The Comprehensive Clinical Trials Unit at UCL



1 November 2018

A randomised, double blind, placebo controlled, single centre, 60 week trial of Exenatide once weekly for the treatment of moderate severity Parkinson's disease

The development of a neuroprotective or disease-modifying therapy that may slow or halt progression of the disease is perhaps the most important unmet need in Parkinson's disease (PD).  Exenatide is a licensed and effective treatment for patients with Diabetes mellitus. The neurotrophic properties of Exenatide has sparked interest into its potential use as a neurodegenerative disease modifying agent not only in Parkinson's disease (PD) but also in neurodegenerative conditions such as Alzheimer's disease.

A small, proof of concept, open label trial of Exenatide in moderate severity PD patients (n=45) by the same investigators demonstrated improvements in motor and cognitive assessments in the active treatment group after twelve months exposure to Exenatide. The effects persisted following a two month wash out period.

The findings from the pilot study has led to the current phase 2 trial to evaluate whether beneficial effects can be replicated using double blind placebo controlled methodology.

The primary objective in this trial is to compare the effectiveness of Exenatide once weekly versus placebo on the MDS UPDRS part 3 motor subscale in the "practically defined OFF medication state" in patients (n=60) with moderate severity PD.


Trial Status: Recruitment has ended and all follow up appointments completed. The results have been analysed and manuscripts are currently being prepared for publication.

Funder: Michael J Fox Foundation

Sponsor: University College London