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Active, not recruiting Phase 1/2

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

NCT06876363 · tracked via the Priya Life Science UK tracker
Sponsor
Ensoma
Phase
Phase 1/2
Started
2025-08-05
Last updated
2026-08-21

Condition(s) studied

X-Linked Chronic Granulomatous Disease

Investigational drug(s) / intervention(s)

EN-374

EN-374: Single dose of EN-374 administered by intravenous infusion after mobilization and followed by enrichment

Study 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

Sex
MALE
Min age
3 Months
Max age
—
Healthy volunteers
No
Inclusion Criteria: * Male * ≥ 18 years of age during dose escalation, then ≥ 3 months of age during dose expansion * Diagnosis of X-CGD with 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 Criteria: * 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

Primary outcome measure(s)

  • Safety of EN-374 — From start of mobilization until Month 12
    Incidence rate across all age groups of: * treatment-emergent adverse events (TEAEs) * treatment-related TEAEs (TRAEs) * serious adverse events (SAEs)

Trial sites (9)

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

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06876363 on ClinicalTrials.gov ↗ ← All trials in the UK