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

Official 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.

Study Design

• Study Type: INTERVENTIONAL
• Allocation: RANDOMIZED
• Model: PARALLEL
• Masking: TRIPLE
• Enrollment: 10 participants

Interventions

• DRUG: NIO752 — Antisense oligonucleotide
• OTHER: Placebo — Saline

Primary Outcomes

• Tau synthesis rate inhibition in individuals with sporadic AD and ADAD (Day 23)

Secondary Outcomes

• Compare efficacy of knockdown of tau production in sporadic AD and ADAD by measuring the synthesis rate of tau by determining the ratio of labelled to unlabeled tau (tracer to tracee ratio) in serial cerebrospinal fluid samples. (Day 23)
• Number of participants with adverse events [safety and tolerability] (Day 0 to Day 145)
• Comparison of number of Adverse Events reported between participants receiving one dose of NIO752 versus those receiving two doses of NIO752. (Day 0 to Day 145)
• Compare rates of tau synthesis and clearance in sporadic AD and ADAD (Day 23)
• Determine CSF tau concentration to tau production relationships in humans (120 days)

Trial Locations

• Washington University in St. Louis, St Louis, Missouri, United States
• University College London Hospitals NHS Foundation Trust, London, United Kingdom

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