A Placebo-Controlled, Double-Blind, Randomized, Cross Over Pilot Study Of The Efficacy And Tolerability Of Incobotulinum Toxin A (Xeomin®) As A Treatment For Focal Task-Specific Dystonia Of The Musician's Hand
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 21
- 试验地点
- 2
- 主要终点
- Physician Global Perception of Change - Blinded Rater 1
研究概览
简要总结
The investigational drug being studied in this protocol is Incobotulinumtoxin A (Xeomin®). Botulinum toxin (BoNT) prevents the release of the acetylcholine from peripheral nerves, inhibiting muscle contractions. BoNT is effective in relaxing overactive muscles. In musician's dystonia, the ability to reduce abnormally overactive muscles in the hand can be critical for the musical professional to continue his or her career. With the use of EMG/electrical stimulation and/or ultrasound guidance, the injector can precisely localize the individual muscles that are affected in this condition with great accuracy. Prior studies have shown that BoNT injections produce beneficial effects in forearm muscles, and less effect in shoulder or proximal arm muscles.
Possible risks in treating patients with BoNT include excessive weakness of the injected muscles. The drug may also affect non-targeted muscles. However these risks will be minimized during the screening period by carefully targeting the affected muscles and by administering low doses of BoNT. Small booster doses may be given at follow up visit (2, 4, 14 and 16-weeks after the primary injection date) if the initial injection was insufficient to produce sufficient efficacy in relief of the focal dystonia and did not produce excess weakness of the targeted muscle.
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
- Double (Participant, Investigator)
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 focal task-specific dystonia of one or both hands, selectively triggered by performance on a musical instrument.
- •Patients must have been evaluated by Dr. Frucht at the Movement Disorders Division of Mount Sinai Medical Center as part of their clinical care.
- •Patients whose performance on an instrument is directly linked to their occupation.
- •Patients must be between the ages of 18 and
- •Impairment in musical performance must be visible and demonstrable.
排除标准
- •Patients whose dystonia is not severe enough to interfere with musical performance in the opinion of a skilled examiner.
- •Patients with unstable medical conditions or psychiatric conditions.
- •Patients with a medical condition that precludes them from receiving BoNT injections.
研究组 & 干预措施
Placebo Then Botulinum Toxin
Placebo dose first does then boosters at week 2 and week 4 then 4 weeks washout, then Incobotulinumtoxin A first dose, with boosters at 2 week and week 4.
干预措施: incobotulinumtoxin A (Drug)
Placebo Then Botulinum Toxin
Placebo dose first does then boosters at week 2 and week 4 then 4 weeks washout, then Incobotulinumtoxin A first dose, with boosters at 2 week and week 4.
干预措施: Placebo (Drug)
Incobotulinumtoxin A Then Placebo
Incobotulinumtoxin A dose first does then boosters at week 2 and week 4 then 4 weeks washout, then Placebo first dose, with boosters at 2 week and week 4.
干预措施: incobotulinumtoxin A (Drug)
Incobotulinumtoxin A Then Placebo
Incobotulinumtoxin A dose first does then boosters at week 2 and week 4 then 4 weeks washout, then Placebo first dose, with boosters at 2 week and week 4.
干预措施: Placebo (Drug)
结局指标
主要结局
Physician Global Perception of Change - Blinded Rater 1
时间窗: at visit 4 (week 8)
The principal outcome measure was improvement in musical performance, measured by blinded rater, using physician global perception of change at post treatment compared to at 8 weeks. Categories are very much improved, much improved, minimal improved, no change, and minimal worse.
Physician Global Perception of Change - Blinded Rater 2
时间窗: at visit 4 (week 8)
The principal outcome measure was improvement in musical performance, measured by blinded rater, using physician global perception of change at post treatment compared to at 8 weeks. Categories are very much improved, much improved, minimal improved, no change, and minimal worse.
Rating of Overall Musical Performance - Blinded Rater 1
时间窗: baseline and at 8 weeks
Rating of overall musical performance based on comparison to baseline video at week 8. Rating from +3, very much improved to -3, very much worse. Higher score indicates more improvement.
Rating of Overall Musical Performance - Blinded Rater 2
时间窗: baseline and week 8
Rating of overall musical performance based on comparison to baseline video at week 8. Best Overall Musical Performance on 7 point scale from +3 Very much improved to -3 Very much worse. Higher score indicates more improvement.
Quantitative MIDI Analysis Comparison
时间窗: baseline at 24 weeks
The principal outcome measure will be improvement in musical performance, measured by self-rated questionnaire, quantitative Musical Instrument Digital Interface (MIDI) analysis and blinded high speed video analysis post treatment compared to baseline and 24 weeks.
Blinded High Speed Video Analysis Comparison
时间窗: baseline and at 24 weeks
The principal outcome measure will be improvement in musical performance, measured by self-rated questionnaire, quantitative MIDI analysis and blinded high speed video analysis post treatment compared to baseline and 24 weeks.
次要结局
- Change in Perceived Stress Scale (PSS)(baseline and at 8 weeks)
- Change in Medical Research Council (MRC) Scale(baseline and at 8 weeks)
- Change in Motor Strength Comparison(baseline and at 8 weeks)
研究者
David M. Simpson
Professor
Icahn School of Medicine at Mount Sinai