A Placebo-controlled, Double-blind, Randomized, Cross Over Pilot Study of the Efficacy and Tolerability of Abobotulinum Toxin A (Dysport) as a Treatment for Task-dependent Upper Limb Tremor
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 发起方
- 入组人数
- 25
- 试验地点
- 1
- 主要终点
- Patient's Impression of Change
研究概览
简要总结
Task-dependent upper limb tremor is a disorder that frequently impairs patients' function and quality of life. Few studies exist that quantify the improvements in motor performance and disability with botulinum toxin (injections). The study team aims to perform an exploratory pilot trial using toxin Type A (Dysport) in 25 patients with task-dependent upper limb tremor, to refine quantitative measures of performance using blinded video analysis, demonstrate efficacy and tolerability of injections, and provide power calculations for a potential future multi-centered double blinded clinical trial.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Investigator, Outcomes Assessor)
盲法说明
Videos will be randomly provided to an outside assessor to determine benefit as a secondary outcome measure
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with task-dependent essential tremor in upper limb. Tremor is present during at least one of the study functional tasks i. pointing task (touch between nose and a pen held 2 ft. in front. ii. pouring task (cup: pouring water out/in). iii. feeding task (weighted fork from plate on table to mouth). iv. string instrument bowing or holding instrument in static position. v. computer mouse (simple vertical or horizontal movements).
- •Patients must be capable of performing informed consent.
- •Patients on stable medications for treatment of task-dependent essential tremor for 1 month prior to study entry and maintain them throughout the study.
- •Patients previously treated with BoNT of any type must have occurred no sooner than 12 weeks prior to study entry.
排除标准
- •Patients with no presence of tremor during study functional tasks.
- •Presence of tremor during the functional task is below minimum amplitude required to be considered for injection based upon investigator's clinical judgment.
- •Patients with unstable medical conditions or psychiatric conditions.
- •Patients with a medical condition that precludes them from receiving BoNT injections.
- •Pregnant women, or premenopausal women not willing to use contraceptive measures throughout the duration of the study.
- •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 evaluations.
研究组 & 干预措施
Abobotulinum toxin A
干预措施: Abobotulinum toxin A (Drug)
Placebo
干预措施: Placebo (Other)
结局指标
主要结局
Patient's Impression of Change
时间窗: Week 18
The principal outcome measure will be patient's impression of change using visual analog scale. Visual Analog Scale is a 100 point scale from 0 (no improvement) to 100 (normal use).
次要结局
- Physician Global Impression of Change(Week 6)
研究者
David M. Simpson
Professor
Icahn School of Medicine at Mount Sinai