Treatment in Advanced Parkinson's Disease: Continuous Intrajejunal Levodopa INfusion VErsus Deep Brain STimulation
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 51
- 试验地点
- 1
- 主要终点
- Cost effectiveness in costs per changed unit on PDQ-39
研究概览
简要总结
Both Continuous intrajejunal Levodopa Infusion (CLI) and Deep Brain Stimulation (DBS) are accepted therapies for the treatment of advanced Parkinson's disease (PD). To date, no comparative studies have been executed. The INVEST study is an open label randomised controlled trial with cost-effectiveness as primary outcome. The main clinical outcome is quality of life; secondary outcomes are motor symptoms and neurological impairments, among others.
详细描述
Rationale: Both Continuous intrajejunal Levodopa Infusion (CLI) and Deep Brain Stimulation (DBS) are accepted therapies for the treatment of advanced Parkinson's disease (PD). As directly comparative studies are lacking, it is unknown whether one of the therapies is more effective. Besides, CLI seems to be more expensive. To determine the optimal treatment in advanced PD, a comparative study of CLI and DBS is warranted.
Hypothesis: We hypothesize that CLI is a more expensive therapy in advanced PD than DBS and that the surplus in costs is not cost-effective with regard to benefits for the patient and caregivers in quality of life, PD symptoms and adverse events.
Objective: To realize a cost-effective treatment strategy in advanced PD. Study design: Prospective, randomized, open label multicentre trial, with two additional patient preference treatment arms ("patient preference randomized trial").
Study population: Patients with PD who, despite optimal pharmacological treatment, have severe response fluctuations, dyskinesias, painful dystonia, or bradykinesia. A total of 66 patients will be randomized, at least 120 patients will be included in the patient preference arms.
Intervention: Patients will be randomized to DBS or CLI. For DBS treatment, 2 electrodes will be implanted in the brain. The electrodes are connected to an implanted pulse generator, which will be placed subcutaneously in the subclavian area. For CLI treatment, a tube will be placed in the jejunum via a percutaneous endoscopic gastrostomy (PEG). This tube is connected to an external pump that delivers the levodopa-gel.
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
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Idiopathic Parkinson's Disease with bradykinesia and at least two of the following signs; resting tremor, rigidity, and asymmetry;
- •Despite optimal pharmacological treatment, at least one of the following symptoms: severe response fluctuations, dyskinesias, painful dystonia or bradykinesia;
- •A life expectancy of at least two years.
排除标准
- •Age below 18 years
- •Previous PD-neurosurgery (e.g., DBS, pallidotomy, thalamotomy);
- •Previous CLI (through a PEG-tube or Nasal Jejuna| tube);
- •Hoehn and Yahr stage 5 at the best moment during the day;
- •Other severely disabling disease;
- •Dementia or signs of severe cognitive impairment
- •Psychosis;
- •Current depression;
- •Contraindications for DBS surgery, such as a physical disorder making surgery hazardous;
- •Contraindications for PEG surgery such as interposed organs, ascites and oesophagogastric varices, or for Duodopa;
- •Pregnancy, breastfeeding, and women of child bearing age not using a reliable method of contraception;
- •No informed consent;
- •Legally incompetent adults;
研究组 & 干预措施
continuous levodopa infusion
continuous intrajejunal infusion of levodopa-carbidopa
干预措施: Continuous intrajejunal infusion of levodopa-carbidopa (Drug)
deep brain stimulation
Bilateral deep brain stimulation (DBS) of the subthalamic nucleus (STN)
干预措施: deep brain stimulation (Device)
结局指标
主要结局
Cost effectiveness in costs per changed unit on PDQ-39
时间窗: 12 months
The costs per changed unit on the PDQ-39.
Cost-utility in costs per changed Quality Adjusted Life Year (QALY, years)
时间窗: 12 months
The costs per QALY. The EuroQol 5D-3L (EQ-5D; 5 questions, each score 1-3, providing a health state, to be translated with provided Valuation set) will be applied as the utility measure.
次要结局
- Patient satisfaction(12, 24 and 36 months)
- Patients attitude to treatment(12, 24 and 36 months)
- Medical costs(12, 24 and 36 months)
- Quality of life (on PDQ-39)(12, 24 and 36 months)
- Dyskinesia(12 and 36 months)
- Neuropsychologic functioning Clock(12 and 36 months)
- Psychiatric disease(12 and 36 months)
- Anxiety(12 and 36 months)
- Adverse effects(12, 24 and 36 months)
- Non-medical care costs(12, 24 and 36 months)
- Caregiver burden(12, 24 and 36 months)
- Motor symptoms: time in off and on-state(12, 24 and 36 months)
- Motor experiences of daily living(12, 24 and 36 months)
- Non-motor symptoms (Non Motor Symptom Checklist)(12, 24 and 36 months)
- Non-motor symptoms (SCOPA-AUT)(12, 24 and 36 months)
- Disability(12, 24 and 36 months)
- Cognitive functioning(12 and 36 months)
- Cognitive functioning Mattis(12 and 36 months)
- Neuropsychologic functioning WAIS IV(12 and 36 months)
- Neuropsychologic functioning Word Test(12 and 36 months)
- Neuropsychologic functioning Trail making(12 and 36 months)
- Non-motor symptoms (Rotterdam Symptom Checklist(12, 24 and 36 months)
- Neuropsychologic functioning Letter Fluency(12 and 36 months)
- Neuropsychologic functioning Color Word(12 and 36 months)
- Suicidality(12 and 36 months)
- Treatment cross-over(12, 24 and 36 months)
- Quality of life (on EQ-5D)(12, 24 and 36 months)
- Neuropsychologic functioning Reading(12 and 36 months)
- Neuropsychologic functioning Memory(12 and 36 months)
- Motor symptoms(12 and 36 months)
- PD-medication (levodopa-equivalent dose)(12, 24 and 36 months)
- Functional health status(12, 24 and 36 months)
- Neuropsychologic functioning Line Orientation(12 and 36 months)
- Neuropsychologic functioning BNT(12 and 36 months)
- Apathy(12, 24 and 36 months)
- Compulsive Disorders(12, 24 and 36 months)
- Depression(12 and 36 months)
- Complications and description of complications(12, 24 and 36 months)
- Stopping allocated treatment(12, 24 and 36 months)
- Treatment failure(12, 24 and 36 months)
研究者
J.M. Dijk
J.M. Dijk, MD, PhD, Neurologist, Principal Investigator
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)