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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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59 people
The number who actually took part.
- Started
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Feb 2021
- Finished
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Oct 2024
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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40 to 75 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Signed informed consent 2. Between 40 to 75 years old (inclusive) 3. Have PSP diagnosed for less than 5 years with a current classification of probable PSP Richardson syndrome, a progressive supranuclear palsy rating scale (PSPRS) score \< 40 and MOCA score \>17 at screening 4. Be able to ambulate independently or able to take at least 5 steps with minimal assistance 5. At least a 12-month history of postural instability or falls within 3 years from disease onset as per medical history 6. Vertical supranuclear gaze palsy, or reduced velocity of vertical saccade 7. Able and willing to meet all study requirements including: Have a study partner who is reliable, competent, and at least 18 years of age, and will be able to accompany the participant to study visits, be knowledgeable of the participant's ongoing condition during the study to provide study related information to study site when required both in person and via a phone Reside in a proximity to the study site to allow a timely unscheduled visit if necessary (ideally less than 2 hours) Able to undergo lumbar puncture (LP), CSF draws and blood draws 8. If the participant is receiving levodopa/carbidopa, levodopa/benserazide, a dopamine agonist, catechol-o-methyltransferase (COMT) inhibitor, rasagiline, CoQ10 or other Parkinson's medications, acetylcholinesterase inhibitors, antipsychotics, memantine, or other non-tau modifying Alzheimer's medication the dose must have been stable for at least 30 days prior to the screening visit and must remain stable for the duration of the study. No such medication can be initiated during the study. Exclusion Criteria: 1. Live in a skilled nursing facility or dementia care facility 2. Evidence of motor neuron disease, or any other neurological disease that could explain symptoms 3. Clinically significant laboratory abnormality 4. Attempted suicide, suicidal ideation with a plan that required hospital admission within 12 months prior to Screening. In addition, patients deemed by the Investigator to be at significant risk of suicide, major depressive episode, psychosis, confusion state, or violent behavior should be excluded. 5. A clear and robust benefit from levodopa by history 6. Use of lithium, methylene blue or other putative disease modifying drugs for PSP within 30 days of screening 7. Any previous use of experimental therapy within 30 days or 5 half-lives prior to Day 1, whichever is greater 8. Any condition that increases risk of meningitis unless participant is receiving appropriate prophylactic treatment 9. History of post-lumbar-puncture headache of moderate or severe intensity and/or blood patch 11\. Hospitalization for any major medical or surgical procedure involving general anesthesia within 12 weeks of Screening or planned during the study 12. Unable to undergo magnetic resonance imaging (MRI) due to for example claustrophobia, or presents absolute contraindications to MRI (e.g., metallic implants, metallic foreign bodies, pacemaker, defibrillator) 13. Patients with other significant brain MRI abnormalities by history or at screening.
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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
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
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