AuthorisedTherapeutic confirmatory (Phase III)Interventional

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.

At a glance

Status

Authorised

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Novartis Pharma AG

Enrolment target

138

Start

21 Apr 2026

Participants with Progressive Supranuclear Palsy Richardson Syndrome (PSP-RS)

What is this study about?

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.

Key takeaways

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.

Who may be eligible?

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.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
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?

Answer every question to see your result.

What does participation involve?

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.

Potential risks and benefits

Taking part in a clinical study can offer potential benefits, such as access to a new treatment before it's widely available and closer monitoring of your health by a specialist team. While NIO752 has shown promise, it's important to remember that it's still being tested, and we don't yet know for sure if it will be effective for everyone or at all. As with any medication, there's a possibility of side effects, which the study team will monitor closely. You will be told about all known potential risks before you decide to join. It's important to remember that participating in any clinical trial is entirely voluntary, and you are free to withdraw at any time for any reason without it affecting your medical care.

Locations (6)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Netherlands
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Unverified
Germany
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Unverified
Spain
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Unverified
Italy
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Unverified
France
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Unverified
Belgium

Common questions

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.

How long will I be involved in the study?

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.