RecruitingAlzheimer DiseaseAutosomal Dominant Alzheimer Disease Due to Mutation of Presenilin 1 (Disorder)Autosomal Dominant Alzheimer Disease Due to Mutation of Presenilin 2 (Disorder)
A Trial Evaluating the Effect of NIO752 on Tau Synthesis Measured by a Process Known as SILK
Eligible age
21–80 yrs
Accepts
All genders
Locations
1 state
Healthy volunteers
No
See if you qualify for this study
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About this study
This study will assess if drug (NIO752) reduces production of a protein, tau, by the brain. Normally tau maintains the internal skeleton of nerve cells. In Alzheimer's disease (AD) it builds up in the brain, causing damage. Abnormal tau proteins cling to each other forming 'tangles' inside nerve cells, which interfere with how the nerve cells work, and eventually die. This is what causes the symptoms of dementia. It is thought that NIO752 reduces production of tau.
Sponsor: University College, London
You may qualify if…
- ✓ 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)
You may not qualify if…
- ✕ 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
Where it's recruiting
Missouri
St Louis
Source: ClinicalTrials.gov · NCT06372821 · last updated 2026-01-27