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
Inclusion Criteria8
- 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
Exclusion Criteria8
- Active bacteremia or fungemia
- History of human immunodeficiency virus (HIV), hepatitis B, or hepatitis C
- History or clinical evidence of any medical or social issues likely to put the participant at additional risk or to interfere with study conduct
- History of HSCT or granulocyte transfusions
- Known hypersensitivity to elements in the treatment regimen
- Undergone investigational gene therapy
- Treated with another investigational drug product within 30 days before screening
- Unable to comply with the visits and requirements of the protocol as determined by the Investigator
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Interventions
Single dose of EN-374 administered by intravenous infusion after mobilization and followed by enrichment
Locations(9)
View Full Details on ClinicalTrials.gov
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NCT06876363