NCT06372821

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

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: University College, London · Academic / Other
• Sex: All
• Age: 21 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• 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)

Interventions

Type	Name	Description
DRUG	NIO752	Antisense oligonucleotide
OTHER	Placebo	Saline

Timeline

• Start date: 2024-11-18
• Primary completion: 2026-07-01
• Completion: 2026-07-01
• First posted: 2024-04-18
• Last updated: 2026-01-27

Locations

2 sites across 2 countries: United States, United Kingdom

Regulatory

• FDA-regulated drug study