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Clinical Trials in China / NCT07498426
Recruiting Phase 3

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

NCT07498426 · tracked via the Priya Life Science China tracker
Sponsor
Novartis Pharmaceuticals
Phase
Phase 3
Started
2026-06-03
Last updated
2026-07-29

Condition(s) studied

Progressive Supranuclear Palsy Richardson Syndrome (PSP-RS)

Investigational drug(s) / intervention(s)

NIO752Placebo

NIO752: Solution of antisense oligonucleotide.

Placebo: Placebo solution

Study 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.

Eligibility

Sex
ALL
Min age
41 Years
Max age
81 Years
Healthy volunteers
No
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

Primary outcome measure(s)

Trial sites (42)

FacilityCityRegionStatus
Mayo Clinic Arizona Scottsdale Arizona Recruiting
Univ of California San Francisco San Francisco California Recruiting
CenExcel Rocky Mtn Clin Research Englewood Colorado Recruiting
Mayo Jacksonville Jacksonville Florida Recruiting
Mayo Clinic Rochester Minnesota Recruiting
Novartis Investigative Site Westmead New South Wales Recruiting
Novartis Investigative Site Melbourne Victoria Recruiting
Novartis Investigative Site Shanghai China Recruiting
Novartis Investigative Site Suzhou 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 Recruiting
Novartis Investigative Site Würzburg Bavaria Recruiting
Novartis Investigative Site Bonn North Rhine-Westphalia Recruiting
Novartis Investigative Site Düsseldorf North Rhine-Westphalia Recruiting
Novartis Investigative Site Dresden Saxony Recruiting
Novartis Investigative Site Leipzig Saxony 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 Milan MI Recruiting
Novartis Investigative Site Roma RM Recruiting
Novartis Investigative Site Kodaira Tokyo Recruiting
Novartis Investigative Site Rotterdam South Holland Recruiting
Novartis Investigative Site Nijmegen Netherlands Recruiting
Novartis Investigative Site Seoul South Korea Recruiting
Novartis Investigative Site Pozuelo de Alarcón Madrid Recruiting
Novartis Investigative Site Barcelona Spain Recruiting
Novartis Investigative Site Barcelona Spain Recruiting
Novartis Investigative Site Las Palmas GC Spain Recruiting
Novartis Investigative Site Madrid Spain Recruiting
Novartis Investigative Site Madrid Spain Recruiting

+ 2 more sites — see the full list on the official registry below.

More Novartis Pharmaceuticals trials in China

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07498426 on ClinicalTrials.gov ↗ ← All trials in China