Actively Recruiting
Study of EN-374 Gene Therapy in Participants With X-Linked Chronic Granulomatous Disease
Led by Ensoma · Updated on 2026-04-27
15
Participants Needed
9
Research Sites
121 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
CONDITIONS
Official Title
Study of EN-374 Gene Therapy in Participants With X-Linked Chronic Granulomatous Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male
- At least 18 years old during dose escalation, then at least 3 months old during dose expansion
- Diagnosis of X-CGD with DHR+ cells 5% or less and a pathogenic mutation in the CYBB gene
- History of at least one severe infection requiring medical intervention or chronic inflammatory disorder
- Does not have a suitable, available, and willing 10/10 HLA-matched related donor
- Use of highly effective contraception if sexually active with female partners of childbearing potential
- Provided informed consent, with assent from capable participants
- Adequate organ function
You will not qualify if you...
- Active bacteremia or fungemia
- History of HIV, hepatitis B, or hepatitis C
- Medical or social issues likely to increase risk or interfere with study conduct
- History of hematopoietic stem cell transplant or granulocyte transfusions
- Known hypersensitivity to elements in the treatment regimen
- Previous investigational gene therapy
- Treatment with another investigational drug within 30 days before screening
- Unable to comply with study visits and requirements as determined by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 9 locations
1
University of California, Los Angeles
Los Angeles, California, United States, 90095
Actively Recruiting
2
University of California, San Francisco
San Francisco, California, United States, 94158
Actively Recruiting
3
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, United States, 33701
Actively Recruiting
4
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
5
University of Minnesota
Minneapolis, Minnesota, United States, 55454
Actively Recruiting
6
Columbia University Irving Medical Center, Morgan Stanley Children's Hospital
New York, New York, United States, 10032
Actively Recruiting
7
Duke University
Durham, North Carolina, United States, 27710
Actively Recruiting
8
University of Utah, Primary Children's Hospital
Salt Lake City, Utah, United States, 84113
Actively Recruiting
9
University College London Hospital
London, United Kingdom, NW1 2PG
Actively Recruiting
Research Team
A
Andrew Dietz, MD, MSCR
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here