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| Name | Class |
|---|---|
| Washington University School of Medicine | OTHER |
| University of Washington | OTHER |
| Alzheimer's Association | OTHER |
| Sigrid Rausing Trust |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| NIO752 | Experimental | Intrathecal administration. |
|
| Saline | Placebo Comparator | 10mL of saline (placebo) is administered intrathecally. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| NIO752 | Drug | Antisense oligonucleotide |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Tau 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. | Day 23 |
| Measure | Description | Time Frame |
|---|---|---|
| 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] |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Ross Paterson | Contact | +44(0)2074483875 | r.paterson@ucl.ac.uk | |
| Lisa French | Contact | l.french@ucl.ac.uk |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Washington University in St. Louis | Recruiting | St Louis | Missouri | 63110 | United States |
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| OTHER |
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| Placebo |
| Other |
Saline |
|
| 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. | 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 |
| University College London Hospitals NHS Foundation Trust | Recruiting | London | NW1 2PG | United Kingdom |
|
| ID | Term |
|---|---|
| D000544 | Alzheimer Disease |
| ID | Term |
|---|---|
| D003704 | Dementia |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D024801 | Tauopathies |
| D019636 | Neurodegenerative Diseases |
| D019965 | Neurocognitive Disorders |
| D001523 | Mental Disorders |
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