RecruitingPHASE1INTERVENTIONAL

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

This research study is looking into a new medication called NIO752. Scientists believe this drug might lower the amount of a protein called tau that the brain produces. In Alzheimer's disease, tau builds up inside brain cells and forms clumps, which then cause damage and lead to memory and thinking problems. The goal of this study is to find out if NIO752 can stop or slow down this build-up. We hope that by reducing tau, we can protect brain cells and potentially help people with Alzheimer's. This is an early-stage study (Phase 1) focused on checking if the drug is safe and if it has the desired effect on tau levels.

At a glance

Status

Recruiting

Phase

PHASE1

Sponsor

University College, London

Enrolment target

Start

18 Nov 2024

Estimated completion

01 Jul 2026

Alzheimer Disease Autosomal 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)

What is this study about?

This study is trying to understand if a new medicine, called NIO752, can help people with Alzheimer's disease. Our brains have many proteins, and one important protein is called tau. Normally, tau helps keep our brain cells strong and healthy. However, in Alzheimer's disease, something goes wrong, and too much tau starts building up inside the brain cells. These extra tau proteins then stick together, forming harmful clumps. These clumps stop brain cells from working properly and eventually cause them to die, which leads to the memory loss and other difficulties seen in Alzheimer's.

Scientists believe that NIO752 might be able to reduce how much tau the brain makes. If we can lower the amount of tau produced, it might prevent these harmful clumps from forming or growing. This could help protect brain cells and slow down the progression of Alzheimer's disease. This is an important step in finding new ways to treat this condition.

To find out if NIO752 works, we will be comparing it to a 'placebo'. A placebo looks like the study drug but doesn't contain any active medicine. This helps researchers see if the real drug is truly having an effect, or if any changes are happening for other reasons.

Key takeaways

This study is testing an experimental drug, NIO752, for early Alzheimer's disease.
The drug aims to reduce levels of a protein called tau, which is involved in Alzheimer's damage.
Participants will receive either the active drug or a placebo (dummy treatment).
It involves regular clinic visits, blood tests, memory tests, and procedures like spinal fluid collection.
Participation requires a confirmed Alzheimer's diagnosis and a reliable care partner.
You can stop participating at any time without affecting your normal medical care.

Who may be eligible?

This study is looking for people aged between 21 and 80 years old who have been diagnosed with mild or moderate Alzheimer's disease. You'll need to be able to understand and sign consent forms yourself, and we'll need to see evidence from previous medical tests (like brain scans or blood tests) that supports your Alzheimer's diagnosis. It's important that you also have a trusted person, like a family member or friend, who can support you throughout the study.

There are also some reasons why you might not be able to join. For example, if you live in a nursing home, have certain serious medical conditions, or have recently taken part in other experimental drug trials for Alzheimer's, you probably won't be eligible. The doctor leading the study will review all your medical information carefully to make sure the study is safe and appropriate for you.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you between 21 and 80 years old?
Do you have a diagnosis of mild or moderate Alzheimer's disease?
Do you have a trusted friend or family member who can be your study partner?
Are you able to undergo various medical procedures, including blood tests and a spinal fluid collection (lumbar puncture)?
Have you spoken with your doctor about participating in research studies for Alzheimer's?
Have you avoided other experimental Alzheimer's treatments in the last six months?

Answer every question to see your result.

What does participation involve?

If you join this study, your participation will involve several visits to the clinic over a period of time. You will receive either the study drug (NIO752) or a placebo, which is a dummy treatment. You won't know which one you are receiving. To check how you're doing and whether the drug is having an effect, you will have various assessments. These will include regular check-ups with the doctor, blood tests, and some memory and thinking tests.

To measure the tau protein in your brain, you will also need to have procedures like a lumbar puncture (spinal tap) to collect a small sample of fluid from around your spine, and possibly MRI brain scans. These tests help us understand how the drug is affecting your body. We will also ask to share your biological samples (like blood) and personal health information with our commercial partner, Novartis, for further research. The study team will explain everything in detail, including the total duration of the study, and answer any questions you have.

Potential risks and benefits

Taking part in a clinical trial like this could potentially offer benefits, such as access to a new treatment before it's widely available, and close medical monitoring. However, there are also potential risks involved. The main risks are side effects from the study drug, or discomfort from the procedures like blood tests, lumbar punctures, and MRI scans. The study team will explain all known potential side effects and risks to you. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (2)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Washington University in St. Louis
Verified postcode
St Louis, United States· Recruiting
University College London Hospitals NHS Foundation Trust
Verified postcode
London, United Kingdom· Recruiting

Common questions

What is 'tau' and why is it important in Alzheimer's disease?

Tau is a protein that normally helps brain cells function. In Alzheimer's, too much tau builds up and forms harmful clumps, damaging brain cells and leading to symptoms.

What is a 'placebo' in this study?

A placebo is a dummy treatment that looks like the real drug but contains no active medicine. It helps researchers see if the actual drug is truly making a difference.

Will I know if I'm getting the real drug or the placebo?

No, neither you nor your doctor will know whether you are receiving the study drug (NIO752) or the placebo. This helps ensure unbiased results.

What is a lumbar puncture (spinal tap) and why do I need one?

A lumbar puncture is a procedure where a small needle is used to collect a sample of fluid from around your spine. We use this fluid to measure tau levels in your brain.

Can I stop participating in the study if I change my mind?

Yes, absolutely. You have the right to withdraw from the study at any time, for any reason, without it affecting the medical care you receive.

How to find out more

Ross Paterson

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

Always speak to your GP or specialist before deciding to take part in a study.