New spinal injection drug tested for rare brain disorder
NCT ID NCT04539041
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Mayo Clinic Rochester
Rochester, Minnesota, 55905, United States
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Novartis Investigative Site
Montreal, Quebec, H2X 1R9, Canada
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Novartis Investigative Site
Montreal, Quebec, H3A 2B4, Canada
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Novartis Investigative Site
Bonn, North Rhine-Westphalia, 53127, Germany
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Novartis Investigative Site
Düsseldorf, 40225, Germany
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Novartis Investigative Site
Hanover, 30625, Germany
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Novartis Investigative Site
München, 81377, Germany
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Novartis Investigative Site
Tübingen, 72076, Germany
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Novartis Investigative Site
Ulm, 89081, Germany
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Novartis Investigative Site
Southampton, SO16 6YD, United Kingdom
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University of California San Diego
La Jolla, California, 92037, United States
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Vanderbilt University Medical CenterX
Nashville, Tennessee, 37221, United States