Evaluation of Botulinum Toxin Injection Efficacy in the Treatment of Head Essential Tremor by a Multi-center, Randomized, Double-Blind, Parallel-group, Placebo-controlled Study
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 120
- 试验地点
- 18
- 主要终点
- clinical state improvement of patients evaluated by the Clinical Global Impression of Change (CGI)
研究概览
简要总结
Essential head tremor (HT) does not usually respond to drug treatment such as propranolol or primidone or to surgical treatment such as deep brain stimulation of the thalamic ventralis intermedius nucleus. Botulinum toxin (Btx) is widely used and efficient in the treatment of blepharospasm and cervical dystonia. Btx prevents the release of acetylcholine in synapses, leading to a reduction of pathological muscle movement. Very little information is available on its efficacy on HT. Two published reports suggested that local Btx A injections could be an effective and safe approach for treating HT. Given the scarcity of published data (small samples, open studies, different doses of Btx used, heterogeneous populations, several muscles injected, etc.), the aim of this study is to demonstrate the efficacy of Btx injections in essential HT by a multi-center, randomized, double-blind, parallel-group, placebo-controlled study.
Principal objective: To evaluate the efficacy of botulinum toxin injections in the treatment of head tremor.
Secondary objectives:
- - To evaluate the tolerance of botulinum toxin injections in the treatment of head tremor.
- - To evaluate the impact of botulinum toxin injections on the patients' quality of life and embarrassment.
- To evaluate the changes of tremor characteristics induced by a Btx treatment using a 3D accelerometer wireless portable system (Xsens MTw wireless motion tracker system) (tremor frequency, tremor displacement, mean peak amplitude, distance traveled) and a new video system device to analyze head tremor characteristics
详细描述
Type of study: multi-center, randomized, double-blind, parallel-group, placebo-controlled therapeutic clinical trial.
Number of centers: 19 centers : Clermont-Ferrand, Paris (Fondation Rothschild, APHP, Hôpital Lariboisière, Hôpital Avicenne de Bobigny), Toulouse, Lyon, Amiens, Bordeaux, Narbonne, Lille, Strasbourg, Nîmes, Marseille, Besançon, Aix, Montpellier, Pau,Poitiers
Medical product Botulinum toxin type A, 200U (BOTOX ® 200 Unités, Allergan)
Patients
Patients eligible for inclusion will be randomized to one of the two groups:
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 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients suffering from a head tremor without (isolated) or with associated
- •tremor in any other body parts.
- •The HT must be troublesome for the patients (TRS 2 for the head tremor
- •severity item).
- •Patients never treated with botulinum toxin or not treated with botulinum toxin for this indication for at least 4 months.
- •Men or women aged from 18 to 80 years old.
- •Social security coverage.
- •Ability to provide informed consent.
排除标准
- •Patients with tremor from cerebellar syndrome (multiple sclerosis, etc.).
- •Patients with a predominant dystonic jerky or myoclonic head tremor using
- •the Tsui scale (>1).
- •Oral treatments for HT are allowed but must be stable during the study.
- •Any contra-indication to Botulinum toxin.
- •Women without efficient contraception.
- •Patients under supervision or (legal) guardianship.
研究组 & 干预措施
CONTROL GROUP
Patients eligible for inclusion will be randomized to one of the two groups:
- Experimental group: 60 patients will receive 2 injections of Btx A (BOTOX - Allergan), one on D0 and one at 12W (into each splenius capitis).
- Control group: 60 patients will receive 2 injections of placebo, one on D0 and one at 12W (into each splenius capitis).
干预措施: Placebo (Drug)
EXPERIMENTAL GROUP
Patients eligible for inclusion will be randomized to one of the two groups:
- Experimental group: 60 patients will receive 2 injections of Btx A (BOTOX - Allergan), one on D0 and one at 12W (into each splenius capitis).
- Control group: 60 patients will receive 2 injections of placebo, one on D0 and one at 12W (into each splenius capitis).
干预措施: BOTOX ® 200 Unités (Drug)
结局指标
主要结局
clinical state improvement of patients evaluated by the Clinical Global Impression of Change (CGI)
时间窗: at 18 weeks
clinical state improvement of patients evaluated by the Clinical Global Impression of Change (CGI) (improvement by at least two points) measured 6 weeks after the 2nd injection of Btx (at 18W).
% of responders defined with CGI
时间窗: at 18 weeks
The Fahn-Tolosa-Marin Tremor Rating Scale (TRS) (sub item head)
时间窗: at 12 weeks
% of responders defined with CGI
时间窗: at 6 weeks
The Fahn-Tolosa-Marin Tremor Rating Scale (TRS) (sub item head)
时间窗: at day 0
The Fahn-Tolosa-Marin Tremor Rating Scale (TRS) (sub item head)
时间窗: at 6 weeks
次要结局
- distance traveled measured by accelerometer(at 24 weeks)
- The Quality of life in Essential Tremor (QUEST)(at 24 weeks)
- tremor frequency measured by accelerometer(at 24 weeks)
- The Fahn-Tolosa-Marin Tremor Rating Scale (TRS) (sub item head)(at 24 weeks)
- The Essential Tremor Embarrassment Assessment (ETEA)(at 24 weeks)
- mean peak amplitude measured by accelerometer(at 24 weeks)
- The Essential Tremor Embarrassment Assessment (ETEA)(at 6 weeks)
- The Essential Tremor Embarrassment Assessment (ETEA)(at 12 weeks)
- The Essential Tremor Embarrassment Assessment (ETEA)(at 18 weeks)
- The Quality of life in Essential Tremor (QUEST)(at day 0)
- The Quality of life in Essential Tremor (QUEST)(at 6 weeks)
- The Quality of life in Essential Tremor (QUEST)(at 12 weeks)
- The Quality of life in Essential Tremor (QUEST)(at 18 weeks)
- tremor frequency measured by accelerometer(at day 0)
- tremor frequency measured by accelerometer(at 6 weeks)
- tremor frequency measured by accelerometer(at 12 weeks)
- tremor frequency measured by accelerometer(at 18 weeks)
- mean peak amplitude measured by accelerometer(at day 0)
- mean peak amplitude measured by accelerometer(at 6 weeks)
- mean peak amplitude measured by accelerometer(at 12 weeks)
- mean peak amplitude measured by accelerometer(at 18 weeks)
- distance traveled measured by accelerometer(at day 0)
- distance traveled measured by accelerometer(at 6 weeks)
- distance traveled measured by accelerometer(at 12 weeks)
- distance traveled measured by accelerometer(at 18 weeks)
- The Fahn-Tolosa-Marin Tremor Rating Scale (TRS) (sub item head)(at 18 weeks)
- The Essential Tremor Embarrassment Assessment (ETEA)(at day 0)