Study of EN-374 Gene Therapy in Participants With X-Linked Chronic Granulomatous Disease
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
Ensoma
15 participants
Aug 5, 2025
INTERVENTIONAL
Conditions
Summary
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
Eligibility
Plain Language Summary
Simplified for easier understanding
This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.
Interested in this trial?
Get notified about updates and connect with the research team.
Interventions
Single dose of EN-374 administered by intravenous infusion after mobilization and followed by enrichment
Locations(9)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT06876363