Safety and Efficacy of a Unilateral Subretinal Administration of HORA PDE6B in Patients with Retinitis Pigmentosa Harbouring Mutations in the PDE6B Gene Leading to a Defect in PDE6ß Expression
This early-stage study is for adults with a type of retinitis pigmentosa caused by changes in the PDE6B gene. It involves a new gene therapy given as a single injection into the back of one eye. Researchers want to make sure the treatment is safe and to see if it can help improve or save vision. They will carefully monitor participants, checking their eye health, vision, and overall well-being. This is one of the first times this treatment is being tried in humans, so it's a very important step in developing new ways to help people with this condition.
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¿De qué trata este estudio?
This study is looking for better ways to treat a condition called retinitis pigmentosa, which causes loss of vision and can eventually lead to blindness. This particular study focuses on people whose retinitis pigmentosa is caused by a specific problem with a gene called PDE6B. This gene normally helps your eyes work properly.
Researchers are testing a new treatment called gene therapy. This therapy aims to put a working copy of the PDE6B gene into the cells at the back of the eye. The hope is that this new gene will help these cells work better and slow down or stop further vision loss. Because this is an early study, the main goal is to make sure the treatment is safe for people, but they will also be looking to see if it has any positive effects on vision.
Patients taking part will receive the treatment in only one eye. This allows doctors to compare how the treated eye does compared to the untreated eye, giving them valuable information about the treatment's effects. It's a very important step in understanding if this type of gene therapy could become a real option for people with this condition in the future.
Puntos clave
- This study is for adults with retinitis pigmentosa caused by a specific PDE6B gene problem.
- It's testing a new gene therapy given as an injection into one eye.
- The main goals are to check the treatment's safety and see if it helps vision.
- You will have many eye and general health checks during the study.
- This is an early-stage study, so potential benefits and risks will be discussed carefully.
¿Quién puede ser elegible?
This study is for adults generally aged 18 and older. It doesn't matter if you are male or female; anyone can take part if they meet the other requirements.
The most important requirement is that your retinitis pigmentosa must be caused by a specific problem with your PDE6B gene. The doctors will need to confirm this with genetic testing.
There will also be other medical checks to make sure you are healthy enough for the treatment and any procedures involved in the study.
¿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.
- ¿Tienes 18 años o más?
- Has a genetic test confirmed your retinitis pigmentosa is caused by a PDE6B gene mutation?
- Are you able to attend regular clinic appointments?
- Are you interested in a new treatment that is still in early development?
- Do you understand the commitment involved in a research study?
¿En qué consiste la participación?
If you join this study, you will have a number of visits to the clinic involving eye exams and medical checks. You will receive the treatment, which is a one-time injection into the back of one eye. Around this time, you'll also be given other eye drops and an eye implant to help with healing and reduce inflammation.
Throughout the study, doctors will closely monitor your safety with regular eye checks, blood tests, and measurements of your general health like blood pressure. They will also do various tests to check your vision, such as how well you see up close and far away, your colour vision, and your peripheral (side) vision. Some special eye scans will also be done to look at the back of your eye. You might also be asked to fill out questionnaires about your quality of life.
The total duration of your involvement in the study will be decided by the research team, but studies like this usually involve follow-up visits over several months or even a few years.
Riesgos y beneficios potenciales
Ubicaciones (1)
- —No verificadoFrancia
Preguntas comunes
¿Qué es la retinitis pigmentosa?
It's a group of eye conditions that damage the light-sensing cells at the back of your eye, leading to gradual loss of vision, often starting with night blindness and side vision.
What does 'gene therapy' mean?
Gene therapy aims to fix health problems by replacing a faulty gene with a healthy one. In this study, it's about putting a good copy of the PDE6B gene into your eye cells.
Why is the treatment only given in one eye?
Giving the treatment in one eye allows doctors to compare it to your untreated eye. This helps them understand the treatment's effects more clearly and safely.
Will this study cure my retinitis pigmentosa?
This is an early-stage study primarily focused on safety. While they hope to see vision improvements, it's too soon to say if it will be a cure. It's an important step toward finding new treatments.
¿Qué tipo de pruebas tendré?
You'll have regular eye check-ups, vision tests, scans of the back of your eye, and overall health checks like blood tests. You might also fill out questionnaires about how you're feeling.
Cómo obtener más información
Siempre hable con su médico de cabecera o especialista antes de decidir participar en un estudio.
¿Interesado en participar?
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Impulsado por nuestro foro en community.patient.info. Por favor, sea respetuoso — esto no es un consejo médico.