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:
$7,402,549
Trial Stage: 
Phase 1/2
Trial Status: 
Suspended
Targeted Enrollment:
10
ClinicalTrials.gov ID:
Details: 

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.

Design: 

X-linked Chronic Granulomatous Disease.

Goal: 

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

Status: 

Enrolling. Early evidence of clinical efficacy.