Exendin-4 as a Treatment for Parkinson's Disease - Pilot Study
Exenatide is a licensed, safe and effective treatment for patients with Diabetes mellitus. Laboratory work has shown strong, reproducible evidence that this drug has beneficial "disease modifying" effects when given to animals with a range of experimental models of Parkinson's disease (PD). This project aims to make an initial evaluation of possible benefits of Exenatide among patients with moderate symptoms of PD. The drug will be given as a twice daily 10microgram injection under the skin in a similar way to one of the conventional "symptomatic" treatments for PD (Apomorphine).
Forty patients with moderate symptoms of PD will be recruited and randomised to receive Exenatide injections twice daily, or to act as controls in this open label trial. Detailed assessments will be made of all patients at baseline and periodically for a total of 14 months. The primary outcome measure will be the change between baseline and follow up, in the severity of a validated PD assessment scale (the UPDRS part 3 motor score) after an overnight period free of conventional PD medication. Secondary measures will include adverse event reports, self completed questionnaires, and blood test results. Aside from these assessments, all patients will continue their regular PD medications throughout the trial with adjustments made only according to clinical need.
In a subgroup of patients (n=10), brain scans that assess the severity of PD, will be performed at both baseline and follow up to help understand possible mechanisms of action of Exenatide.
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||An Open Label, Single Site, 12 Month, Phase II, Randomised Controlled Trial Evaluating the Safety and Efficacy of Exendin-4 (Exenatide) in the Treatment of Patients With Moderate Severity Parkinson's Disease.|
- Change from baseline to 12 months & 14 months between patients on active Exenatide treatment and PD controls in respect of their UPDRS-off-medication motor subscore.
- Adverse event profile among patients treated with Exenatide compared with matched PD controls. Change from baseline to 12 months/ 14 months between patients on active treatment and PD controls in respect of list given below
- the UPDRS on medication motor subscore
- the UPDRS ADL subscore
- dyskinesia rating scale
- timed motor tests
- the Mattis Dementia rating scale
- the Montgomery & Asberg Depression rating scale
- the PDQ39
- the EQ-5D
- the NMS Quest
- the SCOPA Sleep scale
- the SCOPA AUT scale
- the Smell Identification test
- DAT (SPECT) scan appearances.
|Study Start Date:||July 2010|
|Estimated Study Completion Date:||March 2013|
|Estimated Primary Completion Date:||March 2013 (Final data collection date for primary outcome measure)|
|No Intervention: Control|
Exenatide, 5 micrograms twice a day for 1 month and 10 micrograms twice a day for 11 months. Total duration of treatment 12 months