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EN-374 Gene Therapy for X-Linked Chronic Granulomatous Disease

This study is testing a gene therapy called EN-374 for X-linked Chronic Granulomatous Disease (X-CGD), a rare immune disorder. EN-374 aims to help your body produce healthy immune cells by correcting a faulty gene. The main goals are to check how safe EN-374 is and if it helps your immune cells work better. You might be able to join if you are male, at least 3 months old, have X-CGD with specific lab results (DHR+ cells ≤ 5% and a CYBB gene mutation), and a history of severe infections or chronic inflammation. The study will first enroll adult men, then younger males. The current recruitment status is unclear.

Study design
This is an interventional study with a planned enrollment of 15 participants. It will first enroll adults for dose escalation, then children for dose expansion.
What's involved
You would receive a single dose of EN-374 given through an IV after a process to prepare your stem cells.
Compensation
Not stated in the trial record.
Follow-up
Your safety will be monitored from the start of mobilization until Month 12.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06876363

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

Active, Not Recruiting
PHASE1Ages 3+InterventionalTreatment
Ensoma
~15 participants
Updated 2026-08-21 on ClinicalTrials.gov
What's tested:EN-374

At a glance

Recruiting sites
0 of 9 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Safety of EN-374
Measured over From start of mobilization until Month 12

Conditions

X-Linked Chronic Granulomatous Disease

Explore related trials

X-linked Chronic Granulomatous DiseaseCYBB GeneDHFR Gene

NCT06876363

Where you'd take part

This study runs at 9 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Boston Children's Hospital

    Boston, Massachusettsno site contact published

  • Columbia University Irving Medical Center, Morgan Stanley Children's Hospital

    New York, New Yorkno site contact published

  • Duke University

    Durham, North Carolinano site contact published

  • Johns Hopkins All Children's Hospital

    St. Petersburg, Floridano site contact published

  • University College London Hospital

    London, United Kingdomno site contact published

  • University of California, Los Angeles

    Los Angeles, Californiano site contact published

  • University of California, San Francisco

    San Francisco, Californiano site contact published

  • University of Minnesota

    Minneapolis, Minnesotano site contact published

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

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Eligibility criteria

Inclusion

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

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

What this trial measures

  • Safety of EN-374From 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)