Last Update: Jul 29, 2026 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 ClinicalTrials.gov Identifier: [NCT07498426](https://clinicaltrials.gov/ct2/show/NCT07498426) Novartis Reference Number:CNIO752A12301 [See if you Pre-qualify](#trial-eligibility "See if you Pre-qualify") All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. ## Study Description 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. 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. Condition Progressive Supranuclear Palsy Richardson Syndrome (PSP-RS) Phase Phase3 Overall Status Recruiting Number of Participants300 Start Date Jun 03, 2026 Completion Date Jul 18, 2031 Gender All Age(s) 41 Years - 81 Years (Adult, Older Adult) ## Interventions Other ### NIO752 Solution of antisense oligonucleotide. Drug ### Placebo Placebo solution ## Eligibility 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 Australia #### Novartis Investigative Site Recruiting Westmead,New South Wales,2145,Australia #### Novartis Investigative Site Recruiting Melbourne,Victoria,3004,Australia China #### Novartis Investigative Site Recruiting Shanghai,200040,China #### Novartis Investigative Site Recruiting Suzhou,215000,China France #### Novartis Investigative Site Recruiting Lille,59037,France #### Novartis Investigative Site Recruiting Marseille,13885,France #### Novartis Investigative Site Recruiting Nantes,44093,France #### Novartis Investigative Site Recruiting Paris,75013,France #### Novartis Investigative Site Recruiting Caen,14033,France #### Novartis Investigative Site Recruiting Rennes,35033,France #### Novartis Investigative Site Recruiting Créteil,94010,France #### Novartis Investigative Site Recruiting Toulouse,31059,France Germany #### Klinik für Neurologie (Schwerpunkt Neurodegeneration) Recruiting Ulm,89081,Germany ###### Olga aVintonyak ###### Olga Vintonyak Email: #### Novartis Investigative Site Recruiting Munich,Bavaria,81377,Germany #### Novartis Investigative Site Recruiting Würzburg,Bavaria,97080,Germany #### Novartis Investigative Site Recruiting Düsseldorf,North Rhine-Westphalia,40225,Germany #### Novartis Investigative Site Recruiting Leipzig,Saxony,04103,Germany #### Novartis Investigative Site Recruiting Berlin,13353,Germany #### Novartis Investigative Site Recruiting Stadtroda,07646,Germany #### Novartis Investigative Site Recruiting Tübingen,72076,Germany #### Novartis Investigative Site Recruiting Beelitz,14547,Germany #### Novartis Investigative Site Recruiting Dresden,Saxony,01307,Germany #### Novartis Investigative Site Recruiting Bonn,North Rhine-Westphalia,53127,Germany Italy #### Novartis Investigative Site Recruiting Roma,RM,00168,Italy #### Novartis Investigative Site Recruiting Milan,MI,20133,Italy Japan #### Novartis Investigative Site Recruiting Kodaira,Tokyo,187-8551,Japan Netherlands #### Novartis Investigative Site Recruiting Rotterdam,South Holland,3015 gd,Netherlands #### Novartis Investigative Site Recruiting Nijmegen,6525 ga,Netherlands South Korea #### Novartis Investigative Site Recruiting Seoul,03722,South Korea Spain #### Novartis Investigative Site Recruiting Madrid,28041,Spain #### Novartis Investigative Site Recruiting Pozuelo de Alarcón,Madrid,28223,Spain #### Novartis Investigative Site Recruiting Barcelona,08041,Spain #### Novartis Investigative Site Recruiting Madrid,28034,Spain #### Novartis Investigative Site Recruiting Madrid,28009,Spain #### Novartis Investigative Site Recruiting Seville,41013,Spain #### Novartis Investigative Site Recruiting Las Palmas GC,35010,Spain #### Novartis Investigative Site Recruiting Barcelona,08036,Spain United States #### Mayo Clinic Arizona Recruiting Scottsdale,Arizona,85259,United States ###### Heather Esnaola Phone: [480-301-8300](tel:480-301-8300) Email: ###### Erika D Driver-Dunckley #### Mayo Jacksonville Recruiting Jacksonville,Florida,32224,United States ###### Zbigniew K Wszolek ###### Audrey Strongosky Phone: [904-953-2254](tel:904-953-2254) Email: #### Univ of California San Francisco Recruiting San Francisco,California,94158,United States ###### Lawren Vandevrede ###### Brianna Garcia Email: #### Mayo Clinic Recruiting Rochester,Minnesota,55905,United States ###### Jeff Laivell Email: ###### James Bower #### CenExcel Rocky Mtn Clin Research Recruiting Englewood,Colorado,80113,United States ###### Meagen Salinas ###### Jessica Crall Phone: [303-762-6674](tel:303-762-6674) Email: ## Worldwide Contacts If the location of your choosing does not feature any contact detail, please reach out using the information below. #### Novartis Pharmaceuticals Phone: [ +41613241111](tel:+41613241111) Email: [](mailto:) #### Novartis Pharmaceuticals Phone: [ 1-888-669-6682](tel:1-888-669-6682) Email: