Experimental Alzheimer's drug aims to quiet Brain's toxic tau
NCT ID NCT06372821
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial tests a drug called NIO752, given via spinal injection, to see if it can reduce production of tau protein in the brain. Tau buildup is linked to Alzheimer's damage. Ten people with mild to moderate Alzheimer's will receive either NIO752 or a placebo, and researchers will measure tau levels using a special labeling technique. The goal is to see if the drug safely lowers tau production.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- NIO752 (antisense oligonucleotide)
- What this could lead to
- If it works, this could point toward a treatment that slows or stops Alzheimer's by reducing harmful tau buildup in the brain.
- What could go wrong
- This is a very early Phase 1 trial with only 10 participants, so it is too soon to know if the drug is safe or effective. The approach is experimental and may not work in larger groups.
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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About 10 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Nov 2024
- Expected to finish
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Jul 2026
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
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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21 to 80 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. Able to provide signed informed consent. 2. Between 21 to 80 years old (inclusive). 3. A diagnosis of mild or moderate Alzheimer's disease by a Clinical Dementia Rating score of 0.5 to 2, where the investigator believes they will be able to complete the study. 4. A history of cerebrospinal fluid, Positron Emission Topography (PET), or blood-based biomarkers supporting the diagnosis of Alzheimer's disease, or symptomatic approved presenilin (PSEN) or amyloid precursor protein (APP) mutation carriers. If blood biomarkers are equivocal then amyloid status can be confirmed using cerebrospinal fluid. 5. Fluency in English 6. Participant has a reliable study partner or caregiver 7. Able to undergo lumbar punctures, magnetic resonance imaging (MRI), cerebrospinal fluid draws, and blood draws. 8. Individuals will be willing to consent for their biological samples and personal data to be shared with the commercial partner (Novartis) Exclusion Criteria: 1. Live in a skilled nursing facility or dementia care facility. 2. Any clinically significant laboratory abnormality 3. Attempted suicide, suicidal ideation with a plan that required hospital admission within 12 months prior to Screening 4. Any previous use of experimental therapy within 180 days or 5 half-lives prior to Day 1, whichever is greater. 5. Any previous use of MAPT antisense oligonucleotides (ASO) or any other ASO or other gene therapy meant as treatment for Alzheimer's disease. 6. History of hypersensitivity to any of the study treatments or its excipients or to drugs of similar chemical classes. 7. Any condition that increases risk of meningitis unless participant is receiving appropriate prophylactic treatment. 8. Current medical or non-Alzheimer's disease neurological condition that might impact cognition or performance on cognitive assessments 9. Have any other conditions which, in the opinion of the investigator, would make the participant unsuitable for inclusion or could interfere with the patient participating in or completing the study. 10. Unlikely to cooperate in the study; not able to attend scheduled examinations and visits; or not able to follow study instructions per the judgement of the investigator. 11. Current alcohol (\>14 units per week) or current cannabis use; or history of alcohol or drug abuse or dependence (except nicotine dependence) within 2-years before the screening visit. 12. Treatment with immunosuppressants, antipsychotics, lithium, neuroleptics, dopaminergic agonists, L-dopa, or monoamine oxidase inhibitors at the time of screening. Current use of medications, other than cholinesterase inhibitors and/or memantine, that could alter cognition, as determined by the Investigator. If patients are taking cholinesterase inhibitors and/or memantine at screening, the dose must have been stable within 12-weeks prior to screening and must remain stable during the duration of the study. 13. Unable to undergo MRI due to for example claustrophobia, or presents absolute contraindications to MRI (e.g., metallic implants, metallic foreign bodies, pacemaker, defibrillator). 14. Significant signs of major cerebrovascular disease 15. Sexually active males, unless they agree to use a condom during intercourse from the time of consent until a minimum of 15 weeks after treatment. 16. Breast feeding women, pregnant women, and females of reproductive potential unless they use highly effective contraception methods, as specified in the protocol. 17. Patients on regular anticoagulants or anti-platelets that would preclude lumbar puncture are not eligible to participate 18. Seropositive for human immunodeficiency virus (HIV), Hepatitis B or hepatitis C.
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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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
2 sites in 2 countries. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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University College London Hospitals NHS Foundation Trust
RECRUITINGLondon, NW1 2PG, United Kingdom
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Washington University in St. Louis
RECRUITINGSt Louis, Missouri, 63110, United States
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