New spinal injection drug tested for rare brain disorder

NCT ID NCT04539041

Completed Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage trial tested a drug called NIO752 in 59 people with progressive supranuclear palsy (PSP), a rare brain disorder that affects movement and balance. The drug was given as a spinal injection to see if it is safe and how the body processes it. The main goal was to check for side effects, not to measure if it works.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: NIO752 (an antisense oligonucleotide given via spinal injection)
What this could lead to: If successful, this could lead to a treatment that slows or controls the progression of progressive supranuclear palsy.
What could go wrong: This is an early Phase 1 trial with only 59 participants, focused on safety. It is too small and early to know if the drug works, and there may be risks from the spinal injections or the drug itself.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for PROGRESSIVE SUPRANUCLEAR PALSY(PSP) are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

Pick disease progressive supranuclear palsy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PROGRESSIVE SUPRANUCLEAR PALSY(PSP)

Contacts and locations

Locations

Mayo Clinic Rochester

Rochester, Minnesota, 55905, United States
Novartis Investigative Site

Montreal, Quebec, H2X 1R9, Canada
Novartis Investigative Site

Montreal, Quebec, H3A 2B4, Canada
Novartis Investigative Site

Bonn, North Rhine-Westphalia, 53127, Germany
Novartis Investigative Site

Düsseldorf, 40225, Germany
Novartis Investigative Site

Hanover, 30625, Germany
Novartis Investigative Site

München, 81377, Germany
Novartis Investigative Site

Tübingen, 72076, Germany
Novartis Investigative Site

Ulm, 89081, Germany
Novartis Investigative Site

Southampton, SO16 6YD, United Kingdom
University of California San Diego

La Jolla, California, 92037, United States
Vanderbilt University Medical CenterX

Nashville, Tennessee, 37221, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.