A Pilot Clinical Trial of Exendin-4 in Alzheimer's Disease

What Was Tested

In this phase 2 trial, researchers set out to test Exenatide.

Researchers are interested in studying the safety and comparing the effects of Exendin-4 with placebo on cognitive performance, clinical progression of dementia, various chemicals measured in blood and cerebrospinal fluid, and brain MRI, in individuals with early-stage Alzheimer's disease or mild cognitive impairment.

It had two arms (groups): Exendin-4 (experimental) and Placebo (placebo comparator).

Placebo SC twice daily.

The registry classes the interventions as drug.

More on the Plan

Objective: Exendin-4 (or exenatide) is a medication currently used in the treatment of diabetes mellitus (DM).

Specifically, there is pre-clinical evidence that Exendin-4 may be a treatment for Alzheimer's disease (AD).

Design: Enrolled participants will be randomly assigned into one of two groups (Exendin-4 vs.

Placebo) and will be followed at regular intervals for 18 months.

Who Took Part

Enrollment: 57 actual participants took part.

It was open to people of any sex aged from 60 years with no upper age limit.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Age \> 60. Clinical Dementia Rating (global CDR) of 0.5 or 1. Memory box score must be at least 0.5. Mini Mental Status Exam (MMSE) \> 20. Clinical diagnosis of (amnestic or mixed) MCI or early AD and Memory deficit on neuropsychological or clinical testing. Hamilton Depression Scale score of less than or equal to 12 on the 17-item scale.

The full record lists 15 inclusion and 17 exclusion criteria; among the exclusions: positive rpr or hiv; anti-coagulant therapy (such as coumadin). aspirin up to 325 is allowed; investigators unable to obtain csf, failure of lumbar puncture after a limited number of unsuccessful attempts).

How the Study Was Run

It was run as a randomized, parallel-group and quadruple-blind study.

The participants were allocated by chance, so the Exenatide group and the comparison group should start out alike.

Groups ran in parallel: each of the participants stayed in one arm throughout.

As a double-blind Exenatide study, the assignments were hidden from both participants and investigators until the end.

Those kept unaware of assignments: participant, care provider, investigator and outcomes assessor.

It started in 2010-11-21 with completion listed for 2016-11-18.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Number of Participants With Incidence of Nausea (time frame: 18 months).

It also tracked 8 secondary outcomes, for example Mini Mental State Examination (MMSE), Alzheimer's Disease Assessment Scale-cognitive Subscale (ADAS-cog70), Clinical Dementia Rating (CDR) Global Score, Clinical Dementia Rating (CDR) Sum of Boxes, Cerebrospinal Fluid (CSF) Total Tau and Cerebrospinal Fluid phospho181-tau (CSF p181-tau).

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of Exenatide in 57 people.

It ended early (terminated); results are posted on the registry, and this page does not restate them.

More context: the Exenatide compound page, all human trials.