ClinicalTrials.gov record NCT07498426

A Study to Evaluate the Efficacy of NIO752 in Participants With Progressive Supranuclear Palsy

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Aug 25, 2026

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

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 3A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.
Age41 years old to 81 years old
SexAll
LocationAustralia, China, France, Germany, Italy, Japan, Netherlands, South Korea, Spain, United States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase III, randomized, double-blind, placebo-controlled study tests NIO752 in people with Progressive Supranuclear Palsy Richardson syndrome. Participants are randomized 2:1 to receive NIO752 or placebo in a parallel design and are followed through the core study (primary measures to week 72); an open-label extension is offered after the core period. The study collects clinical rating scales, cognitive tests, CSF biomarkers, MRI measurements, and safety data.

What it is studying. The study is testing the effects of NIO752 compared with placebo on disease-related outcomes in PSP-RS. The primary measurement is change from baseline in the 10-item modified PSP Rating Scale (mPSPRS-10) between baseline and Week 72. Secondary measurements include the full 28-item PSPRS, activities of daily living (CBFS), quality of life (PSP-ShoQoL), several cognitive tests, CSF biomarkers (including NfL and tau species), MRI volume changes, and rates of adverse events and serious adverse events.

What the researchers hope to learn. Scientifically, the study aims to evaluate whether NIO752 changes clinical disability scores, cognitive and functional measures, CSF biomarkers of neurodegeneration and tau, MRI brain volumes, and to characterize safety and adverse events.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

This Phase III study is intended to evaluate the efficacy and safety of NIO752 in participants with Progressive Supranuclear Palsy (PSP). Eligible participants will be randomized to receive either NIO752 or placebo followed by an open-label extension.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Placebo
Other
  • NIO752

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • Signed informed consent must be obtained prior to participation in the study.
  • Male or female participants, age between 41-81 yrs inclusive.
  • Diagnosis of mild-moderate, probable/possible PSP Richardson syndrome as per MDS-PSP 2017 criteria with symptoms onset < 5 years.
  • PSPRS total score less than 40 at Baseline.
  • Reliable study partner such as spouse, sibling, close friend, or caregiver able and willing to provide accurate information (including clinical symptoms and medical history) about the participant and to participate in study visits and informant-based assessments for the duration of the study. A reliable study partner is expected to spend enough time (at least 5 hours per week) with the study participant.
  • Participant is able to ambulate defined as the ability to take at least 10 steps independently or with minimal assistance (stabilization of one arm to minimize fall risk).
  • Mini Mental State Examination (MMSE) score ≥ 20 at Screening.

Exclusion criteria

  • Diagnosis of other significant neurological or psychiatric disorders including (but not limited to) Parkinsons' Disease (which has not subsequently been revised to a diagnosis of PSP); Alzheimer's disease (AD), dementia with Lewy bodies; prion disease; any psychotic disorders; severe Major depressive disorder; seizure; brain tumor or other space-occupying lesion; history of clinically significant stroke (e.g., stroke with permanent neurological deficit); history of head injury with loss of consciousness for at least 15 minutes within the past 20 years.
  • Diagnosis of amyotrophic lateral sclerosis or other motor neuron diseases.
  • Diagnosis of cerebellar ataxia, choreoathetosis, and early symptomatic autonomic dysfunction.
  • History of or screening brain MRI scan indicative of significant abnormality, including, but not limited to, prior hemorrhage or infarct >1 cm3, >3 lacunar infarcts, cerebral contusion, aneurysm, vascular malformation >1 cm3, subdural hematoma, hydrocephalus, and space-occupying lesion (e.g., abscess or brain tumor).
  • Contraindications to undergo MRI procedure, including metal (ferromagnetic) implants and/or a cardiac pacemaker that is not compatible with MRI.
  • Medical conditions that would, as per Investigator's judgement, prevent the participant from undergoing lumbar puncture, including but not limited to:
  • Known allergy to local anesthetic
  • History of back surgery (with the exception of microdiscectomy or laminectomy over 1 level)
  • Spinal deformities
  • Current dermatological infection at the lumbar puncture spot and/or significant skin alterations at the planned puncture place
  • Risk of increased or uncontrolled bleeding and/or risk of bleeding that if not managed optimally, could place a participant at an increased risk for procedural bleeding. These could include, but are not limited, to anatomical factors at or near the LP site (e.g., vascular abnormalities, neoplasms) and underlying disorders of coagulation, platelet function or platelet count (e.g. abnormal coagulation parameters, hemophilia, Von Willebrand's disease, liver disease).
  • History of deep brain stimulator surgery other than sham surgery for participation in a deep brain stimulation clinical trial.
  • Other protocol-defined inclusion/exclusion criteria may apply

