A Randomised, Double Blind, Placebo Controlled, Single Centre, 60 Week Trial of Exenatide Once Weekly for the Treatment of Moderate Severity Parkinson's Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 60
- 试验地点
- 1
- 主要终点
- Efficacy
研究概览
简要总结
This study is a clinical trial in patients with Parkinson's disease, of a drug called Exenatide which is already licensed for the treatment of patients with Type 2 Diabetes. There have been several groups that have confirmed that Exenatide has beneficial effects on nerve cells when tested in the laboratory, that raises the possibility that Exenatide may slow down or stop the degenerative process of Parkinson's disease. In an open label trial in patients with Parkinson's disease who self administered the drug for 1 year, we have previously shown that the drug is well tolerated and shows encouraging effects on the movement and non-movement aspects of the disease, even 2 months after patients stopped administering the drug. The next step is therefore to formally evaluate whether Exenatide really is a potential "neuroprotective" drug, i.e. stops the nerve cells dying in Parkinson's disease, by conducting a double blind, placebo controlled trial.
详细描述
This trial aims to generate further data to explore whether 48 weeks exposure to Exenatide has an advantage over placebo based on a standard validated assessment of Parkinson's disease severity (the MDS UPDRS part 3 motor subscale). This will be measured during the "practically defined OFF medication state" i.e. after patients have withheld their conventional PD medication overnight. The hypothesis is that Exenatide will be associated with reduced MDS UPDRS part 3 scores at the study end.
To further examine the safety and tolerability of 48 weeks exposure to Exenatide in patients with moderate severity PD.
To collect Pharmacokinetic data regarding the degree of penetration of Exenatide across the blood brain barrier. We hypothesise that any central effects of Exenatide will be mediated through penetration of Exenatide across the blood brain barrier. Data obtained from rodents suggests that blood brain barrier penetration is excellent.
We propose to use a simple parallel group randomised controlled trial design.
No double blind data to support the use of Exenatide are currently available therefore equipoise exists. The null hypothesis is that Exenatide (as Bydureon) has no effect on disease progression.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 25 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of Parkinson's disease.
- •Males or Females.
- •Hoehn and Yahr stage ≤ 2.5 in the On medication state.
- •Between 25 and 75 years of age.
- •On dopaminergic treatment with wearing off phenomena.
- •Ability to self-administer, or to arrange carer administration of trial drug.
- •Documented informed consent to participate.
排除标准
- •Diagnosis or suspicion of other cause for parkinsonism.
- •Body mass index <18.
- •Known abnormality on CT or MRI brain imaging considered likely to compromise compliance with trial protocol/DaTSCAN acquisition.
- •Concurrent dementia defined by a score lower than 120 on the Mattis Dementia Rating Scale.
- •Concurrent severe depression defined by a score >16 on the MADRS.
- •Prior intra-cerebral surgical intervention for Parkinson's disease.
- •Already actively participating in a trial of a device, drug or surgical treatment for Parkinson's disease.
- •Severe gastrointestinal disease (e.g. gastroparesis).
- •Previous exposure to Exenatide.
- •Severely impaired renal function with creatinine clearance <30ml/min.
- •History of pancreatitis.
- •Hyperlipidaemia.
- •History or suspicion of thyroid cancer
- •Known or suspected intolerance of DaTSCAN or Potassium Iodide administration.
- •Females that are pregnant or breast feeding.
- •Participants who lack the capacity to give informed consent
- •Any medical or psychiatric condition which in the investigator's opinion compromises the potential participant's ability to participate.
研究组 & 干预措施
Exenatide
Bydureon- 2mg administered subcutaneously once weekly
干预措施: Exenatide (Drug)
Placebo
Placebo, 2mg administered subcutaneously once weekly
干预措施: Placebo (Other)
结局指标
主要结局
Efficacy
时间窗: 60 weeks
The primary objective is to compare the effectiveness of Exenatide versus placebo on the MDS UPDRS part 3 motor subscale in the "practically defined OFF medication state" in patients with moderate severity PD.
次要结局
- Safety and tolerability(60 weeks)