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Looking for participantsPhase1

A Trial Evaluating the Effect of NIO752 on Tau Synthesis Measured by a Process Known as SILK

Sponsor: University College, London

NCT ID: NCT06372821

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
NIO752 (drug), Placebo (other)
How long the study runs
Study runs about 20 months (dates as stated)
About the drug or intervention
NIO752 — drug: Antisense oligonucleotide · Placebo — other: Saline
Patient visit burden
Not specified by the sponsor

In plain English

This study looks at whether a drug called NIO752 can reduce the making of a protein called tau in the brain, which is linked to Alzheimer's disease. The trial is run by University College London with a company called Novartis. Tau levels will be measured using a process called SILK.

Who can take part

  • Aged 21 to 80 years old
  • Diagnosed with mild or moderate Alzheimer's disease (Clinical Dementia Rating score of 0.5 to 2)
  • Tests (spinal fluid, PET scan, or blood tests) that support the Alzheimer's diagnosis, or carrying certain gene changes (presenilin or amyloid precursor protein) that cause inherited Alzheimer's disease
  • Fluent in English
  • Has a reliable study partner or carer
  • Able to have lumbar punctures, MRI scans, and spinal fluid and blood samples taken
  • Willing to allow samples and personal data to be shared with Novartis

Who may not be able to

  • Living in a nursing or dementia care facility
  • Significant abnormal laboratory test results
  • Suicide attempt or suicidal thoughts needing hospital admission in the past 12 months
  • Use of any experimental therapy within 180 days (or 5 half-lives, if longer) before the study
  • Previous use of MAPT antisense oligonucleotides, other antisense oligonucleotides, or gene therapy for Alzheimer's disease
  • Allergic reactions to the study treatment, similar drugs, or their ingredients
  • Any condition raising the risk of meningitis, unless receiving suitable preventive treatment
  • Another medical or brain condition (not Alzheimer's) that could affect thinking or memory tests
  • Any other condition the researcher believes would make taking part unsuitable
  • Unlikely to attend visits or follow study instructions
  • Drinking more than 14 units of alcohol a week, current cannabis use, or alcohol or drug misuse in the past 2 years (smoking nicotine is allowed)
  • Taking certain medicines, including immunosuppressants, antipsychotics, lithium, neuroleptics, dopaminergic agonists, L-dopa, or monoamine oxidase inhibitors; memory medicines (cholinesterase inhibitors or memantine) are allowed if the dose has been stable for 12 weeks
  • Unable to have an MRI scan (for example due to claustrophobia, metal implants, or a pacemaker)
  • Significant cerebrovascular (blood vessel) disease in the brain
  • Sexually active men who will not use a condom during intercourse until at least 15 weeks after treatment
  • Breastfeeding or pregnant women, and women who could become pregnant unless using highly effective contraception
  • Taking regular blood-thinning medicines (anticoagulants or anti-platelets) that would prevent lumbar puncture
  • Testing positive for HIV, hepatitis B, or hepatitis C

What taking part involves

  • • Taking the study drug NIO752 (how it is given is not stated — ask the trial team)
  • • Lumbar punctures (a needle in the lower back to collect spinal fluid)
  • • MRI scans of the brain
  • • Blood tests
  • • Tau production measured using a process called SILK

Time commitment: Number and length of visits, and total study duration, are not stated — ask the trial team; taking part involves lumbar punctures, MRI scans, spinal fluid and blood samples.

Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.

Type of study
Testing a treatment
Ages
21 Years to 80 Years
Who
All
Number of participants
10
Started
2024-11-18
Last checked
2026-01

Plain English Summary

What is this study?

  • • Testing a new treatment for alzheimer disease
  • • Phase1 - 10 participants
  • • This study will assess if drug (NIO752) reduces production of a protein, tau, by the brain

Who can take part?

  • • Ages 21 Years to 80 Years
  • • Diagnosed with alzheimer disease

Where?

  • • London - University College London Hospitals NHS Foundation Trust

This is a simplified summary. Always discuss with your doctor before making any decisions.

About This Trial

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.

Alzheimer DiseaseAutosomal Dominant Alzheimer Disease Due to Mutation of Presenilin 1 (Disorder)Autosomal Dominant Alzheimer Disease Due to Mutation of Presenilin 2 (Disorder)Autosomal Dominant Alzheimer Disease Due to Mutation of Amyloid Precursor Protein (Disorder)

How this trial compares with your answers

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What we know so far

Condition· Matched your search
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Gender· Tell us your sex for better matching

Still need:

  • • Tell us your age for better matching
  • • Tell us your sex for better matching

Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 21 Years - 80 Years
  • Who can join: All genders

What the study is looking for

  • ✓Able to provide signed agreement to take part.
  • ✓Between 21 to 80 years old (inclusive).
  • ✓A diagnosis of mild or moderate Alzheimer's disease by a Clinical Dementia Rating score of 0.5 to 2, where the...
  • ✓Fluency in English
  • ✓Participant has a reliable study partner or caregiver

Who cannot take part

  • ✗Live in a skilled nursing facility or dementia care facility.
  • ✗Any clinically significant laboratory abnormality
  • ✗Attempted suicide, suicidal ideation with a plan that required hospital admission within 12 months prior to Screening
  • ✗Any previous use of experimental therapy within 180 days or 5 half-lives prior to Day 1, whichever is greater.
  • ✗Any previous use of MAPT antisense oligonucleotides (ASO) or any other ASO or other gene therapy meant as treatment...
See the full criteria
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.

Where Is This Study? (1 UK site)

University College London Hospitals NHS Foundation Trust

London NW1 2PG, United Kingdom

Recruiting
Site contact (verified)
Ross PatersonPrincipal Investigator

How to Get in Touch

Ross Paterson

Sponsor contact

CONTACT

+44(0)2074483875 r.paterson@ucl.ac.uk

Lisa French

Sponsor contact

CONTACT

l.french@ucl.ac.uk
Data sourced from ClinicalTrials.gov · Last verified: 2026-01