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

NCT07498426 | Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: CNIO752A12301
• Study Type: interventional
• Target: 300
• Locations: 10 countries
• Sites: 42

Brief 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.

Conditions

Progressive Supranuclear Palsy Richardson Syndrome (PSP-RS)

Keywords

Progressive Supranuclear Palsy (PSP); NIO752; Anti-Sense Oligonucleotide (ASO); Microtubule Associated Protein Tau (MAPT)

Outcome Measures

Primary Outcomes (1)

• 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.

  Baseline, Week 72

Secondary Outcomes (11)

• Change from baseline in the PSPRS-28 items score

  Baseline, Week 72

• Changes from baseline in activities of daily living on the Cortical Basal ganglia Functional Scale (CBFS)

  From baseline up to week 72

• Change from baseline on the PSP-ShoQoL

  From baseline up to week 72

• Change from baseline in Category (or Semantic) Fluency test over time

  From baseline up to week 72

• Change from baseline in Letter (or Phonemic) Fluency test over time

  From baseline up to week 72

Study Arms (2)

NIO752

EXPERIMENTAL

NIO752 solution

Other: NIO752

Placebo

PLACEBO COMPARATOR

Placebo in solution

Drug: Placebo

Interventions

NIO752 OTHER

Solution of antisense oligonucleotide.

NIO752

Placebo DRUG

Placebo solution

Placebo

Eligibility Criteria

• Age: 41 Years - 81 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (42)

Mayo Clinic Arizona

Scottsdale, Arizona, 85259, United States

RECRUITING

Univ of California San Francisco

San Francisco, California, 94158, United States

RECRUITING

CenExcel Rocky Mtn Clin Research

Englewood, Colorado, 80113, United States

RECRUITING

Mayo Jacksonville

Jacksonville, Florida, 32224, United States

RECRUITING

Mayo Clinic

Rochester, Minnesota, 55905, United States

RECRUITING

Novartis Investigative Site

Westmead, New South Wales, 2145, Australia

RECRUITING

Novartis Investigative Site

Melbourne, Victoria, 3004, Australia

RECRUITING

Novartis Investigative Site

Shanghai, 200040, China

RECRUITING

Novartis Investigative Site

Suzhou, 215000, China

RECRUITING

Novartis Investigative Site

Caen, 14033, France

RECRUITING

Novartis Investigative Site

Créteil, 94010, France

RECRUITING

Novartis Investigative Site

Lille, 59037, France

RECRUITING

Novartis Investigative Site

Marseille, 13885, France

RECRUITING

Novartis Investigative Site

Nantes, 44093, France

RECRUITING

Novartis Investigative Site

Paris, 75013, France

RECRUITING

Novartis Investigative Site

Rennes, 35033, France

RECRUITING

Novartis Investigative Site

Toulouse, 31059, France

RECRUITING

Novartis Investigative Site

Munich, Bavaria, 81377, Germany

RECRUITING

Novartis Investigative Site

Würzburg, Bavaria, 97080, Germany

RECRUITING

Novartis Investigative Site

Bonn, North Rhine-Westphalia, 53127, Germany

RECRUITING

Novartis Investigative Site

Düsseldorf, North Rhine-Westphalia, 40225, Germany

RECRUITING

Novartis Investigative Site

Dresden, Saxony, 01307, Germany

RECRUITING

Novartis Investigative Site

Leipzig, Saxony, 04103, Germany

RECRUITING

Novartis Investigative Site

Beelitz, 14547, Germany

RECRUITING

Novartis Investigative Site

Berlin, 13353, Germany

RECRUITING

Novartis Investigative Site

Stadtroda, 07646, Germany

RECRUITING

Novartis Investigative Site

Tübingen, 72076, Germany

RECRUITING

Klinik für Neurologie (Schwerpunkt Neurodegeneration)

Ulm, 89081, Germany

RECRUITING

Novartis Investigative Site

Milan, MI, 20133, Italy

RECRUITING

Novartis Investigative Site

Roma, RM, 00168, Italy

RECRUITING

Novartis Investigative Site

Kodaira, Tokyo, 187-8551, Japan

RECRUITING

Novartis Investigative Site

Rotterdam, South Holland, 3015 GD, Netherlands

RECRUITING

Novartis Investigative Site

Nijmegen, 6525 GA, Netherlands

RECRUITING

Novartis Investigative Site

Seoul, 03722, South Korea

RECRUITING

Novartis Investigative Site

Pozuelo de Alarcón, Madrid, 28223, Spain

RECRUITING

Novartis Investigative Site

Barcelona, 08036, Spain

RECRUITING

Novartis Investigative Site

Barcelona, 08041, Spain

RECRUITING

Novartis Investigative Site

Las Palmas GC, 35010, Spain

RECRUITING

Novartis Investigative Site

Madrid, 28009, Spain

RECRUITING

Novartis Investigative Site

Madrid, 28034, Spain

RECRUITING

Novartis Investigative Site

Madrid, 28041, Spain

RECRUITING

Novartis Investigative Site

Seville, 41013, Spain

RECRUITING

MeSH Terms

Conditions

Supranuclear Palsy, Progressive; Pick Disease of the Brain

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Ophthalmoplegia; Ocular Motility Disorders; Cranial Nerve Diseases; Tauopathies; Neurodegenerative Diseases; Paralysis; Neurologic Manifestations; Eye Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Frontotemporal Dementia; Frontotemporal Lobar Degeneration; Dementia; Neurocognitive Disorders; Mental Disorders

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

1-888-669-6682; novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

+41613241111

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 23, 2026
• First Posted: March 27, 2026
• Study Start: June 3, 2026
• Primary Completion (Estimated): July 20, 2029
• Study Completion (Estimated): July 18, 2031
• Last Updated: July 29, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will share

Locations

JP (1); NL (2); ES (8); KR (1); CN (2); DE (11); IT (2); AU (2); FR (8); US (5)