En öppen, enarmad, fas 1/2 först-i-människa-studie för att bedöma säkerheten och effektiviteten av autologa CD34+ celler transducerade med en lentiviral vektor som kodar för den mänskliga NCF1-genen (SGX-001) hos pediatriska och vuxna patienter med kronisk granulomatös sjukdom orsakad av p47phox-brist
This research is a first-in-human study for a new treatment, SGX-001, for chronic granulomatous disease (CGD) caused by a p47phox deficiency. CGD is a condition where the immune system struggles to fight certain infections. The study will look at how safe SGX-001 is and how well it works. This involves taking special cells from a patient, changing them in a lab, and then putting them back. Doctors will closely monitor patients for a year after treatment to check for any side effects and see if their immune system improves. The goal is to help patients lead healthier lives and reduce infections.
Överblick
Vad handlar denna studie om?
This study is about a new way to treat a rare immune system condition called Chronic Granulomatous Disease (CGD). Specifically, it's for people whose CGD is caused by a problem with a protein called p47phox. In CGD, certain immune cells, called phagocytes, don't work properly to kill germs, making people very prone to serious infections.
The new treatment, called SGX-001, is a type of gene therapy. It involves taking some of your own blood stem cells (special cells that can turn into different types of blood cells), adding a working copy of the gene that makes the p47phox protein in a laboratory, and then giving these corrected cells back to you. The idea is that these new, fixed cells will start producing healthy immune cells that can fight off infections effectively.
Because this is a 'first-in-human' study, it's designed to carefully check the safety of SGX-001 first, and then to see if it actually improves health. Doctors will be looking at many things, like how well your immune system starts to work, if you get fewer infections, and if your quality of life improves. It's a key step in understanding if this new approach can become a real treatment option for people with CGD.
Viktiga punkter
- This study tests a new gene therapy (SGX-001) for a specific type of CGD.
- It's a 'first-in-human' study, focusing on safety and how well the treatment works.
- The treatment aims to correct immune cells to fight infections better.
- Participation involves cell collection, treatment, and regular check-ups for at least a year.
- It's for children and adults with CGD caused by p47phox deficiency.
- Potential for reducing reliance on other CGD medications.
Vem kan vara berättigad?
This study is looking for both children and adults who have Chronic Granulomatous Disease (CGD) specifically caused by a problem with the p47phox protein. There isn't an upper age limit mentioned, so adults of any age might be considered.
While the study aims to include a wide range of patients with this specific condition, other health factors will be carefully checked. For instance, doctors will need to make sure you're healthy enough to safely receive the treatment and undergo all the necessary tests and follow-up appointments. Both males and females can take part.
Kan denna studie passa dig?
Svara på dessa snabba frågor för att se om du kan vara berättigad. Detta är endast en vägledning — forskarteamet fattar det slutgiltiga beslutet.
- Do I have Chronic Granulomatous Disease (CGD)?
- Has my doctor told me my CGD is caused by a 'p47phox deficiency'?
- Am I generally well enough to undergo a medical treatment and regular check-ups?
- Am I comfortable with regular hospital visits and monitoring for at least a year?
Vad innebär deltagande?
If you decide to join this study, you would first have health checks to make sure you're suitable. The treatment involves taking some of your own stem cells, which will then be specially prepared in a lab. After that, you'll receive the treated cells back into your body. This usually means a stay in the hospital.
After receiving the treatment, you'll have regular hospital visits for at least 12 months (one year). During these visits, the doctors and nurses will do various checks. This includes blood tests, measuring your heart rate and blood pressure, checking your heart with an ECG (a simple heart tracing), and general health check-ups. They will closely monitor your immune system, how you're feeling, any side effects, and if the treatment is helping to reduce your infections. You might also have bone marrow tests to see how the new cells are doing.
Potentiella risker och fördelar
Platser (2)
- —ObekräftadTyskland
- —ObekräftadSpanien
Vanliga frågor
What is Chronic Granulomatous Disease (CGD)?
CGD is a rare inherited condition where your immune system's infection-fighting cells don't work properly, making you very vulnerable to serious infections, especially from certain bacteria and fungi.
What is SGX-001?
SGX-001 is an investigational gene therapy. It aims to correct the genetic fault in your own stem cells so they can produce proper infection-fighting immune cells.
What does 'first-in-human' mean?
It means this is one of the very first times this specific treatment is being tested in people. Because of this, patient safety is the highest priority, and you'll be very closely monitored.
Hur länge kommer jag att vara involverad i studien?
You'll be closely monitored for at least 12 months (one year) after receiving the treatment, with regular check-ups and tests.
Will I still need my usual medicines if I take part?
The study will also look at whether participants can reduce or stop other medicines they currently take for CGD, like antibiotics or antifungals, but this will be carefully assessed by the doctors involved.
Hur du får reda på mer
Prata alltid med din läkare eller specialist innan du bestämmer dig för att delta i en studie.
Diskussion
Gemenskapsdiskussion
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