Chronic Granulomatous Disease clinical trials at UCLA
2 in progress, 1 open to eligible people
EN-374 Gene Therapy in Participants With X-Linked Chronic Granulomatous Disease
open to eligible males ages 3 months and up
The goal of this clinical trial is to evaluate the safety and potential efficacy of the EN-374 treatment regimen and identify a dose level for further evaluation in participants with x-linked chronic granulomatous disease. The main questions it aims to answer are: - safety of the EN-374 treatment regimen - effect of the EN-374 treatment regimen on the production of functional neutrophils with NADPH oxidase activity
Los Angeles, California and other locations
Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )
Sorry, in progress, not accepting new patients
This is an open-label, single-arm, multicenter Phase 1/2 study evaluating the safety and efficacy of gene therapy by transplantation of Prime Edited autologous CD34+ stem cells modified ex vivo (PM359) in participants with autosomal recessive Chronic Granulomatous Disease (CGD) caused by mutations in the NCF1 (Neutrophil Cytosolic Factor 1) gene.
Los Angeles, California and other locations
Our lead scientists for Chronic Granulomatous Disease research studies include Donald Kohn, MD.
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