Progressive Supranuclear Palsy Richardson Syndrome (PSP-RS)Phase 3NCT07498426

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

A Phase 3 study testing NIO752 in people with Progressive Supranuclear Palsy Richardson Syndrome (PSP-RS), run by Novartis Pharmaceuticals at 64 sites in 12 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts

Official record

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Trial summary

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. Read more about this study

Who is it for?: Ages 41 to 81 years
Conditions the study is for: Progressive Supranuclear Palsy Richardson Syndrome (PSP-RS)
What is being tested?: NIO752
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 300 participants
Where: 64 sitesin 12 countries
Study dates: 3 Jun 2026to 18 Jul 2031
Phase: Phase 3

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 41 to 81 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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.

Who cannot join

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:

Show all 13Show fewer

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

Trial locations and contacts

64 sites in 12 countries. Contact the study team or a site near you to ask about taking part.

Novartis Pharmaceuticals contact

1-888-669-6682

novartis.email@novartis.com

Novartis Pharmaceuticals contact

+41613241111

Country

City, postcode or hospital

Sites recruiting now only

64 sites in 12 countries

Germany11 sites, 11 recruiting

Novartis Investigative Site
Beelitz, 14547
Recruiting
Novartis Investigative Site
Berlin, 13353
Recruiting
Novartis Investigative Site
Bonn, North Rhine-Westphalia, 53127
Recruiting
Novartis Investigative Site
Dresden, Saxony, 01307
Recruiting
Novartis Investigative Site
Düsseldorf, North Rhine-Westphalia, 40225
Recruiting
Novartis Investigative Site
Leipzig, Saxony, 04103
Recruiting
Novartis Investigative Site
Munich, Bavaria, 81377
Recruiting
Novartis Investigative Site
Stadtroda, 07646
Recruiting
Novartis Investigative Site
Tübingen, 72076
Recruiting
Klinik für Neurologie (Schwerpunkt Neurodegeneration)
Ulm, 89081
Olga Vintonyak, olga.vintonyak@uni-ulm.de
Olga aVintonyak
Recruiting
Novartis Investigative Site
Würzburg, Bavaria, 97080
Recruiting

Other trials in Germany

United States9 sites, 9 recruiting

Parkinsons Disease and Movement Disorders Center of Boca Raton
Boca Raton, Florida, 33486
Lily Turchin, +1 561 392 1818, lturchin@ParkinsonsCenter.org
Stuart H Isaacson
Recruiting
CenExcel Rocky Mtn Clin Research
Englewood, Colorado, 80113
Jessica Crall, +1 303 762 6674, j.crall@cenexel.com
Meagen Salinas
Recruiting
Mayo Jacksonville
Jacksonville, Florida, 32224
Audrey Strongosky, +1 904 953 2254, strongosky.audrey@mayo.edu
Zbigniew K Wszolek
Recruiting
University of Kentucky - C201 Clinic
Lexington, Kentucky, 40536
Lynne Cagle, lynne.cagle@uky.edu
John T Slevin
Recruiting
Ochsner Clinic Foundation
New Orleans, Louisiana, 70121
Anna Leonard-Leumas, anna.leonardleumas@ochsner.org
David Jeffery Houghton
Recruiting
Mayo Clinic
Rochester, Minnesota, 55905
Jeff Laivell, laivell.jeffrey@mayo.edu
James Bower
Recruiting
Central TX Neuro Consultants P A
Round Rock, Texas, 78681
Hadie Mendoza, +1 512 218 1222, hmendoza@dermresearch.net
Elizabeth Peckham
Recruiting
Univ of California San Francisco
San Francisco, California, 94158
Brianna Garcia, brianna.garcia2@ucsf.edu
Lawren Vandevrede
Recruiting
Mayo Clinic Arizona
Scottsdale, Arizona, 85259
Heather Esnaola, +1 480 301 8300, esnaola.heather@mayo.edu
Erika D Driver-Dunckley
Recruiting

