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) is a rare immune disorder that prevents white blood cells from killing foreign invaders. This results in severe, recurrent infections that can impact quality and length of a patient's life. X-CGD is usually diagnosed before age 5, but without treatment, children die before age 10. A team at UCLA is using the patient’s own genetically modified blood stem cells to create a new blood supply and a healthy immune system, with the aim of curing patients with this disease.
X-linked Chronic Granulomatous Disease.
Primary: Safety and Efficacy. Secondary: Restoration of immune function
Enrolling. Early evidence of clinical efficacy.