Subthalamic Nucleus Deep Brain Stimulation in Isolated Generalized or Segmental Dystonia: A Multicenter, Randomized, Double-blind, Sham-controlled, Parallel-group Trial
试验速览
- 阶段
- 不适用
- 入组人数
- 38
- 试验地点
- 1
- 主要终点
- The change from baseline to 3 months after stimulation of Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS) score
研究概览
简要总结
Dystonia is a group of movement disorders characterized by twisting, repetitive movements, or abnormal postures caused by involuntary muscle contractions and is characterized by a young age of onset and a high disability rate. Early intervention can reduce disability incidence, improve the patient's quality of life, and reduce the burden on families and society. Multiple international guidelines on dystonia have found deep brain stimulation (DBS) to be a safe and effective treatment for refractory dystonia. The globus pallidal internus (GPi) is the mostly widely used target for dystonia. However, there are limitations on the GPi DBS treatment, including slow onset of beneficial effects, poor improvement of axis symptoms, and potential stimulation-related side effects. Previous studies have described the highly successful use of subthalamic nucleus deep brain stimulation (STN DBS) in patients with refractory dystonia, suggesting that STN DBS is an effective and persisting alternative to pallidal deep brain stimulation. However, all STN DBS treated cases have been analyzed in open-label uncontrolled cohort studies, leading to limited data with a high level of evidence on the STN DBS in dystonia. Further, the investigators hypothesized STN has potentially more effectiveness when compared with GPi, and may be more power-saving and quick-acting. In this study, the investigators will organize a prospective randomized, double-blind, parallel-group, multicenter study comparing active versus sham stimulation in isolated segmental or generalized dystonia to evaluate the effectiveness and safety of STN DBS by measuring the impact on motor status, mental status, quality of life, the rate of response of the patients (the number of patients with ≥30% improvement in the movement score on the Burke-Fahn-Marsden Dystonia Rating Scale) and the rate of adverse events during the trial.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
The randomization will be conducted by the Coordination Center of Clinical Trials (CenTrial) in order to keep the researchers managing the data and the statistician blind to group assignment and the study conditions. All the clinical assessments done during the trial period will be double-blind, e.g., neither the patient not the clinician or study personnel involved in the scoring will not be aware of the condition of stimulation. All personnel, except the physician-programmer responsible for the DBS setting, will be blinded to the identity of the parameters. While programming the programmer will not sit face-to-face with the patient, but in another room adjusting the parameter wirelessly with the help of the attending doctor who will inform the programmer of the patient's reaction. The electrical parameters will be tested in all patients which means they all will perceive similar stimulation related sensations during each follow-up.
入排标准
- 年龄范围
- 14 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients must meet criteria for the diagnosis of isolated generalized or segmental dystonia, including idiopathic and inherited dystonia, as defined by the Phenomenology and Classification of Dystonia: A Consensus Update 2013;
- •Patients will be ≥ 14 years old;
- •The course of disease will be ≥ 3 years;
- •Patients will have:
- •Significant dystonia symptoms;
- •Compromised life quality;
- •Unsatisfactory response to oral treatment with anticholinergic agents antiepileptic agents, anti-dopamine agents, dopaminergic agents, or muscle relaxants;
- •Unsatisfactory response to or contraindication for previous botulinum toxin treatment; and
- •Ability to provide written informed consent.
排除标准
- •Patients with a diagnosis or probable diagnosis of acquired, compound, and complex dystonia, as defined by the Phenomenology and Classification of Dystonia: A Consensus Update 2013;
- •Previous brain surgery for dystonia;
- •Patients with cognitive impairment (MMSE score <24) or moderate-severe depressive disorder (BDI>25);
- •Patients with marked brain atrophy identified by magnetic resonance imaging (MRI) or computed tomography (CT);
- •Patients with other medical or psychiatric comorbidities that could increase the surgical risk or interfere with completion of the trial;
- •Patients with increased bleeding risk, or other factors contraindicating neurosurgery or general anesthesia;
- •Patients unable to cooperate with the assessments during the follow-up.
结局指标
主要结局
The change from baseline to 3 months after stimulation of Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS) score
时间窗: Baseline; 3months after stimulation
The scale consists of a movement and disability subscale with scores ranging from 0 to 120 and 0 to 30, respectively, higher scores indicating greater impairment.
次要结局
- Beck Depression Inventory-II (BDI)(Baseline; 1 week, 1 month and 3months after stimulation)
- Abnormal Involuntary Movement Scale (AIMS)(Baseline; 1 week, 1 month and 3months after stimulation)
- 36-item Short-Form General Health survey (SF-36)(Baseline; 1 week, 1 month and 3months after stimulation)
- The rate of response(1 week, 1 month and 3months after stimulation)
- Montreal Cognitive Assessment (MoCA)(Baseline; 3months after stimulation)
- Cambridge Neuropsychological Test Automated Battery (CANTAB)(Baseline; 3months after stimulation)
- Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS)(1 week and 1 month after stimulation)
- Beck Anxiety Inventory (BAI)(Baseline; 1 week, 1 month and 3months after stimulation)
- The rate of adverse event (AE)(Within 1 week after surgery; 1 week, 1 month and 3months after stimulation)
研究者
Bomin Sun
Director of Center for Functional Neurosurgery, Ruijin Hospital, Shanghai Jiaotong University School of Medicine
Ruijin Hospital