Clinical trial Recruiting at 64 sites Phase 3 Interventional Ages 41 to 81

NIO752 treatment for people with Progressive Supranuclear Palsy Richardson syndrome.

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

Run by Novartis · NCT07498426 · Data updated 2026-11-12

This trial will try NIO752 versus placebo in adults with mild-to-moderate PSP-RS to see if it slows symptom progression.

Quick facts

PhasePhase 3
Study typeInterventional
Ages41 Years to 81 Years
SexAll
Enrollment300 (estimated)
SponsorNovartis
Industry-sponsored
Locations64 sites (Scottsdale, Arizona and 63 other locations)
StatusRecruiting
StartedJune 3, 2026
Main results expectedJuly 20, 2029
Study endsJuly 18, 2031
Registry record updatedOctober 15, 2026
Trial IDNCT07498426 on ClinicalTrials.gov

Is this trial a fit for you?

Likely a good fit

Adults aged 41–81 with probable or possible PSP Richardson syndrome of under 5 years' duration, PSPRS <40, MMSE ≥20, able to take at least 10 steps (with minimal assistance if needed), and who have a reliable study partner are the intended participants.

Probably not a fit

People with more advanced PSP (longer disease duration or higher PSPRS), significant cognitive impairment (MMSE <20), inability to ambulate, or those outside the age range are unlikely to qualify or benefit from this trial.

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a randomized, double-blind, placebo-controlled, parallel-group Phase 3 trial that assigns participants in a 2:1 ratio to NIO752 or placebo, followed by an open-label extension where participants can continue NIO752. NIO752 is an antisense oligonucleotide designed to reduce MAPT (tau) expression, with the goal of slowing neurodegeneration. Eligible participants are adults 41–81 years old with probable/possible PSP-RS of less than 5 years' duration, a PSPRS score under 40, MMSE ≥20, ability to walk at least 10 steps (with minimal assistance if needed), and a reliable study partner. The study includes regular clinical visits and informant-based assessments to measure changes in function, motor signs, and cognition over the double-blind period and extension.

Why it matters

Potential benefit: If successful, NIO752 could slow disease progression and help preserve function and daily abilities in people with PSP-RS.

How similar studies have gone: Tau-targeting antisense approaches are relatively novel in PSP and have shown limited or mixed results in early-phase studies, so clinical benefit remains unproven to date.

Who can join: the full rules

Show full inclusion / exclusion criteria
Inclusion Criteria:

1. Signed informed consent must be obtained prior to participation in the study.
2. Male or female participants, age between 41-81 yrs inclusive.
3. Diagnosis of mild-moderate, probable/possible PSP Richardson syndrome as per MDS-PSP 2017 criteria with symptoms onset \< 5 years.
4. PSPRS total score less than 40 at Baseline.
5. 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.
6. 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).
7. Mini Mental State Examination (MMSE) score ≥ 20 at Screening.

Exclusion Criteria:

1. 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.
2. Diagnosis of amyotrophic lateral sclerosis or other motor neuron diseases.
3. Diagnosis of cerebellar ataxia, choreoathetosis, and early symptomatic autonomic dysfunction.
4. 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).
5. Contraindications to undergo MRI procedure, including metal (ferromagnetic) implants and/or a cardiac pacemaker that is not compatible with MRI.
6. Medical conditions that would, as per Investigator's judgement, prevent the participant from undergoing lumbar puncture, including but not limited to:

   1. Known allergy to local anesthetic
   2. History of back surgery (with the exception of microdiscectomy or laminectomy over 1 level)
   3. Spinal deformities
   4. Current dermatological infection at the lumbar puncture spot and/or significant skin alterations at the planned puncture place
   5. 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).
7. 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

