NCT04539041

Safety, Tolerability and Pharmacokinetics of Multiple Ascending Doses of NIO752 in Progressive Supranuclear Palsy

A Randomized, Participant, Investigator and Sponsor Blinded, Placebo-Controlled Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Multiple Ascending Doses of Intrathecally Administered NIO752 in Participants With Progressive Supranuclear Palsy

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 59 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 40 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

This is a phase 1, multi-center, double-blind, placebo-controlled, multiple dose escalation study with NIO752 in progressive supranuclear palsy (PSP) participants.

Detailed description

This is a phase 1, multi-center, double-blind, placebo-controlled, multiple dose escalation study with NIO752 in progressive supranuclear palsy (PSP) participants. Approximately 58 PSP participants in 5 cohorts will be randomized to receive NIO752 or placebo in a ratio of 3:1. Intrathecal (IT) injections will be given multiple times over 3 months and participants will remain in study for an additional 9-month follow-up period; or will be given multiple times over 9 months and participants will remain in study for an additional 3-month follow-up period. Cohorts will be enrolled sequentially. Safety assessments will include physical and neurological examinations, ECGs, vital signs, standard clinical laboratory evaluations (hematology, blood chemistry, and urinalysis), CSF laboratory test, adverse event, and serious adverse event monitoring.

Conditions

• Progressive Supranuclear Palsy (PSP)

Interventions

Type	Name	Description
DRUG	antisense oligonucleotide	solution of antisense oligonucleotide injected intrathecally (spine tap) at multiple dose levels
DRUG	placebo	placebo for each dose level

Timeline

• Start date: 2021-02-16
• Primary completion: 2024-10-17
• Completion: 2024-10-17
• First posted: 2020-09-04
• Last updated: 2025-12-24

Locations

12 sites across 4 countries: United States, Canada, Germany, United Kingdom

Regulatory

• FDA-regulated drug study