NCT07498426

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

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

• Status: Not Yet Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 300 (estimated)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 41 Years – 81 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Progressive Supranuclear Palsy Richardson Syndrome (PSP-RS)

Interventions

Type	Name	Description
OTHER	NIO752	Solution of antisense oligonucleotide.
DRUG	Placebo	Placebo solution

Timeline

• Start date: 2026-05-29
• Primary completion: 2029-07-20
• Completion: 2031-07-18
• First posted: 2026-03-27
• Last updated: 2026-03-27

Regulatory

• FDA-regulated drug study