A Phase 3 interventional study of NIO752 and Placebo in Progressive Supranuclear Palsy Richardson Syndrome (PSP-RS), sponsored by Novartis Pharmaceuticals. Recruiting at 42 sites in 10 countries. Open to participants aged 41 Years to 81 Years. Per ClinicalTrials.gov, last updated 2026-07-29.
Sponsored by Novartis Pharmaceuticals · Phase 3, Interventional, and Treatment
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.
162 studies on the registry are indexed under Supranuclear Palsy, Progressive; 48 are open to participants now.
This study's planned enrollment of 300 is above the median of 40 across 96 interventional studies indexed under Supranuclear Palsy, Progressive.
Browse Supranuclear Palsy, Progressive studies →Novartis Pharmaceuticals is the lead sponsor of 2,673 studies on the registry; 228 are open to participants now.
Of its 576 completed or terminated interventional studies of FDA-regulated products, 431 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Medical conditions that would, as per Investigator's judgement, prevent the participant from undergoing lumbar puncture, including but not limited to:
Other protocol-defined inclusion/exclusion criteria may apply
NIO752 solution
Other: NIO752
Placebo in solution
Drug: Placebo
Solution of antisense oligonucleotide.
Placebo solution
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.
Time frame: Baseline, Week 72
Change from baseline in the PSPRS-28 items score
28-item Progressive Supranuclear Palsy Rating Scale (PSPRS-28) , scale ranges between 0 and 100 with higher scores indicating greater disability.
Time frame: Baseline, Week 72
Changes from baseline in activities of daily living on the Cortical Basal ganglia Functional Scale (CBFS)
The CBFS is a patient/study partner reported rating scale designed to evaluate the functional impact of 4 repeat tauopathies (4RTs), including PSP. The level of disability assessed by the CBFS correlates with motor, cognitive and psychiatric impairments. The CBFS consists of 14 questions on Motor experiences in daily living (EDL's) and 17 questions on non-Motor EDL's, each rated on a Likert 5-point scale that goes from 0 (Normal/No problems) to 4 (Severe problems).
Time frame: From baseline up to week 72
Change from baseline on the PSP-ShoQoL
The Progressive Supranuclear Palsy - Short version Quality of Life (PSP-ShoQoL) is a patient reported outcome that comprises 12 items, covering phyisical (7 items) and mental health (5 items) states. Items are scored from 0 (no problem) to 4 (extreme problems), total score ranges from 0 to 48 with higher scores indicating greater impact of the disease on the quality of life.
Time frame: From baseline up to week 72
Change from baseline in Category (or Semantic) Fluency test over time
The Category Fluency test is a measure of semantic retrieval and executive functions. It requires the study participant to name as many objects as possible belonging to a given category within one minute. The total score is given by the number of correct words generated within the time limit.
Time frame: From baseline up to week 72
Change from baseline in Letter (or Phonemic) Fluency test over time
The Letter Fluency test is a measure of language and executive functions. It requires the study participant to name as many words as possible starting with a certain letter within one minute. The total score is given by the number of correct words generated within the time limit.
Time frame: From baseline up to week 72
Change from baseline in Symbol Digit Modality Test (SDMT) over time
The SDMT is a measure of attention and processing speed. It requires the study participant to orally pair specific numbers with symbols as quickly and as accurately as possible using a reference key.
Time frame: From baseline up to week 72
Change from baseline in Letter-Number Sequencing task (LNS) over time
The LNS is a sub-test of the Weschler Adult Intelligence Scale - Fourth Edition (WAIS-IV) and it is a measure of working memory and executive function. The study participant is read a sequence of numbers and letters and is required to recall the numbers in ascending order followed by the letters in alphabetical order.
Time frame: From baseline up to week 72
Ratio to baseline of NfL (CSF)
Neurofilament light chain (NfL) is a specific marker of neuroaxonal damage. NfL in CSF will be used to assess the effect of NIO752 on neurodegeneration.
Time frame: From baseline up to week 72
Ratio to baseline of CSF phospho-Tau-181 and CSF Total-Tau
Ratio to baseline in Total -Tau and Phospho-Tau-181 will be used as measures of target engagement
Time frame: From baseline up to week 72
Changes from baseline in volumes of brain structures as measured by MRI
MRI measured volumes will be collected for ventricles, whole brain, midbrain, pons, superior cerebellar peduncle, third ventricle and frontal lobe
Time frame: From baseline up to week 72
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Incidence and severity of AEs and SAEs by treatment group, including clinical laboratory evaluations, vital signs, ECG, C-SSRS qualifying and reported as AEs.
Time frame: From first treatment administration up to 72 weeks
Plan to share: Yes — Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
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