NIO752 for Alzheimer's Disease

(NIO-SILK Trial)

Recruiting at 1 trial location

Overseen By Lisa French

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: University College, London

Must be taking: Cholinesterase inhibitors, Memantine

Must not be taking: Immunosuppressants, Antipsychotics, Lithium, others

Disqualifiers: Skilled nursing facility, Suicidal ideation, Alcohol use, others

Compensation: Varies

Number of Visits: Unknown

Duration: 23 days

10 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new drug, NIO752, to determine if it can reduce harmful proteins in the brain that cause Alzheimer's disease. In Alzheimer's, these proteins accumulate and form tangles, damaging nerve cells and leading to memory and thinking problems. Participants will receive either NIO752 or a placebo (a harmless substance resembling the drug) to compare effects. Individuals with mild to moderate Alzheimer's who can undergo medical procedures like MRIs and blood tests might be suitable candidates. As a Phase 1 trial, this research aims to understand how NIO752 works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

You may need to stop taking certain medications to join this trial. Specifically, you cannot be on immunosuppressants, antipsychotics, lithium, neuroleptics, dopaminergic agonists, L-dopa, or monoamine oxidase inhibitors. If you are taking cholinesterase inhibitors or memantine, your dose must be stable for at least 12 weeks before the trial and remain stable during the study.

Is there any evidence suggesting that NIO752 is likely to be safe for humans?

Research has shown that people who received NIO752 exhibited more signs of inflammation in the cerebrospinal fluid (CSF), the fluid around the brain and spinal cord. However, no signs of infection were present. This suggests an immune response without indicating an infection.

As a Phase 1 trial, this is the initial step in testing NIO752's safety in humans. At this stage, researchers focus on observing the body's reaction to the treatment and identifying any side effects. Although data remains limited, the ongoing trial indicates that NIO752 has shown enough promise in earlier tests to proceed.

Prospective participants should know that researchers closely monitor for side effects and will provide detailed information about potential risks. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Alzheimer's?

NIO752 is unique because it is administered intrathecally, which means it is delivered directly into the spinal canal, allowing for more targeted delivery to the brain. Unlike standard Alzheimer's treatments like donepezil or memantine, which mainly focus on managing symptoms, NIO752 may offer a new approach by potentially addressing underlying disease mechanisms. Researchers are excited about this treatment because it could lead to more effective ways to slow down or alter the progression of Alzheimer's disease, rather than just temporarily alleviating symptoms.

What evidence suggests that NIO752 might be an effective treatment for Alzheimer's?

Research has shown that NIO752, which participants in this trial may receive, might help lower tau levels, a protein that can accumulate and cause brain cells to malfunction and die. In Alzheimer's disease, this tau buildup leads to dementia symptoms. Early studies suggest that NIO752 can reduce tau levels, which is promising because less tau might mean less harm to brain cells. Although the data remains in the early stages, the aim is to determine if reducing tau can slow or improve Alzheimer's symptoms. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

This trial is for adults aged 21-80 with mild or moderate Alzheimer's, confirmed by specific tests and a Clinical Dementia Rating score of 0.5 to 2. Participants must be able to undergo various medical procedures like lumbar punctures and MRI scans, have a reliable caregiver, speak English fluently, and consent to data sharing.

Inclusion Criteria

I can sign a consent form.

I am between 21 and 80 years old.

I have been diagnosed with mild or moderate Alzheimer's disease.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive intrathecal administration of NIO752 or placebo to assess tau synthesis rate inhibition

23 days

Multiple visits for administration and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

20 weeks

Regular visits for safety assessments

What Are the Treatments Tested in This Trial?

Interventions

• NIO752

Trial Overview

The study is testing NIO752 against a placebo to see if it can reduce the production of tau protein in the brain. Tau buildup leads to nerve cell damage in Alzheimer's disease. The trial will measure how well NIO752 works compared to not receiving the active drug.

How Is the Trial Designed?

Group I: NIO752 Experimental Treatment 1 Intervention

Intrathecal administration.

Group II: Saline Placebo Group 2 Interventions

10mL of saline (placebo) is administered intrathecally.

Who Is Running the Clinical Trial?

University College, London

Lead Sponsor

Trials

884

Recruited

38,770,000+

Alzheimer's Association

Collaborator

Trials

103

Recruited

44,300+

Washington University School of Medicine

Collaborator

Trials

2,027

Recruited

2,353,000+

University of Washington

Collaborator

Trials

1,858

Recruited

2,023,000+

Sigrid Rausing Trust

Collaborator

Trials

2

Recruited

810+

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT06372821

A Trial Evaluating the Effect of NIO752 on Tau Synthesis ...

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.

2.

novartis.com

novartis.com/clinicaltrials/study/nct05469360

Study of Safety, Tolerability, Pharmacodynamics and ...

Phase 1b study to assess the pharmacodynamics, safety, tolerability, and pharmacokinetics of NIO752 in patients with early Alzheimer's disease (AD)

3.

alzforum.org

alzforum.org/therapeutics/nio752

NIO752

According to study results posted on the Novartis clinical trial website, six doses were tested. Twenty-two participants had serious adverse ...

4.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC11497713/

Amyloid-targeting therapies - Alzheimer's disease efficacy ...

Recent clinical trials of amyloid beta (Aβ)‐targeting therapies in Alzheimer's disease (AD) have demonstrated a clinical benefit over 18 months, ...

5.

alz-journals.onlinelibrary.wiley.com

alz-journals.onlinelibrary.wiley.com/doi/10.1002/trc2.12465

Alzheimer's disease drug development pipeline: 2024

There are 164 trials assessing 127 drugs across the 2024 AD pipeline. There were 48 trials in Phase 3 testing 32 drugs, 90 trials in Phase 2 assessing 81 drugs ...

6.

trialx.com

trialx.com/clinical-trials/listings/243801/study-of-safety-tolerability-pharmacodynamics-and-pharmacokinetics-of-nio752-in-early-alzheimers-disease-participants/?&geo_lat=41.4469883&geo_lng=2.2450325&radius=10&place=Badalona&user_country=Spain

Clinical Research Trial Listing ( Alzheimer Disease

Study of Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of NIO752 in Early Alzheimer's Disease Participants.

7.

novctrd.com

novctrd.com/ctrdweb/trialresult/trialresults/pdf?trialResultId=18339

Sponsor Generic Drug Name Trial Indication(s) Protocol ...

These safety and tolerability data coupled with PK findings support the future development of NIO752 in the PSP program. Date of Clinical ...