Study of EN-374 Gene Therapy in Participants With X-Linked Chronic Granulomatous Disease

Recruiting now · Phase 1/Phase 2

Conditions studied: X-Linked Chronic Granulomatous Disease

In brief

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

Key facts

Study ID

NCT06876363

Run by

Ensoma

People needed

15

Starts

2025-08-05

Expected to finish

2027-12-01

Last updated by the study team

2026-04-27

Who can join

Age: 0 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• University of California, Los Angeles — Los Angeles, California, United States (enrolling)
• University of California, San Francisco — San Francisco, California, United States (enrolling)
• Johns Hopkins All Children's Hospital — St. Petersburg, Florida, United States (enrolling)
• Boston Children's Hospital — Boston, Massachusetts, United States (enrolling)
• University of Minnesota — Minneapolis, Minnesota, United States (enrolling)
• Columbia University Irving Medical Center, Morgan Stanley Children's Hospital — New York, New York, United States (enrolling)
• Duke University — Durham, North Carolina, United States (enrolling)
• University of Utah, Primary Children's Hospital — Salt Lake City, Utah, United States (enrolling)
• University College London Hospital — London, United Kingdom (enrolling)

Full record on ClinicalTrials.gov

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