Where it's running

  • Mayo Clinic Arizona
    Scottsdale, Arizona, United States
    Recruiting
  • Univ of California San Francisco
    San Francisco, California, United States
    Recruiting
  • CenExcel Rocky Mtn Clin Research
    Englewood, Colorado, United States
    Recruiting
  • Parkinsons Disease and Movement Disorders Center of Boca Raton
    Boca Raton, Florida, United States
    Recruiting
  • Mayo Jacksonville
    Jacksonville, Florida, United States
    Recruiting
  • University of Kentucky - C201 Clinic
    Lexington, Kentucky, United States
    Recruiting
Show 44 more locations
  • Ochsner Clinic Foundation
    New Orleans, Louisiana, United States
    Recruiting
  • Mayo Clinic
    Rochester, Minnesota, United States
    Recruiting
  • Central TX Neuro Consultants P A
    Round Rock, Texas, United States
    Recruiting
  • Novartis Investigative Site
    Kogarah, New South Wales, Australia
    Recruiting
  • Novartis Investigative Site
    Westmead, New South Wales, Australia
    Recruiting
  • Novartis Investigative Site
    Melbourne, Victoria, Australia
    Recruiting
  • Novartis Investigative Site
    Leuven, Vlaams Brabant, Belgium
    Recruiting
  • Novartis Investigative Site
    Bruges, Belgium
    Recruiting
  • Novartis Investigative Site
    Chengdu, Sichuan, China
    Recruiting
  • Novartis Investigative Site
    Beijing, China
    Recruiting
  • Novartis Investigative Site
    Beijing, China
    Recruiting
  • Novartis Investigative Site
    Jinan, China
    Recruiting
  • Novartis Investigative Site
    Shanghai, China
    Recruiting
  • Novartis Investigative Site
    Suzhou, China
    Recruiting
  • Novartis Investigative Site
    Wuhan, China
    Recruiting
  • Novartis Investigative Site
    Caen, France
    Recruiting
  • Novartis Investigative Site
    Créteil, France
    Recruiting
  • Novartis Investigative Site
    Lille, France
    Recruiting
  • Novartis Investigative Site
    Marseille, France
    Recruiting
  • Novartis Investigative Site
    Nantes, France
    Recruiting
  • Novartis Investigative Site
    Paris, France
    Recruiting
  • Novartis Investigative Site
    Rennes, France
    Recruiting
  • Novartis Investigative Site
    Toulouse, France
    Recruiting
  • Novartis Investigative Site
    Munich, Bavaria, Germany
    Recruiting
  • Novartis Investigative Site
    Würzburg, Bavaria, Germany
    Recruiting
  • Novartis Investigative Site
    Bonn, North Rhine-Westphalia, Germany
    Recruiting
  • Novartis Investigative Site
    Düsseldorf, North Rhine-Westphalia, Germany
    Recruiting
  • Novartis Investigative Site
    Dresden, Saxony, Germany
    Recruiting
  • Novartis Investigative Site
    Leipzig, Saxony, Germany
    Recruiting
  • Novartis Investigative Site
    Beelitz, Germany
    Recruiting
  • Novartis Investigative Site
    Berlin, Germany
    Recruiting
  • Novartis Investigative Site
    Stadtroda, Germany
    Recruiting
  • Novartis Investigative Site
    Tübingen, Germany
    Recruiting
  • Klinik für Neurologie (Schwerpunkt Neurodegeneration)
    Ulm, Germany
    Recruiting
  • Novartis Investigative Site
    Genova, Ge, Italy
    Recruiting
  • Novartis Investigative Site
    Milan, Mi, Italy
    Recruiting
  • Novartis Investigative Site
    Padova, Pd, Italy
    Recruiting
  • Novartis Investigative Site
    Roma, Rm, Italy
    Recruiting
  • Novartis Investigative Site
    Salerno, Sa, Italy
    Recruiting
  • Novartis Investigative Site
    Nagoya, Aichi-ken, Japan
    Recruiting
  • Novartis Investigative Site
    Gifu, Gifu, Japan
    Recruiting
  • Novartis Investigative Site
    Bunkyo Ku, Tokyo, Japan
    Recruiting
  • Novartis Investigative Site
    Kodaira, Tokyo, Japan
    Recruiting
  • Novartis Investigative Site
    Yonago, Tottori, Japan
    Recruiting

+14 more sites. See ClinicalTrials.gov for the full list.

Study contacts

How to take part

  1. Review the eligibility criteria above with your treating physician.
  2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
  3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.
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Last reviewed 2026-11-14 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.