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Gene therapy aims to fix immune system in rare disease

NCT ID NCT06876363

Disease control Sponsor: Ensoma Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 21, 2026 · Updated 1 time

Summary

This study tests a one-time gene therapy called EN-374 for people with X-linked chronic granulomatous disease (X-CGD), a rare immune disorder that causes severe infections. The treatment uses a modified virus to deliver a working gene to blood stem cells, aiming to restore the ability of white blood cells to kill germs. The study will enroll about 15 males aged 3 months and older, and will check safety and whether the therapy increases functional immune cells.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 15 people

The number the study aims to enrol. It can still change while the study runs.

Started

Aug 2025

Expected to finish

Dec 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

3 months and older

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

chronic granulomatous disease granulomatous disease, chronic, X-linked

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

X-linked chronic granulomatous disease

Contacts and locations

Locations

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

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

    New York, New York, 10032, United States

  • Duke University

    Durham, North Carolina, 27710, United States

  • Johns Hopkins All Children's Hospital

    St. Petersburg, Florida, 33701, United States

  • University College London Hospital

    London, NW1 2PG, United Kingdom

  • University of California, Los Angeles

    Los Angeles, California, 90095, United States

  • University of California, San Francisco

    San Francisco, California, 94158, United States

  • University of Minnesota

    Minneapolis, Minnesota, 55454, United States

  • University of Utah, Primary Children's Hospital

    Salt Lake City, Utah, 84113, United States

More trials for these conditions

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