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

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What is this study about?

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.

Who Can Join the Study?

  • 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 Study?

  • 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.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Fondazione Policlinico Universitario Agostino Gemelli IRCCS Rome Italy
Centre Hospitalier Universitaire De Lille Lille France
Oncopole Claudius Regaud Toulouse France
Technische Universitaet Dresden Dresden Germany
Katholieke Universiteit te Leuven Leuven Belgium

Other Sites

Site Name City Country Status
Hospital Universitari General De Catalunya Sant Cugat Del Valles Spain
Kliniken Beelitz GmbH Beelitz Germany
Centre Hospitalier Universitaire De Nimes Nimes France
Hospital General Universitario Gregorio Maranon Madrid Spain
Hospital Universitario 12 De Octubre Madrid Spain
El Hospital Universitario De Gran Canaria Dr. Negrin Las Palmas De Gran Canaria Spain
Azienda Ospedaliera Universitaria San Giovanni Di Dio E Ruggi d’Aragona Salerno Italy
Deutsches Zentrum Fuer Neurodegenerative Erkrankungen e.V. Bonn Germany
Azienda Ospedaliera di Padova Padua Italy
Hospital Universitario Ramon Y Cajal Madrid Spain
Virgen del Rocío University Hospital Sevilla Spain
Universitaetsklinikum Tuebingen AöR Tuebingen Germany
Hospital Clinic De Barcelona Barcelona Spain
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Az St-Jan Brugge-Oostende A.V. Brugge Belgium
Stichting Radboud University Medical Center Nijmegen The Netherlands
IRCCS Ospedale Policlinico San Martino Genoa Italy
Hospital Universitario Quironsalud Madrid Pozuelo De Alarcon Spain
Centre Hospitalier Universitaire De Montpellier Montpellier France
Centre Hospitalier Universitaire De Nantes Nantes France
Centre Hospitalier Universitaire De Rennes Rennes France
Institut fuer Klinische Transfusionsmedizin und Immungenetik Ulm gGmbH Ulm Germany
Centre Hospitalier Lyon Sud Pierre Benite France
Hopital Beaujon Clichy France
Fondazione I.R.C.C.S. Istituto Neurologico Besta Milan Italy
Centre Hospitalier Universitaire De Caen Normandie Caen France
Universitaetsklinikum Leipzig AöR Leipzig Germany
Azjkdtosdc Pyzbqzmz Hnuqkstx Dw Muxddlywe Marseille France
Upmnflbubzvxtqfbnjtyw Dhjlsotscru Azd Duesseldorf Germany
Edkpnni Uxfouaayrapd Msulogw Cljtnmb Rcwehcywm (ofyxcco Mug Rotterdam The Netherlands
Kgglbzqu dhx Usuhamkkaois Mzuxrzhk Atk Munich Germany
Ujohkhnubkyllzjueapoz Wkerjeltm Acr Wuerzburg Germany
Hlmppgws Dr Lx Sqzzf Chhk I Svhe Plr Barcelona Spain
Aapctuayl Fhsajnbjtbre Siwzlrkyq Gspf Stadtroda Germany
Hesoyede Ucedsolqqcykho Sxjzphkqbv &nghqos Hcbtvnw db Hcgajukrecc STRASBOURG, Alsace France

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Belgium Belgium
Not yet recruiting
04.06.2026
France France
Not yet recruiting
04.06.2026
Germany Germany
Not yet recruiting
04.06.2026
Italy Italy
Not yet recruiting
04.06.2026
Spain Spain
Not yet recruiting
04.06.2026
The Netherlands The Netherlands
Not yet recruiting
04.06.2026

Trial locations

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.

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 ID:
2025-523481-24-00
Protocol code:
CNIO752A12301
Trial Phase:
Therapeutic confirmatory (Phase III)

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