Prospective, Randomized, Double-blind, Placebo-controlled, Parallel-group Study of the Efficacy and Safety of NT 201 in the Unilateral Treatment of Essential Tremor of the Upper Limb
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 30
- 试验地点
- 6
- 主要终点
- Change From Baseline to Week 4 in Maximum Angular Tremor Amplitude of the Wrist (Injected Limb)
研究概览
简要总结
The objective of this study is to assess the efficacy and safety of a single, kinematic-analysis-based intramuscular injection of NT 201, compared with placebo, in moderate to marked essential tremor of the upper limb.
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
- Double (Participant, Investigator)
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
- •Diagnosis of 'definite essential tremor' in accordance with modified TRIG criteria, as follows:
- •Bilateral postural tremor with or without kinetic tremor, involving hands and forearms, that is visible and persistent.
- •It is to be noted that:
- •Tremor of other body parts may be present in addition to upper limb tremor.
- •Bilateral tremor may be asymmetric.
- •Tremor is reported by patient to be persistent, although the amplitude may fluctuate.
- •First onset of essential tremor at least 6 months before screening with stability of the tremor symptoms over 4 weeks and in the opinion of the investigator definite diagnosis of essential tremor.
- •Moderate-to-marked upper-limb postural and/or kinetic tremor at wrist level, corresponding to Fahn-Tolosa-Marin upper-limb tremor rating of at least 2 categories (scale part C, items 16-23) in the limb to be treated between of 2 or higher.
- •Visible tremor at wrist level in at least one of the four positions/tasks used in kinematic assessments
- •Tremor deemed by the investigator to require a treatment with 30 - 200 U NT 201 for a treatment of up to three joints of the selected upper limb (wrist treatment mandatory).
- •Stable concomitant anti-tremor medication and no clinically relevant findings in routine laboratory examinations.
排除标准
- •Any neurological signs abnormal for the subject's age, other than the tremor itself and Froment's maneuver.
- •Exposure to the following tremorogenic drugs: Lithium, Valproic acid, Amiodarone, typical and atypical neuroleptics. Exposure to other than the listed tremorogenic or potentially tremorogenic drugs is allowed only if, in the opinion of the investigator, this will not interfere with the study drug evaluation. In these cases, a stable medication should be reached 4 weeks before screening and intended for the time during the study drug evaluation.
- •Trauma to the central nervous system or the nerves of the target limb within the three months preceding the onset of tremor.
- •Evidence of psychogenic origins of tremor.
- •Life habits (e.g. smoking, alcohol or substance abuse) prejudicial to study participation.
- •Prior surgery to treat tremor
- •Recent (16 weeks) treatment with any Botulinum toxin product for any reason.
- •Relevant recent or planned surgery or other specified relevant treatments and/or concomitant disorders.
研究组 & 干预措施
200 Units incobotulinumtoxinA (Xeomin)
Single injection cycle, total dose of up to 200 Units, intramuscular injection into muscles of wrist (mandatory) and shoulder and/or elbow (both optional).
干预措施: IncobotulinumtoxinA (Drug)
Placebo
Single injection cycle, intramuscular injection into muscles of wrist (mandatory) and shoulder and/or elbow (both optional).
干预措施: Placebo (Drug)
结局指标
主要结局
Change From Baseline to Week 4 in Maximum Angular Tremor Amplitude of the Wrist (Injected Limb)
时间窗: Baseline up to Week 4
No primary efficacy variables are defined for this study. One of the secondary outcome measures is used for CTgov protocol registration and results reporting purposes only. TremorTek is a tremor kinematic analytics investigational device which is used to measure maximum angular tremor amplitude at wrist of the injected limb (unit: degrees). Angular tremor amplitude is the measure of tremor severity. Reduction of angular tremor amplitude at wrist of the injected limb represents tremor improvement.
次要结局
- Change From Baseline to Week 4 in Maximum Log-transformed Accelerometric Tremor Amplitude at Wrist Level (Injected Limb)(Baseline up to Week 4)
- Change From Baseline to Week 4 in FTM Motor Performance Score (Items 11-15)(Baseline up to Week 4)
- Investigator's Global Impression of Change Scale (GICS) at Week 4(Week 4)
- Participant's Global Impression of Change Scale (GICS) at Week 4(Week 4)
- Change From Baseline to Week 4 in Fahn-Tolosa-Marin (FTM) Tremor Score in Injected Limb (Item 5 [Right Upper Extremity] or 6 [Left Upper Extremity])(Baseline up to Week 4)