RecruitingPHASE1, PHASE2INTERVENTIONAL

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

This study is looking into a new treatment called EN-374 for a rare inherited condition called X-linked Chronic Granulomatous Disease, or X-CGD. People with X-CGD have trouble fighting off serious infections. The main goals are to find out if EN-374 is safe to use and how well it helps the body produce infection-fighting cells, called neutrophils. Researchers also want to find the right amount of the treatment to give. The study will first test EN-374 in adults and then in children of different age groups. This new treatment is given through a drip into a vein and aims to correct the genetic problem that causes X-CGD.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

Ensoma

Enrolment target

Start

05 Aug 2025

Estimated completion

01 Dec 2027

X-Linked Chronic Granulomatous Disease

What is this study about?

Chronic Granulomatous Disease (CGD) is a rare condition that affects the immune system, which is your body's defence against illness. People with CGD, especially the X-linked type (X-CGD), find it difficult to fight off infections from bacteria and fungi. This means they can get very sick, even from common bugs, and might need strong medicines or long hospital stays. This problem starts early in life because a specific gene, called CYBB, isn't working correctly, which stops certain white blood cells (called neutrophils) from doing their job properly.

This study is testing a new treatment called EN-374. It's a type of 'gene therapy' that aims to fix the root cause of X-CGD. Think of it like this: the treatment uses a harmless virus to deliver a working copy of the CYBB gene into your body's blood stem cells. These stem cells are like master cells that make all your different blood cells, including the infection-fighting neutrophils. By adding the correct gene, the hope is that these new, corrected stem cells will start making healthy, working neutrophils that can fight off infections.

The main purpose of this study is to see if EN-374 is safe and if it helps people with X-CGD make more active neutrophils to better fight infections. They will also look for the best dose to give. The study will start with adults and then carefully include children, starting with older children and moving to younger ones. This phased approach helps ensure the treatment is as safe as possible before it's given to younger patients.

Key takeaways

Tests a new gene therapy (EN-374) for X-linked Chronic Granulomatous Disease (X-CGD).
Aims to improve the body's ability to fight serious infections.
First evaluates safety and effectiveness in adults, then in children.
Involves a drip infusion to correct genetic problem in blood stem cells.
Requires regular clinic visits and monitoring before and after treatment.
Discuss all potential benefits and risks with your doctor.

Who may be eligible?

To be considered for this study, you must be male and have been diagnosed with X-CGD, confirmed by specific genetic tests and showing that your infection-fighting cells aren't working properly.

You would generally need to have had at least one serious infection in the past or a long-lasting inflammatory problem. Importantly, you shouldn't have a suitable family donor for a bone marrow transplant, as that's another treatment option. Adults will be included first, and if the treatment shows promise, it will then be available for children as young as 3 months old, in different age groups.

There are also some reasons why you wouldn't be able to join. For example, if you currently have a severe infection, or if you have certain other serious health conditions like HIV or hepatitis. You also can't have received another gene therapy or an experimental drug recently, and you shouldn't be allergic to any part of the treatment. Participants need to be able to attend all the required appointments and follow the study instructions.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Am I a male with a confirmed diagnosis of X-CGD?
Have I had serious infections or inflammatory problems related to X-CGD?
Do I currently have any serious active infections?
Am I willing to attend all required appointments for the study?
Do I have a suitable family donor for a bone marrow transplant?

Answer every question to see your result.

What does participation involve?

Taking part in this study involves several steps leading up to and after the main treatment. First, you'll have screening tests to make sure you're suitable. Once approved, you'll undergo a process to prepare your body for the gene therapy. This includes taking medicines to help your stem cells move into your bloodstream and other medicines to temporarily reduce your immune system's activity.

The EN-374 treatment itself is given through a drip into a vein (like a blood transfusion). After the treatment, you'll need regular check-ups to monitor your health, how well the treatment is working, and to catch any potential side effects. These check-ups will involve blood tests and other assessments. The study will involve multiple visits to the clinic over a period of time, and you'll be given a schedule for these visits. The total duration of your involvement in the study will be explained in detail by the study team.

Potential risks and benefits

Like any new treatment, EN-374 has potential benefits and potential risks. The main potential benefit is that the gene therapy could help your body produce healthy cells that can properly fight off infections, potentially leading to fewer and less severe illnesses. However, there are also risks, such as side effects from the treatment or the preparation medicines, which could include reactions to the infusion or effects on your blood counts. The study team will monitor you very closely for any problems. It's important to remember that you can choose to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (9)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

University of California, Los Angeles
Verified postcode
Los Angeles, United States· Recruiting
University of California, San Francisco
Verified postcode
San Francisco, United States· Recruiting
Johns Hopkins All Children's Hospital
Verified postcode
St. Petersburg, United States· Recruiting
Boston Children's Hospital
Verified postcode
Boston, United States· Recruiting
University of Minnesota
Verified postcode
Minneapolis, United States· Recruiting
Columbia University Irving Medical Center, Morgan Stanley Children's Hospital
Verified postcode
New York, United States· Recruiting
Duke University
Verified postcode
Durham, United States· Recruiting
University of Utah, Primary Children's Hospital
Verified postcode
Salt Lake City, United States· Recruiting
University College London Hospital
Verified postcode
London, United Kingdom· Recruiting

Common questions

What is X-linked Chronic Granulomatous Disease (X-CGD)?

X-CGD is a rare inherited condition where your immune system struggles to fight off certain infections, making you more prone to serious bacterial and fungal illnesses.

What is EN-374?

EN-374 is a new type of gene therapy being tested. It aims to correct the genetic problem in your body's blood-forming cells so they can properly fight infections.

How is the EN-374 treatment given?

The treatment is given through a drip directly into a vein, like an infusion.

Will this study involve children?

Yes, after initial testing in adults, the study will expand to include children in different age groups, starting from older children down to infants.

What is the main goal of this study?

The main goal is to find out if EN-374 is safe and if it helps people with X-CGD produce healthy infection-fighting cells, as well as to determine the best dose.

How to find out more

Andrew Dietz, MD, MSCR

ddietz@ensoma.com 617-766-3917 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.