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A study to evaluate the safety and effectiveness of NIO752 in patients with progressive supranuclear palsy

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on individuals living with Progressive Supranuclear Palsy Richardson Syndrome, a rare brain disorder that affects movement, balance, and eye movements. The purpose of the study is to evaluate the effectiveness and safety of a drug called NIO752 compared to a placebo, which is a substance containing sodium chloride. This medication is administered through intrathecal use, meaning it is delivered directly into the fluid surrounding the brain and spinal cord.

During the first part of the study, participants are randomly assigned to receive either NIO752 or the placebo. The study is double-blind, which means neither the participants nor the researchers know which substance is being administered. Throughout this period, various measurements are taken to monitor changes in movement, daily activities, and quality of life. Additionally, an MRI, a specialized imaging test that uses magnets to create detailed pictures of the brain, is used to look at different brain structures.

Following the initial phase, there is an open label extension, where participants may have the opportunity to receive the active medication. The study also monitors safety by tracking any side effects or changes in vital signs and electrocardiogram results, which is a test that records the electrical activity of the heart.

The research process

The trial runs in 3 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    <b>initial phase</b>

    Participation begins in a double-blind study, meaning neither the participant nor the medical staff knows whether the test medication or a placebo (an inactive substance) is being administered.

    The participant is assigned to receive either NIO752, which is a concentrate for solution for injection, or a placebo consisting of sodium chloride (a salt solution).

    The medication is administered via intrathecal use, which means it is delivered into the fluid-filled space around the spinal cord.

  2. Step 2

    <b>monitoring and evaluation</b>

    Over a period of 72 weeks, progress is tracked using various scales to measure movement, daily activities, quality of life, and mental functions such as memory and processing speed.

    magnetic resonance imaging (mri), a type of detailed medical scan, is used to monitor changes in specific areas of the brain and its fluid-filled spaces.

    Safety is monitored through regular checks of vital signs, electrocardiograms (ecg), and clinical laboratory evaluations to detect any adverse events or side effects.

  3. Step 3

    <b>open label extension</b>

    Following the completion of the 72-week period, the study moves into an open label extension.

    During this stage, the blinded nature of the study is removed, meaning it is known which medication is being used.

Who can join the trial?

8 criteria

  • You must provide a signed informed consent, which is a written document showing that you understand the study and agree to take part.
  • You must be a male or female between the ages of 41 and 81 years old.
  • You must have a diagnosis of Progressive Supranuclear Palsy Richardson Syndrome, a condition that affects movement and balance, which is classified as mild to moderate.
  • Your symptoms must have started less than 5 years ago.
  • Your PSPRS total score, which is a specific scale used to measure the severity of your symptoms, must be less than 40 at the start of the study.
  • You must have a reliable study partner, such as a spouse, sibling, friend, or caregiver, who can provide accurate information about your health and spend at least 5 hours per week with you.
  • You must be able to ambulate, which means you are able to walk at least 10 steps on your own or with very little help.
  • Your MMSE score, a quick test used to check your memory and thinking skills, must be 20 or higher.

Who cannot join the trial?

10 criteria

  • Having other major brain or mental health conditions, such as Parkinson’s disease (a movement disorder), Alzheimer’s disease (a type of memory loss), dementia with Lewy bodies (a condition affecting thinking and movement), prion disease (a rare brain disorder), or any psychotic disorders (conditions that change how you perceive reality).
  • Having severe Major depressive disorder (a serious form of long-term sadness and loss of interest), seizures (sudden electrical disturbances in the brain), or a brain tumor (an abnormal growth in the brain).
  • Having a space-occupying lesion, which is any growth or mass in the brain that takes up space and may cause pressure.
  • A history of a clinically significant stroke, which is a sudden loss of brain function due to blood flow problems that results in permanent neurological deficit (long-lasting damage to brain functions like movement or speech).
  • A history of a head injury that caused you to lose consciousness for at least 15 minutes within the last 20 years.
  • A diagnosis of amyotrophic lateral sclerosis (a disease that affects the nerves controlling muscles) or other motor neuron diseases (disorders that damage the cells responsible for muscle movement).
  • A diagnosis of cerebellar ataxia (a lack of muscle coordination), choreoathetosis (uncontrollable, jerky, or twisting movements), or early symptomatic autonomic dysfunction (problems with the body systems that work automatically, like heart rate or digestion).
  • An MRI scan (a medical imaging test using magnets) that shows significant brain abnormalities, such as a hemorrhage (bleeding in the brain), an infarct (an area of dead tissue caused by lack of blood supply), a cerebral contusion (a bruise on the brain), an aneurysm (a bulge in a blood vessel), a vascular malformation (an abnormal connection between blood vessels), a subdural hematoma (a collection of blood between the brain and its outer covering), or hydrocephalus (an accumulation of fluid in the brain).
  • Being unable to undergo an MRI due to having ferromagnetic implants (metal objects in the body that are attracted to magnets) or a cardiac pacemaker (a device implanted in the chest to control the heartbeat) that is not safe to use inside the MRI machine.
  • A history of deep brain stimulator surgery, which is a procedure where wires are placed in the brain to control tremors or movement, unless it was a sham surgery (a fake surgery used in studies where no device is actually implanted) for a different clinical trial.
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Investigated drugs

NIO752 is an experimental treatment that is injected into the fluid surrounding the spinal cord to study how well it can slow down the progression of Progressive Supranuclear Palsy.

What is already known about the treatment

  • NIO752

    This medication is an experimental drug provided as a concentrated liquid that is injected directly into the spinal fluid. Currently being studied in advanced clinical trials, it is being tested to see if it can slow down the progression of a rare brain disorder called Progressive Supranuclear Palsy. It works at a molecular level by targeting specific biological processes to help protect nerve cells from damage. This substance is classified as an investigational orphan drug for treating nervous system diseases.

  • Sodium chloride

    This substance is a common salt solution administered through an injection into the spinal fluid to serve as a placebo in this study. It is a widely used, well-established medical tool used to maintain the balance of fluids and electrolytes in the body. In this context, it has no active therapeutic effect on the disease being studied and acts as a control to compare against the test drug. It is pharmacologically classified as an electrolyte.

Investigated diseases

Progressive Supranuclear Palsy Richardson Syndrome - This is a rare brain disorder that affects movement and balance. It involves the gradual loss of nerve cells in specific areas of the brain. Over time, it typically causes difficulties with eye movements and posture. People may experience increased stiffness in their muscles and challenges with walking. The condition often leads to a decline in the ability to perform daily activities.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2025-523481-24-00Protocol codeCNIO752A12301Estimated enrolment287 patientsSponsorNovartis Pharma AG

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