Exendin-4 as a treatment for Parkinson's disease- pilot trial

• Research type

  Research Study

• Full 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.

• IRAS ID

  37888

• Contact name

  Thomas Foltynie

• Sponsor organisation

  University College London

• Eudract number

  2009-018137-37

• ISRCTN Number

  Not Sbmitted

• Research summary

  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 can be given as a twice daily 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 from the National Hospital for Neurology 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. Aside from these assessments, all patients will continue their regular PD medications throughout the trial with adjustments made only according to clinical need. Secondary objectives of the trial are; to confirm the safety profile of Exenatide in PD patients that has been previously seen among Diabetes patients, and to evaluate any effects of Exenatide on other symptoms experienced by PD patients such as problems with memory or depression. In a subgroup of patients (n=10), brain scans that assess the severity or activity of PD, will be performed at both baseline and follow up to help understand possible mechanisms of action of Exenatide.

• REC name

  London - Hampstead Research Ethics Committee

• REC reference

  10/H0720/22

• Date of REC Opinion

  14 Apr 2010

• REC opinion

  Further Information Favourable Opinion