The Effect of Deep Brain Stimulation of the Subthalamic Nucleus (STN-DBS) on Quality of Life in Comparison to Best Medical Treatment in Patients With Complicated Parkinson's Disease and Preserved Psychosocial Competence (EARLYSTIM-study)
试验速览
- 阶段
- 4 期
- 发起方
- 入组人数
- 251
- 试验地点
- 16
- 主要终点
- PDQ-39
研究概览
简要总结
Earlystim Study: Patients are randomized either to medical treatment or subthalamic stimulation. The observation period was 2 years. The primary outcome criterium: PDQ-39.
Post study Follow up studies: After the 24 months observation period also BMT patients could be operated and all patients will be observed for 10 years or longer to elucidate whether earlier stimulation has advantages (or drawbacks) compared to later stimulation.
详细描述
Parkinsons' disease is one of the most disabling chronic neurological diseases. It can be treated sufficiently until motor complications with fluctuations of mobility and dyskinesias develop. The quality of life and the social and occupational functioning is relentlessly deteriorating with longer disease duration once the complications of conservative therapy develop. High-frequency stimulation of the subthalamic nucleus especially improves the motor complications of Parkinson's disease and preliminary data suggest that also the quality of life and psychosocial handicap are improved. So far this therapy is only used for patients which have already undergone personal, professional and social degradation due to motor complications of the disease. The aim of this study is to assess the use of this therapy in earlier stages of the disease, when motor complications have just developed and before patients are significantly affected in their social and occupational functioning.
The main study (Earlystim) was finished in March 2012 and published in February 2013 (Schuepbach WM, Rau J, Knudsen K, et al. Neurostimulation for Parkinson's disease with early motor complications. N Engl J Med. Feb 14 2013;368(7):610-622.) Patients, who were treated with BMT only in the Earlystim Study were privileged to be operated after the 24 months and a follow up phase of 5 years was planned to elucidate whether earlier stimulation has advantages (or drawbacks) compared to later stimulation.
As operated patients fare better in terms of quality of life and other outcomes (see publication), it will be important to know if patients who are operated earlier keep an advantage in all thoses parameters over those who were operated later or if those operated later will catch up after surgery. Also the pattern of adverse events among earlier and later operated patients may differ. These issues can be addressed with the post-study follow-up (PSFU) studies of the patients of the Earlystim trial. The results of these investigations elucidate longterm issues of DBS in PD and may affect the recommendations of surgery for patients.
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 60 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Idiopathic Parkinson's Disease
- •Hoehn and Yahr stage ≤ 2.5 in the best "on"med condition
- •Disease duration > 4 years
- •Presence of fluctuations and/or dyskinesias for no more than 3 years
- •One of the two following forms of impairment:
- •Impairment in activities of daily living (UPDRS II > 6) due to PD-symptoms despite medical treatment in the "worst" condition or
- •Impairment of social and occupational functioning (measured with a modified SOFAS) due to PD-symptoms despite medical treatment (51-80%)
- •PDQ-39 completed
- •Written informed consent
- •For the patients in France a social security number is required
- •Exclusion Criteria
- •Major depression with suicidal thoughts (Beck Depression Inventory > 25)
- •Dementia (Mattis Score ≤ 130)
- •Acute psychosis
- •Need for nursing care
- •Any medical or psychological problems which may interfere with a smooth conduction of the study protocol (e.g. cancer with a limited life expectancy)
- •Drug or alcohol addiction
- •Surgical contraindications
- •Fertile women not using adequate contraceptive methods
- •Women who are pregnant or breast feeding
- •Illiteracy or insufficient language skills (German or French) to complete the questionnaires
- •Simultaneous participation in another clinical trial except that other trial does not affect the Earlystim study as approved and documented by the steering committee
排除标准
- 未提供
研究组 & 干预措施
DBS treatment
Patients in this arm are treated with Deep Brain Stimulation (DBS) of the Nucleus subthalamicus with the device Kinetra and Soletra (neurostimulator, Medtronic) and addtionally get best medical treatment
干预措施: Kinetra and Soletra (neurostimulator, Medtronic) (Device)
BMT treatment
Patients in this arm get best medical treatment only.
干预措施: Best Medical Treatment (Drug)
结局指标
主要结局
PDQ-39
时间窗: 24 months
Difference in the PDQ-39 summary index at 24 months compared to the baseline assessment.
次要结局
- Speech(24 months)
- Safety(24 months)
- UPDRS VI scale(24 months)
- SCOPA-PS(24 months)
- Mattis Dementia Scale(24 months)
- BDI scale(24 months)
- SF-36 scale(24 months)
- Sleeping-hours per day(24 months)
- Gait(24 months)
- UPDRS part III(24 months)
- UPDRS II scale(24 months)
- MADRS scale(24 months)
- BPRS scale(24 months)
- Ardouin Behaviour Scale(24 months)
- Starkstein-Apathy Scale(24 months)
- professional Fitness scale(24 months)
- pain (VAS) scale,(24 months)
- clinical global impression (CGI-GI) scale(24 months)
- "best"-state(24 months)
- "best" state dyskinesias(24 months)