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NCT07498426PHASE3Recruiting

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

Novartis Pharmaceuticals

Start Date

6/3/2026

Completion Date

7/18/2031

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.

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.

Eligibility Criteria

Age Range: 41 years to 81 years

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

Interventions

OTHER

NIO752

DRUG

Placebo

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Conditions

Progressive Supranuclear Palsy Richardson Syndrome (PSP-RS)

Locations

Mayo Clinic Arizona

Scottsdale, Arizona 85259

United States

Univ of California San Francisco

San Francisco, California 94158

United States

CenExcel Rocky Mtn Clin Research

Englewood, Colorado 80113

United States

Mayo Jacksonville

Jacksonville, Florida 32224

United States

Mayo Clinic

Rochester, Minnesota 55905

United States

Novartis Investigative Site

Westmead, New South Wales 2145

Australia

Novartis Investigative Site

Melbourne, Victoria 3004

Australia

Novartis Investigative Site

Shanghai, 200040

China

Novartis Investigative Site

Suzhou, 215000

China

Novartis Investigative Site

Caen, 14033

France

Novartis Investigative Site

Créteil, 94010

France

Novartis Investigative Site

Lille, 59037

France

Novartis Investigative Site

Marseille, 13885

France

Novartis Investigative Site

Nantes, 44093

France

Novartis Investigative Site

Paris, 75013

France

Novartis Investigative Site

Rennes, 35033

France

Novartis Investigative Site

Toulouse, 31059

France

Novartis Investigative Site

Munich, Bavaria 81377

Germany

Novartis Investigative Site

Würzburg, Bavaria 97080

Germany

Novartis Investigative Site

Bonn, North Rhine-Westphalia 53127

Germany

Novartis Investigative Site

Düsseldorf, North Rhine-Westphalia 40225

Germany

Novartis Investigative Site

Dresden, Saxony 01307

Germany

Novartis Investigative Site

Leipzig, Saxony 04103

Germany

Novartis Investigative Site

Beelitz, 14547

Germany

Novartis Investigative Site

Berlin, 13353

Germany

Novartis Investigative Site

Stadtroda, 07646

Germany

Novartis Investigative Site

Tübingen, 72076

Germany

Klinik für Neurologie (Schwerpunkt Neurodegeneration)

Ulm, 89081

Germany

Novartis Investigative Site

Milan, MI 20133

Italy

Novartis Investigative Site

Roma, RM 00168

Italy

Novartis Investigative Site

Kodaira, Tokyo 187-8551

Japan

Novartis Investigative Site

Rotterdam, South Holland 3015 GD

Netherlands

Novartis Investigative Site

Nijmegen, 6525 GA

Netherlands

Novartis Investigative Site

Seoul, 03722

South Korea

Novartis Investigative Site

Pozuelo de Alarcón, Madrid 28223

Spain

Novartis Investigative Site

Barcelona, 08036

Spain

Novartis Investigative Site

Barcelona, 08041

Spain

Novartis Investigative Site

Las Palmas GC, 35010

Spain

Novartis Investigative Site

Madrid, 28009

Spain

Novartis Investigative Site

Madrid, 28034

Spain

Novartis Investigative Site

Madrid, 28041

Spain

Novartis Investigative Site

Seville, 41013

Spain