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 genetic condition called X-linked chronic granulomatous disease (X-CGD). X-CGD makes people, usually males, very vulnerable to severe infections. EN-374 is a type of gene therapy designed to help the body fight off these infections better. Researchers will first test EN-374 in adult men with X-CGD to check its safety and how well different doses work. Once that's complete, the study will then include children with X-CGD. The goal is to learn if this new treatment can improve the health of people living with X-CGD and provide valuable information for future patients.
Em resumo
Sobre o que é este estudo?
This study is about a new treatment called EN-374 for a rare health condition known as X-linked chronic granulomatous disease, often shortened to X-CGD. People with X-CGD have trouble fighting off certain bacteria and fungi, leading to severe and frequent infections, often starting very early in life. This happens because of a change in a specific gene called CYBB. Currently, there isn't a simple cure for X-CGD, so new treatments are really important.
EN-374 is what's called a 'gene therapy.' Think of it like a special delivery system designed to introduce a working copy of the missing or faulty CYBB gene into your body's cells. The idea is that with this working gene, your body will be better equipped to fight off infections. This particular therapy uses a modified, harmless virus to carry the new gene, targeting certain cells that are key to your immune system. This could potentially offer a long-term solution or significantly improve the lives of those with X-CGD.
This is a 'Phase I/II' study, which means it's one of the first times this treatment is being tested in humans. The main goals are to find out if EN-374 is safe, how much to give, and if it shows any signs of working to improve X-CGD. They will start by testing it in adult men with X-CGD, and once they understand the safety and dose in adults, they will then open the study to children. The information gathered from this study will be crucial for developing better treatments for X-CGD in the future.
Pontos principais
- Tests a new gene therapy (EN-374) for X-linked chronic granulomatous disease (X-CGD).
- Aims to replace a faulty gene to help the body fight infections.
- First recruiting adult men, then children with X-CGD.
- It's a Phase I/II study, focusing on safety and early signs of effectiveness.
- Participation involves IV treatment, other medications, and regular check-ups.
- Potential benefits for participants are not guaranteed, but data will help future patients.
Quem pode ser elegível?
The study is currently looking for adult men aged 18 and over who have been diagnosed with X-CGD. You would need to have specific blood test results that show your immune cells aren't working properly because of the X-CGD, and a history of serious infections or ongoing inflammation related to your condition. Importantly, you shouldn't have a suitable sibling donor for a more traditional bone marrow transplant.
There are also some reasons why you might not be able to join. For instance, if you have active infections, or a history of certain viruses like HIV or Hepatitis B/C, you wouldn't be able to take part. Also, if you've had a bone marrow transplant before, or received other experimental gene therapies, you might not be eligible. The team will also check for other health issues or medications that could make it unsafe for you to join.
If you're a man with X-CGD and you're interested, the study team will go through all the detailed requirements with you to see if you're a good fit. They'll also make sure you understand everything before you decide to take part.
Este estudo pode ser adequado para você?
Responda a estas perguntas rápidas para ver se você pode ser elegível. Este é apenas um guia — a decisão final é da equipe de pesquisa.
- Você é do sexo masculino?
- Você tem pelo menos 18 anos?
- Have you been diagnosed with X-CGD?
- Do you have a history of serious infections or ongoing inflammation?
- Have you NOT had a bone marrow transplant in the past?
- Are you able to use effective contraception if needed?
O que envolve a participação?
If you decide to take part in this study, you would first have a number of tests to check if you are suitable. Once confirmed, you would receive the study treatment, EN-374, which is given directly into a vein as an infusion. Before this, you would have some other treatments to prepare your body, including medicine to help release special stem cells into your bloodstream, and medicines to help reduce your immune system's response to the treatment.
The study involves regular visits to the clinic for check-ups and tests. These will include blood tests, physical examinations, and other assessments to monitor your health and see how your body is responding to EN-374 and to the various other medicines. The entire study is expected to run from August 2025 until December 2027, and you would be involved for a significant part of this time, with ongoing follow-up to track your long-term health and the effects of the treatment.
Riscos e benefícios potenciais
Localizações (1)
- University College LondonAproximadoLondon, England
Perguntas frequentes
What is X-linked chronic granulomatous disease (X-CGD)?
It's a rare inherited condition that affects the immune system, making people very prone to severe infections, particularly in males.
O que é terapia genética?
Gene therapy is a new type of treatment that aims to fix faulty genes or replace missing ones in your body's cells to treat a disease.
Who can join the study right now?
Currently, the study is open to adult men (18 years and older) who have X-CGD and meet other specific health requirements.
Eu definitivamente vou melhorar se eu participar?
It's important to know that this is a research study, and we don't know for certain if EN-374 will help. Your health might improve, stay the same, or get worse.
Onde este estudo está ocorrendo?
The study is being run from Catalyst Clinical Research in the UK.
Como saber mais
Allan Robinson
Sempre consulte seu médico ou especialista antes de decidir participar de um estudo.
Interessado em participar?
Discussão
Discussão da comunidade
Desenvolvido pelo nosso fórum em community.patient.info. Por favor, seja respeitoso — isto não é um conselho médico.