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Bisnorcymserine in Healthy Adult Volunteers

Phase 1
Completed
Conditions
Healthy Volunteers
Interventions
Other: Placebo
Registration Number
NCT01747213
Lead Sponsor
National Institute on Aging (NIA)
Brief Summary

Background:

- Alzheimer s disease (AD) is a brain disease that impairs memory, cognitive abilities and the ability to function independently. It is the most common cause of dementia in older people. It is caused by abnormal proteins in the brain that affect how neurons communicate with each other. Researchers are looking for drugs that can slow down the disease or treat its symptoms. One drug, called bisnorcymserine (BNC), may help improve brain function and symptoms in people with AD. BNC is designed to block a chemical that affects how neurons communicate with each other. Researchers want to see how BNC works in healthy older volunteers.

Objectives:

- To look at how the body processes bisnorcymserine taken by mouth and how safe it is for healthy older volunteers.

Eligibility:

- Healthy volunteers at least 55 years of age.

Design:

* Participants will be screened with a physical exam, medical history, and blood and urine tests.

* Within 3 weeks from the screening visit, participants will come to the National Institute on Aging clinical unit for a 2-night stay. On the morning of the second day, they will take either a BNC capsule or a placebo. They will not know which tablet they are taking.

* Blood samples will be collected frequently throughout the second and third days of the study visit. The last blood sample will be collected about 32 hours after taking the study capsule. Participants will have heart function tests and other exams during the visit. Once the tests are done, they will leave the clinical center.

* Participants will have a final follow-up visit about 1 week after leaving the clinical center.

Detailed Description

Objective: Alzheimer s disease (AD) is a progressive neurodegenerative disease that impairs memory and other cognitive abilities, as well as behavior and the ability to function independently. It is the most common cause of dementia among older people. Alzheimer s disease is characterized by deficits in several neurotransmitter systems, most prominently acetylcholine (Ach). The cholinergic deficit in the AD brain is associated with the cognitive and functional symptoms in AD. Restoring this deficit in the cholinergic system is one proven approach for symptomatic treatment in AD. The action of Ach in the brain is terminated mainly by two enzymes called cholinesterases (ChEs): acetylcholinesterase (AChE) and butyrylcholinesterase (BChE). Inhibitors of these enzymes therefore augment the activity of surviving Ach neurons in AD. All ChEs inhibitors currently approved for the treatment of AD mainly inhibit AChE and, secondarily and to a varying extent, BChE. Reversible and brain-specific BChE inhibitors have been developed as a class of drugs called cymserine analogs. Scientists at the NIA/NIH have developed a novel BChE inhibitor called Bisnorcymserine (BNC). Pre-clinical evidence suggests that BNC may be a safe treatment for Alzheimer s disease. Based on this, we propose this first-in-human study to evaluate the safety, tolerability and pharmacokinetics of single doses of BNC tartrate administered orally.

Study population: Healthy volunteers aged 55 years and older.

Design: Double blind placebo-controlled Phase I clinical Trial of single oral doses of BNC doses in an ascending schedule: 20, 40mg, 80 mg, 120 mg, 160 mg, 270 mg and 380 mg. Each dose will be tested in groups of 8 subjects. Six subjects in each cohort will receive active drug and two will receive placebo. Subjects will be kept in the unit and followed clinically and with laboratory tests for adverse effects for 32 hours; they will return for a follow-up visit to assess safety in about 7 days. A Data Safety Monitoring Board will evaluate the safety and tolerability associated with each dose level before the next higher dose is tested in a new cohort. All research will be performed at the National Institute on Aging (NIA) Clinical Research Unit located on the 5th floor of MedStar Harbor Hospital.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
75
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PlaceboPlacebomicrocrystalline celluose
BNCBNCBisnorcymserine tartrate
Primary Outcome Measures
NameTimeMethod
Safety and TolerabilityWill be completed in one year

Safety and tolerability will be determined based on the frequency of AEs of any grade in BNC and placebo groups. AEs will be determined based on clinical symptoms and signs, vital signs, safety laboratory tests, EKG and continuous cardiac monitoring, MMSE, and C-SSRS.

Secondary Outcome Measures
NameTimeMethod
pharmacokinetic profile (parameters)Will be completed in one year

The pharmacokinetic profile of a single oral dose of BNC in a capsule will be determined, including evaluation of AUC, Tmax, Cmax, elimination rate constant, T1/2, clearance and volume of distribution. In addition, we will measure pharmacodynamics parameters, including acetyl-cholinesterase and butyryl-cholinesterase activity

Trial Locations

Locations (2)

National Institute on Aging, Clinical Research Unit

🇺🇸

Baltimore, Maryland, United States

Harbor Hospital Center

🇺🇸

Baltimore, Maryland, United States

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