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Spain9 sites, 9 recruiting

Novartis Investigative Site
Barcelona, 08036
Recruiting
Novartis Investigative Site
Barcelona, 08041
Recruiting
Novartis Investigative Site
Las Palmas GC, 35010
Recruiting
Novartis Investigative Site
Madrid, 28009
Recruiting
Novartis Investigative Site
Madrid, 28034
Recruiting
Novartis Investigative Site
Madrid, 28041
Recruiting
Novartis Investigative Site
Pozuelo de Alarcón, Madrid, 28223
Recruiting
Novartis Investigative Site
Sant Cugat Del V, Barcelona, 08190
Recruiting
Novartis Investigative Site
Seville, 41013
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France8 sites, 8 recruiting

Novartis Investigative Site
Caen, 14033
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Novartis Investigative Site
Créteil, 94010
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Novartis Investigative Site
Lille, 59037
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Novartis Investigative Site
Marseille, 13885
Recruiting
Novartis Investigative Site
Nantes, 44093
Recruiting
Novartis Investigative Site
Paris, 75013
Recruiting
Novartis Investigative Site
Rennes, 35033
Recruiting
Novartis Investigative Site
Toulouse, 31059
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China7 sites, 7 recruiting

Novartis Investigative Site
Beijing, 100034
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Novartis Investigative Site
Beijing, 100730
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Novartis Investigative Site
Chengdu, Sichuan, 610041
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Novartis Investigative Site
Jinan, 250012
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Novartis Investigative Site
Shanghai, 200040
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Novartis Investigative Site
Suzhou, 215000
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Novartis Investigative Site
Wuhan, 430022
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Italy5 sites, 5 recruiting

Novartis Investigative Site
Genova, GE, 16132
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Novartis Investigative Site
Milan, MI, 20133
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Novartis Investigative Site
Padova, PD, 35128
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Novartis Investigative Site
Roma, RM, 00168
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Novartis Investigative Site
Salerno, SA, 84131
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Japan5 sites, 5 recruiting

Novartis Investigative Site
Bunkyo Ku, Tokyo, 1138431
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Gifu, Gifu, 5011194
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Novartis Investigative Site
Kodaira, Tokyo, 1878551
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Novartis Investigative Site
Nagoya, Aichi-ken, 4650065
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Novartis Investigative Site
Yonago, Tottori, 6838504
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Australia3 sites, 3 recruiting

Novartis Investigative Site
Kogarah, New South Wales, 2217
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Novartis Investigative Site
Melbourne, Victoria, 3004
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Novartis Investigative Site
Westmead, New South Wales, 2145
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Belgium2 sites, 2 recruiting

Novartis Investigative Site
Bruges, 8000
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Novartis Investigative Site
Leuven, Vlaams Brabant, 3000
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Netherlands2 sites, 2 recruiting

Novartis Investigative Site
Nijmegen, 6525 GA
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Novartis Investigative Site
Rotterdam, South Holland, 3015 GD
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United Kingdom2 sites, 2 recruiting

Novartis Investigative Site
Cambridge, CB2 0QQ
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Novartis Investigative Site
London, WC1N 3BG
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South Korea1 site, 1 recruiting

Novartis Investigative Site
Seoul, 03722
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About this study

Official title: 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

Read the detailed description

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.

Conditions: Progressive Supranuclear Palsy Richardson Syndrome (PSP-RS)
Being tested: NIO752, Placebo
Keywords: Progressive Supranuclear Palsy (PSP), NIO752, Anti-Sense Oligonucleotide (ASO), Microtubule Associated Protein Tau (MAPT)
Medical subject headings: Supranuclear Palsy, Progressive, Pick Disease of the Brain

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

NIO752Other
Solution of antisense oligonucleotide.
PlaceboDrug
Placebo solution

Study groups in NCT07498426
Study group	Role	What they receive
NIO752	Experimental	NIO752 solution Receives: Other: NIO752
Placebo	Placebo comparator	Placebo in solution Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Change from baseline in the mPSPRS-10 score
The 10-item Progressive Supranuclear Palsy Rating Scale (mPSPRS-10) is a modified version of the original 28 item PSPRS developed to improve clinical meaningfulness and statistical performance. The 10 items measure three key motor domains: gait, limb function, and bulbar. The mPSPRS-10 ranges between 0 and 30, with higher scores indicating greater disability.
Measured: Baseline, Week 72

