A Phase 2 Multicenter, Randomized, Double-blind, Placebo-controlled Study of BOTOX® (Botulinum Toxin Type A) Purified Neurotoxin Complex for the Treatment of Upper Limb Essential Tremor
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 174
- 试验地点
- 40
- 主要终点
- Number of Participants with Treatment Emergent Adverse Events (AEs)
研究概览
简要总结
Upper limb essential tremor (UL ET) is a movement disorder characterized by postural and/or kinetic tremor. It can cause difficulty with everyday tasks such as writing, pouring, and eating, and patients also experience associated social embarrassment. This study will assess how safe and effective BOTOX is in treating UL ET. Adverse events and change in disease activity will be evaluated.
BOTOX is an investigational drug being developed for the treatment of UL ET. Participants are randomly assigned to 1 of the 4 groups, called treatment arms. Each group receives different treatment. There is 1 in 2 chance that participants will be assigned to placebo. Around 174 participants, aged 18 to 80 years with UL ET will be enrolled in approximately 40 sites in North America.
Participants will receive BOTOX or placebo injections in Cycle 1 and Cycle 2. In Cycle 3, participants will receive unilateral or bilateral BOTOX injections. Each cycle is 12 weeks.
There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.
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
- Triple (Participant, Investigator, Outcomes Assessor)
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
- •Diagnosis of essential tremor in accordance with modified Tremor Investigation Group (TRIG) criteria as described in the protocol.
- •TREDS-Revised Scale (1-4 scale, whole numbers) unilateral score of >= 3 for the Tremor Disability Scale (TREDS) on any of the 7 unilateral items; no more than a single item score of 1 among the 7 unilateral items in the dominant limb.
- •TETRAS activities of daily living (ADL) (recorded on a 0-4 scale, whole numbers) minimum score of >= 3 on any of the 5 unilateral items; no more than a single item score of <=1 among the 5 unilateral items in the dominant limb.
- •At least one of the following criteria must also be met:
- •TETRAS UL score (0-4 scale, with 0.5 increments) of > 2 in the dominant limb on at least one of the 3 maneuvers OR
- •TETRAS Archimedes spiral task score (0-4 scale, with 0.5 increments) of > 2 in the dominant limb
排除标准
- •Any uncontrolled clinically significant medical condition other than the one under study.
- •Any medical condition that may put the participant at increased risk with exposure to BOTOX Purified Neurotoxin Complex.
研究组 & 干预措施
BOTOX/BOTOX Unilateral
Participants will receive BOTOX in Cycle 1 and Cycle 2 and unilateral BOTOX in Cycle 3
干预措施: BOTOX (Biological)
BOTOX/BOTOX Bilateral
Participants will receive BOTOX in Cycle 1 and Cycle 2 and bilateral BOTOX in Cycle 3
干预措施: BOTOX (Biological)
Placebo/BOTOX Unilateral
Participants will receive placebo in Cycle 1 and Cycle 2 followed by unilateral BOTOX in Cycle 3.
干预措施: BOTOX (Biological)
Placebo/BOTOX Unilateral
Participants will receive placebo in Cycle 1 and Cycle 2 followed by unilateral BOTOX in Cycle 3.
干预措施: Placebo for BOTOX (Drug)
Placebo/BOTOX Bilateral
Participants will receive placebo in Cycle 1 and Cycle 2 followed by bilateral BOTOX in Cycle 3.
干预措施: BOTOX (Biological)
Placebo/BOTOX Bilateral
Participants will receive placebo in Cycle 1 and Cycle 2 followed by bilateral BOTOX in Cycle 3.
干预措施: Placebo for BOTOX (Drug)
结局指标
主要结局
Number of Participants with Treatment Emergent Adverse Events (AEs)
时间窗: Up to approximately 36 weeks
An AE is defined as any untoward medical occurrence in a patient or clinical investigation in which a participant is administered a pharmaceutical product which does not necessarily have a causal relationship with this treatment.
Change From Baseline in Tremor Disability Scale-Revised (TREDS-R) Total Score Across 7 Unilateral Items
时间窗: Week 24
Tremor Disability Scale-Revised (TREDS-R) is a scale to assess activities of daily living (ADL) through a self-questionnaire asking participants to rate how easy or difficult it is to perform a particular activity. The scale consists of 20 items with each item rated on a scale of 1 (able to do the activity without difficulty) to 4 (cannot do the activity by yourself). 7 items included in this subset will be: use a spoon to drink soup, hold a cup of tea, pour milk from a bottle or carton, dial a telephone, pick up your change in a shop, insert an electric plug into a socket, unlock your front door with a key.
次要结局
- Change From Baseline in TETRAS Activity of Daily Living (TETRAS ADL) Total Score Across 5 Unilateral Items(Week 24)
- Change From Baseline in TETRAS Handwriting Score(Week 24)
- Change From Baseline in Clinical Global Impression of Severity (CGI-S)(Week 24)
- Change From Baseline in Essential Tremor Rating Assessment (TETRAS UL) Score(Week 24)
- Change From Baseline in TETRAS Archimedes Spiral Score(Week 24)
- Change From Baseline in Patient Global Impression of Severity (PGI-S)(Week 24)