CIRM Funded Clinical Trials

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


Donald Kohn
Disease Area: 
X-linked Chronic Granulomatous Disease
Investigator:
CIRM Grant:
Award Value:
$7,083,364
Trial Stage: 
Phase 1/2
Trial Status: 
Suspended
Targeted Enrollment:
10
ClinicalTrials.gov ID:
Details: 

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.

Design: 

X-linked Chronic Granulomatous Disease.

Goal: 

Primary: Safety and Efficacy. Secondary: Restoration of immune function

Updates: 

Enrolling. Early evidence of clinical efficacy.