Study of EN-374 Gene Therapy in Participants with X-Linked Chronic Granulomatous Disease
Research type
Research Study
Full title
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
IRAS ID
1011755
Contact name
Andrew Dietz
Contact email
Sponsor organisation
Ensoma, Inc.
Clinicaltrials.gov Identifier
Research summary
This study is investigating a treatment for a rare condition called X-Linked Chronic Granulomatous Disease (X-CGD) which affects peoples immune system and makes it difficult to fight off bacterial and fungal infections. People with this disorder may also have a history of severe infections or chronic/long-term inflammatory disorders which can result in chronic inflammation in the gut or lung scarring and stiffening.
In this study, about 12 adults will take part in dose-escalation. This study will test EN-374 which is a new gene therapy drug that is being developed for treating people with X-CGD. It is not yet approved for treating X-CGD and can only be used in a study like this one.The goal of this study is to see how well people with X-CGD respond to EN 374 treatment. It will also check if the treatment regimen is safe and works well for the people in the study. Another key objective is to find a dose that can be used for more testing. It is hoped that the result of this study would result in less infections and disease.
REC name
South Central - Oxford A Research Ethics Committee
REC reference
25/SC/0165
Date of REC Opinion
5 Nov 2025
REC opinion
Further Information Favourable Opinion