable tissue infections. Affects 1:200,000 in US. Usually diagnosed before age 5, without treatment children die before age 10. Project plan is transplantation of severe X-CGD patients that lack matched donors using gene-corrected autologous HSCT.
X-linked Chronic Granulomatous Disease.
Primary: Safety and Efficacy. Secondary: Restoration of immune function
Enrolling. Early evidence of clinical efficacy.