Secondary outcomes (11)

Change from baseline in the PSPRS-28 items score
28-item Progressive Supranuclear Palsy Rating Scale (PSPRS-28) , scale ranges between 0 and 100 with higher scores indicating greater disability.
Measured: Baseline, Week 72
Changes from baseline in activities of daily living on the Cortical Basal ganglia Functional Scale (CBFS)
The CBFS is a patient/study partner reported rating scale designed to evaluate the functional impact of 4 repeat tauopathies (4RTs), including PSP. The level of disability assessed by the CBFS correlates with motor, cognitive and psychiatric impairments.
The CBFS consists of 14 questions on Motor experiences in daily living (EDL's) and 17 questions on non-Motor EDL's, each rated on a Likert 5-point scale that goes from 0 (Normal/No problems) to 4 (Severe problems).
Measured: From baseline up to week 72
Change from baseline on the PSP-ShoQoL
The Progressive Supranuclear Palsy - Short version Quality of Life (PSP-ShoQoL) is a patient reported outcome that comprises 12 items, covering phyisical (7 items) and mental health (5 items) states. Items are scored from 0 (no problem) to 4 (extreme problems), total score ranges from 0 to 48 with higher scores indicating greater impact of the disease on the quality of life.
Measured: From baseline up to week 72
Change from baseline in Category (or Semantic) Fluency test over time
The Category Fluency test is a measure of semantic retrieval and executive functions. It requires the study participant to name as many objects as possible belonging to a given category within one minute. The total score is given by the number of correct words generated within the time limit.
Measured: From baseline up to week 72
Change from baseline in Letter (or Phonemic) Fluency test over time
The Letter Fluency test is a measure of language and executive functions. It requires the study participant to name as many words as possible starting with a certain letter within one minute. The total score is given by the number of correct words generated within the time limit.
Measured: From baseline up to week 72
Change from baseline in Symbol Digit Modality Test (SDMT) over time
The SDMT is a measure of attention and processing speed. It requires the study participant to orally pair specific numbers with symbols as quickly and as accurately as possible using a reference key.
Measured: From baseline up to week 72
Change from baseline in Letter-Number Sequencing task (LNS) over time
The LNS is a sub-test of the Weschler Adult Intelligence Scale - Fourth Edition (WAIS-IV) and it is a measure of working memory and executive function. The study participant is read a sequence of numbers and letters and is required to recall the numbers in ascending order followed by the letters in alphabetical order.
Measured: From baseline up to week 72
Ratio to baseline of NfL (CSF)
Neurofilament light chain (NfL) is a specific marker of neuroaxonal damage. NfL in CSF will be used to assess the effect of NIO752 on neurodegeneration.
Measured: From baseline up to week 72
Ratio to baseline of CSF phospho-Tau-181 and CSF Total-Tau
Ratio to baseline in Total -Tau and Phospho-Tau-181 will be used as measures of target engagement
Measured: From baseline up to week 72
Changes from baseline in volumes of brain structures as measured by MRI
MRI measured volumes will be collected for ventricles, whole brain, midbrain, pons, superior cerebellar peduncle, third ventricle and frontal lobe
Measured: From baseline up to week 72
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Incidence and severity of AEs and SAEs by treatment group, including clinical laboratory evaluations, vital signs, ECG, C-SSRS qualifying and reported as AEs.
Measured: From first treatment administration up to 72 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

3 Jun 202618 Jul 2031

First posted27 Mar 2026
Study start3 Jun 2026
Primary completion20 Jul 2029 (estimated)
Study completion18 Jul 2031 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Progressive Supranuclear Palsy Richardson Syndrome (PSP-RS)Studies of the same condition, open and under way.
More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 3
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 300 participants (estimated)

IDs, dates and publications

ClinicalTrials.gov ID: NCT07498426
Sponsor's protocol ID: CNIO752A12301
Status last verified: Oct 2026
First submitted: 23 Mar 2026
Last updated: 15 Oct 2026
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com