Chronic Granulomatous Disease clinical trials at University of California Health
2 research studies open to eligible people
Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )
open to eligible people ages 6 years and up
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.
at UCLA
Prevalence of Antibodies and Cytokines in Participants With Chronic Granulomatous Disease
open to all eligible people
This research study is designed to assess the prevalence of specific antibodies and inflammatory cytokines in adult and pediatric participants with CGD.
at UCSF
Our lead scientists for Chronic Granulomatous Disease research studies include Donald B. Kohn, MD Morna Dorsey, MD.