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
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Ages | 41 Years to 81 Years |
| Sex | All |
| Enrollment | 300 (estimated) |
| Sponsor | Novartis Industry-sponsored |
| Locations | 64 sites (Scottsdale, Arizona and 63 other locations) |
| Status | Recruiting |
| Started | June 3, 2026 |
| Main results expected | July 20, 2029 |
| Study ends | July 18, 2031 |
| Registry record updated | October 15, 2026 |
| Trial ID | NCT07498426 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
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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
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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 -
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Chengdu, Sichuan, China Recruiting -
Novartis Investigative Site
Beijing, China Recruiting -
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Beijing, China Recruiting -
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Jinan, China Recruiting -
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Shanghai, China Recruiting -
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Suzhou, China Recruiting -
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Wuhan, China Recruiting -
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Caen, France Recruiting -
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Créteil, France Recruiting -
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Lille, France Recruiting -
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Marseille, France Recruiting -
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Nantes, France Recruiting -
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Paris, France Recruiting -
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Rennes, France Recruiting -
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Toulouse, France Recruiting -
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Munich, Bavaria, Germany Recruiting -
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Würzburg, Bavaria, Germany Recruiting -
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Bonn, North Rhine-Westphalia, Germany Recruiting -
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Düsseldorf, North Rhine-Westphalia, Germany Recruiting -
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Dresden, Saxony, Germany Recruiting -
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Leipzig, Saxony, Germany Recruiting -
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Beelitz, Germany Recruiting -
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Berlin, Germany Recruiting -
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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 -
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Padova, Pd, Italy Recruiting -
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Roma, Rm, Italy Recruiting -
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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
- Study coordinator: Novartis Pharmaceuticals
- Email: novartis.email@novartis.com
- Phone: 1-888-669-6682
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.