A Phase III, randomized, placebo-controlled, parallel group, double-blind study to evaluate the efficacy and safety of NIO752 in participants with Progressive Supranuclear Palsy followed by an Open Label Extension
This research study is looking into a new treatment called NIO752 for people living with Progressive Supranuclear Palsy, specifically a type called Richardson Syndrome. It's a 'Phase III' study, which means it's a large, important trial to confirm if the new medicine works and is safe. Participants will be given either the new medicine or a dummy treatment (a placebo) without knowing which one they are receiving. Researchers will carefully check how participants' symptoms change over 72 weeks using special scores and tests, including daily activities, quality of life, and thinking skills. They will also monitor for any side effects to make sure the treatment is safe. After the main part of the study, everyone will have the chance to receive the new medicine.
نظرة سريعة
ما هو موضوع هذه الدراسة؟
This study is investigating a new medication called NIO752 for a condition known as Progressive Supranuclear Palsy, which is often shortened to PSP. Specifically, it's focusing on a particular form of PSP called Richardson Syndrome. PSP is a rare neurological condition that can affect movement, balance, speech, swallowing, and eye movements. Researchers want to find out if NIO752 can help improve symptoms for people with PSP and if it's a safe treatment option.
The study is designed as a 'Phase III' trial. This means it's a crucial stage in drug development, where the new medicine is tested on a larger group of people to confirm its effectiveness and safety compared to existing treatments or a placebo (a dummy treatment). Participants will be randomly assigned to receive either NIO752 or the placebo. This helps ensure the results are fair and reliable, as neither the participants nor the healthcare team will know who is getting which treatment until the study is over.
Over 72 weeks (about a year and a half), the research team will regularly check how participants are doing. They will use specific scoring systems to measure changes in PSP symptoms, how well people can manage daily activities, and their overall quality of life. They will also look at thinking skills and use special scans, like MRI, to see if there are any changes in the brain. Safety is very important, so they will carefully record any side effects or health problems that might occur during the study.
النقاط الرئيسية
- Tests a new medicine (NIO752) for Progressive Supranuclear Palsy (PSP).
- Compares the new medicine to a dummy treatment (placebo).
- Study lasts for about a year and a half, with regular check-ups.
- Aims to see if the medicine improves symptoms and is safe.
- All participants have the option to receive the active medicine after the main study.
من قد يكون مؤهلاً؟
To join this study, you need to be an adult, 18 years old or older, and there's no upper age limit. The study is open to both men and women.
The most important requirement is that you must have been diagnosed with Progressive Supranuclear Palsy, specifically the type called Richardson Syndrome (PSP-RS).
There will also be other specific medical checks and criteria that the study doctors will go through with you to make sure the study is right and safe for you. These might include checking your overall health and any other medications you are taking.
هل يمكن أن تناسبك هذه الدراسة؟
أجب عن هذه الأسئلة السريعة لترى ما إذا كنت مؤهلاً. هذا دليل فقط - الفريق البحثي هو الذي يتخذ القرار النهائي.
- هل عمرك 18 سنة أو أكثر؟
- Have you been diagnosed with Progressive Supranuclear Palsy (PSP)?
- Have you been diagnosed specifically with Richardson Syndrome (PSP-RS)?
- Are you able to attend regular clinic visits for about 72 weeks?
- Are you willing to potentially receive either the study drug or a placebo?
ماذا يتضمن المشاركة؟
If you decide to take part in this study, you'll be involved for about 72 weeks (around a year and a half). During this time, you'll have regular visits to the clinic where the study team will carry out various assessments. These will include medical check-ups, neurological exams, and tests to see how your symptoms are changing, such as your movement, balance, and thinking skills.
You'll also receive regular doses of the study medication, which will be either NIO752 or a placebo (a dummy treatment that looks like the real medicine but contains no active drug). You won't know which one you're receiving. The study will also involve regular blood tests, vital sign checks (like blood pressure and heart rate), ECGs (heart tracings), and potentially MRI scans of your brain at different points to monitor any changes. After the main 72-week period, there will be an 'Open Label Extension' – this means all participants will then get the chance to receive the active NIO752 medicine if they choose to continue.
المخاطر والفوائد المحتملة
المواقع (6)
- —غير مؤكدهولندا
- —غير مؤكدألمانيا
- —غير مؤكدإسبانيا
- —غير مؤكدإيطاليا
- —غير مؤكدفرنسا
- —غير مؤكدبلجيكا
أسئلة شائعة
What is Progressive Supranuclear Palsy (PSP)?
PSP is a rare brain condition that affects movement, balance, speech, and sometimes thinking abilities.
What does a 'Phase III' study mean?
It's a big study where a new medicine is tested on many people to confirm if it works and is safe, comparing it to a dummy treatment or usual care.
Will I know if I'm getting the medicine or a dummy treatment?
No, this is a 'double-blind' study, meaning neither you nor the study team will know who is getting the active medicine or the dummy treatment.
ما هي المدة التي سأكون مشاركًا فيها في الدراسة؟
The main part of the study lasts for about 72 weeks, which is roughly a year and a half.
What happens after the main study finishes?
After 72 weeks, there's an 'open label' part where all participants can choose to receive the active NIO752 medicine.
كيفية معرفة المزيد
تحدث دائمًا إلى طبيبك العام أو الأخصائي قبل اتخاذ قرار بالمشاركة في دراسة.
نقاش
مناقشة المجتمع
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