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

This is a randomized, double-blind, placebo-controlled, parallel group, study to evaluate the efficacy of NIO752 in participants with Progressive Supranuclear Palsy followed by an open-label extension (OLE). The participants with or without symptomatic therapy will be randomly allocated to either NIO752 or placebo treatment in a 2:1 randomization ratio. Upon completion of the core double-blind treatment period, participants will be offered to continue with NIO752 treatment in the OLE.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants are randomly assigned (2:1) to receive either NIO752 or placebo in a double-blind core treatment period and may be offered NIO752 in a subsequent open‑label extension. During the study they will attend regular clinic visits for motor and cognitive assessments, questionnaires about daily function and quality of life, and safety checks; brain MRI scans and lumbar punctures (spinal taps) are performed for biomarker and imaging assessments. Safety monitoring (laboratory tests, ECG, and adverse-event reporting) is part of participation. A reliable study partner must attend visits and provide informant-based information throughout the study.

How the study is structured. Participants are randomly assigned (2:1) to receive either NIO752 or matching placebo in a parallel group, double-blind format (participants, care providers, investigators, and outcomes assessors masked). This is a Phase III interventional drug trial with a core blinded period through Week 72 and an optional open-label extension where participants may receive NIO752.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
  • The listed sites are in Australia, China, France, Germany, Italy, Japan, Netherlands, South Korea, Spain, United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on progressive supranuclear palsy

Clinical trials45 trials listed for progressive supranuclear palsyEvery trial the registry lists for progressive supranuclear palsy, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in progressive supranuclear palsy researchLatest monthly update: September 2026. Includes: PSP vs Parkinsons. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Jun 3, 2026
Primary completion
Jul 20, 2029 (estimated)
Study completion
Jul 18, 2031 (estimated)
Target enrollment
300 participants (estimated)
Last updated on registry
Jul 29, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Novartis Pharmaceuticals

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (42)

  • Mayo Clinic ArizonaRecruiting

    Scottsdale, Arizona, 85259 · United States

  • Univ of California San FranciscoRecruiting

    San Francisco, California, 94158 · United States

  • CenExcel Rocky Mtn Clin ResearchRecruiting

    Englewood, Colorado, 80113 · United States

  • Mayo JacksonvilleRecruiting

    Jacksonville, Florida, 32224 · United States

  • Mayo ClinicRecruiting

    Rochester, Minnesota, 55905 · United States

  • Novartis Investigative SiteRecruiting

    Westmead, New South Wales, 2145 · Australia

  • Novartis Investigative SiteRecruiting

    Melbourne, Victoria, 3004 · Australia

  • Novartis Investigative SiteRecruiting

    Shanghai, 200040 · China

  • Novartis Investigative SiteRecruiting

    Suzhou, 215000 · China

  • Novartis Investigative SiteRecruiting

    Caen, 14033 · France

Terms used on this page · explanations by MedicalUpdates.ai

randomized
Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
placebo
A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.
open-label extension
A continuation study in which participants who completed an earlier study can keep receiving the treatment, usually with everyone knowing what they are given.
informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
Baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
at Screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
MRI
A scan that uses magnets and radio waves, not X-rays, to make detailed pictures of the inside of the body. Some implants and metal objects mean a person cannot have one.
Contraindications
A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
lumbar puncture
A procedure in which a needle is placed between the bones of the lower back to collect spinal fluid or to give a treatment.
sham
A procedure designed to resemble the real procedure, for example the same visit and equipment, without its active part. It is the procedure equivalent of a placebo.
exclusion criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
double-blind
Two types of people involved in the study do not know which intervention a participant is receiving. The ClinicalTrials.gov record specifies who is masked, for example participants, care providers, investigators or outcome assessors.
placebo-controlled
A study design in which one group receives a placebo so that the study treatment's effects can be compared with no active treatment.
parallel group
Participants are divided into two or more groups, and each group receives a different intervention at the same time, so the groups can be compared.
2:1
How many participants go to each group. In a 2:1 ratio, two are assigned to the first group for every one assigned to the second.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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