A Phase 1/2 Open-Label, Single-Ascending-Dose Study of EN-374, a Helper-Dependent Adenoviral-Based Gene Therapy, in Participants With X-Linked Chronic Granulomatous Disease
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
• Male
• ≥ 18 years of age during dose escalation, then ≥ 3 months of age during dose expansion
• Diagnosis of X-CGD with DHR+ cells ≤ 5% and a pathogenic mutation in the CYBB gene
• History of at least 1 severe infection requiring medical intervention or chronic inflammatory disorder
• Does not have a suitable, available, and willing human leukocyte antigens (HLA)-matched (10/10) related donor
• Non-sterile male participants who are or may become sexually active with female partners of childbearing potential are required to use highly effective contraception
• Informed consent, with informed assent from capable participants
• Adequate organ function