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 Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
10
Washington University in St. Louis
St Louis, Missouri, United States
RECRUITINGUniversity College London Hospitals NHS Foundation Trust
London, United Kingdom
RECRUITINGTau synthesis rate inhibition in individuals with sporadic AD and ADAD
Tau synthesis rate calculated using tracer to tracee ratio of tau specific peptide calculated on day 20 post leucine administration in individuals with sporadic and ADAD (analyzed collectively) receiving intrathecal NIO752 compared to placebo.
Time frame: Day 23
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.
Time frame: Day 23
Number of participants with adverse events [safety and tolerability]
Time frame: 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.
Compare safety between 1 and 2 doses.
Time frame: Day 0 to Day 145
Compare rates of tau synthesis and clearance in sporadic AD and ADAD
Time frame: Day 23
Determine CSF tau concentration to tau production relationships in humans
Time frame: 120 days
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