Prevent Cognitive Decline in GBA-associated Parkinson's Disease
This research study is looking at whether a new medicine, prasinezumab, can help to prevent or slow down memory and thinking difficulties in people with Parkinson's disease. This is for individuals who have a particular genetic change in their GBA gene. Participants will receive monthly infusions of either prasinezumab or a placebo (a dummy medicine) for about two years. The study aims to recruit 120 people aged 35 to 80 across several European countries, including the UK. It’s a 'proof-of-concept' trial, meaning it's an early stage study to see if the treatment works before larger trials are planned.
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¿De qué trata este estudio?
This study is about a new medicine called prasinezumab. Researchers want to find out if it can help slow down or prevent problems with memory and thinking in people who have Parkinson's disease. This is particularly for those who have a specific type of genetic change in a gene called GBA. This GBA gene change is thought to play a role in how Parkinson's affects the brain, especially memory and thinking skills.
The medicine, prasinezumab, is designed to target certain proteins in the brain that are believed to build up in Parkinson's disease. The study is called a 'proof-of-concept' trial, which means it's an early step to see if the medicine has a positive effect. If it does, then more extensive studies would be carried out in the future.
Over two years, participants in the study will receive either the active medicine or a placebo (a harmless, inactive substance that looks just like the medicine). This helps researchers compare the effects accurately. The study will involve 120 people from various European countries, including the UK, to understand if this medicine could make a difference for people with Parkinson's and this specific genetic change.
Puntos clave
- This study tests a new medicine for memory/thinking problems in Parkinson's.
- It's for people with Parkinson's who have a specific GBA gene change.
- Participation involves monthly infusions for about two years.
- You might receive the active medicine or a placebo (dummy treatment).
- It's an early-stage study to see if the treatment works (proof-of-concept).
¿Quién puede ser elegible?
To join this study, you would need to be between 35 and 80 years old and have a diagnosis of Parkinson's disease. A key requirement is that you must carry a specific genetic change in your GBA gene. Your doctors will be able to confirm if you have this gene change through a blood test.
Your memory and thinking abilities will also be checked to make sure they fall within certain levels needed for the study. You also shouldn't have very advanced Parkinson's symptoms that affect your movement significantly while on your usual medication.
You would not be able to join if you have other specific genetic changes linked to Parkinson's, or if your doctors believe you have a different type of Parkinson's-like condition. Having certain brain conditions, previous brain surgery like Deep Brain Stimulation, or other serious medical issues that could interfere with the study would also prevent you from taking part.
¿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.
- Are you between 35 and 80 years old?
- Do you have a diagnosis of Parkinson's disease?
- Has your doctor confirmed you have a specific genetic change in your GBA gene?
- Do you have good overall memory and thinking skills, checked by your doctor?
- Have you not had Deep Brain Stimulation or similar brain surgery?
- Do you not have other specific types of Parkinson’s or certain other severe medical conditions?
¿En qué consiste la participación?
If you decide to take part, you would receive monthly infusions through a drip, which would last for about two years (104 weeks). During these infusions, you would either get the study medicine, prasinezumab, or a placebo (a dummy treatment).
Throughout the study, you would come for regular appointments where doctors would check your health, memory, and thinking skills. They would also monitor how your Parkinson's disease is progressing. These appointments would help the researchers understand if the study medicine is having any effect and to keep an eye on your safety. After the two years of infusions, there would likely be some follow-up visits to see how you are doing.
Riesgos y beneficios potenciales
Locations (11)
- Sorbonne University, Pitié-Salpêtrière HospitalCódigo postal verificadoParís, Francia· Aún no reclutando
- University Hospital C. G. Carus TU DresdenCódigo postal verificadoDresde, Alemania· Aún no reclutando
- University Hospital TubingenCódigo postal verificadoTübingen, Alemania· Reclutamiento
- University of Brescia and ASST Spedali Civili of BresciaCódigo postal verificadoBrescia, Italy· Aún no reclutando
- Fondazione IRCCS Ca' Granda Policlinico HospitalCódigo postal verificadoMilan, Italy· Aún no reclutando
- University of PaviaCódigo postal verificadoPavía, Italia· Aún no reclutando
- University of LuxemburgCódigo postal verificadoLuxembourg, Luxembourg· Aún no reclutando
- Hospital Universitario HM Puerta del Sur, MadridCódigo postal verificadoMadrid, España· Aún no reclutando
- Hospital Universitario Virgen del Rocío, SevillaCódigo postal verificadoSeville, Spain· Aún no reclutando
- Karolinska University of StockholmCódigo postal verificadoStockholm, Sweden· Aún no reclutando
- Universidad de OxfordCódigo postal verificadoOxford, Reino Unido· Aún no reclutando
Preguntas comunes
¿Qué es un 'placebo'?
A placebo is a 'dummy' treatment that looks just like the real medicine but contains no active ingredients. It helps researchers compare the effects of the active medicine accurately.
What does 'GBA gene mutation' mean?
It means you have a specific change in your GBA gene. This gene is important for how your cells work, and changes in it are linked to a higher risk of Parkinson's and sometimes memory problems.
¿Sabré si estoy recibiendo el medicamento activo o el placebo?
No, neither you nor your study doctor will know. This is called 'double-blind' and it helps ensure the study results are unbiased.
What's the difference between a Phase 2 and other phases of trials?
Phase 2 trials are early studies focusing on whether a treatment works, what dose to use, and checking for side effects, usually in a smaller group of people. If successful, it moves to larger Phase 3 trials.
What if I decide I don't want to continue in the study?
You are free to leave the study at any time, for any reason. Your decision will not affect your relationship with your healthcare team or your usual care.
Cómo obtener más información
Kathrin Brockmann, Prof. Dr.
Siempre hable con su médico de cabecera o especialista antes de decidir participar en un estudio.
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Impulsado por nuestro foro en community.patient.info. Por favor, sea respetuoso — esto no es un consejo médico.