RecruitingPhase 1

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

A Randomized, Participant & Investigator Blinded, Placebo-Controlled Study to Evaluate the Ability of Intrathecally Administered NIO752 to Lower CSF Total Tau Synthesis in Participants With AD Measured by Stable Isotope Labelling Kinetics

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
21 Years to 80 Years
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests whether NIO752 lowers the amount of tau protein made by the brain. Tau can build up and damage nerve cells in Alzheimer's disease. The study is for adults ages 21 to 80 with mild or moderate Alzheimer's disease.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Can sign a form agreeing to take part in the study.
  • Are 21 to 80 years old.
  • Have mild or moderate Alzheimer's disease, with a Clinical Dementia Rating score of 0.5 to 2, and can complete the study.
  • Have tests of spinal fluid, blood, or a PET brain scan that support the diagnosis, or have symptoms and an accepted PSEN or APP gene change linked to Alzheimer's disease.
  • Speak English fluently.
  • Have a reliable study partner or caregiver.
  • Can have spinal taps to collect fluid around the brain and spine, MRI scans, and blood draws.
  • Agree to share samples from your body and personal data with Novartis, the study's business partner.

Who can't join

  • Live in a nursing facility with skilled care or a dementia care facility; cannot follow study instructions or attend visits; or have another condition the study doctor believes would prevent safe participation.
  • Have important abnormal lab results, major disease of the brain's blood vessels, or another illness that affects thinking or memory. Also excluded: HIV, hepatitis B or C, or a higher risk of meningitis (infection around the brain and spine) without proper preventive treatment.
  • Attempted suicide, or had thoughts of suicide with a plan that required a hospital stay, within the past 12 months.
  • Used an experimental treatment within 180 days before treatment starts, or longer if it stays in the body longer. Ever used a MAPT antisense treatment, or another antisense or gene treatment for Alzheimer's disease. These treatments act on genetic instructions.
  • Have an allergy to a study treatment, its ingredients, or similar drugs; cannot have an MRI; or regularly take blood-thinning medicines that make spinal taps unsafe.
  • Currently use cannabis or drink more than 14 units of alcohol a week, or had alcohol or drug abuse or dependence in the past 2 years, except nicotine dependence.
  • Take certain medicines that suppress the immune system, treat mental health or movement disorders, or affect thinking. Allowed Alzheimer's medicines, such as memantine or cholinesterase inhibitors, must have a stable dose for 12 weeks before screening and throughout the study.
  • Are pregnant or breastfeeding, or could become pregnant and do not use highly effective birth control. Sexually active men must agree to use condoms from consent until at least 15 weeks after treatment.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility 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.

Study sites (1)

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