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.
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¿De qué trata este estudio?
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.
Puntos clave
- 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.
¿Quién puede ser elegible?
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.
¿Podría este estudio ser adecuado para ti?
Responde estas preguntas rápidas para ver si puedes ser elegible. Esto es solo una guía: el equipo de investigación toma la decisión final.
- 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?
¿En qué consiste la participación?
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.
Riesgos y beneficios potenciales
Locations (9)
- University of California, Los AngelesCódigo postal verificadoLos Angeles, United States
- University of California, San FranciscoCódigo postal verificadoSan Francisco, Estados Unidos
- Johns Hopkins All Children's HospitalCódigo postal verificadoSt. Petersburg, United States
- Boston Children's HospitalCódigo postal verificadoBoston, Estados Unidos
- University of MinnesotaCódigo postal verificadoMinneapolis, United States
- Columbia University Irving Medical Center, Morgan Stanley Children's HospitalCódigo postal verificadoNueva York, Estados Unidos
- Duke UniversityCódigo postal verificadoDurham, Estados Unidos
- University of Utah, Primary Children's HospitalCódigo postal verificadoSalt Lake City, Estados Unidos
- Hospital Universitario College de LondresCódigo postal verificadoLondres, Reino Unido
Preguntas comunes
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.
¿Cuál es el objetivo principal de este estudio?
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.
Cómo obtener más información
Siempre hable con su médico de cabecera o especialista antes de decidir participar en un estudio.
Discusión
Discusión comunitaria
Impulsado por nuestro foro en community.patient.info. Por favor, sea respetuoso — esto no es un consejo médico.