Prospective, Randomized, Double-blind, Placebo-controlled, Parallel-group Trial With an Open-label Period to Investigate the Efficacy and Safety of NT 201 in the Unilateral and Bilateral 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
- 78
- 试验地点
- 27
- 主要终点
- Change from study baseline to Week 6 in maximum tremor amplitude measurement at wrist level during the unilateral treatment period
研究概览
简要总结
The purpose of this study is to determine whether a single treatment with NT 201 (botulinum toxin) is superior to placebo (no medicine) for one-sided treatment of essential tremor in the motor-dominant arm (Unilateral Period). Participants will be assigned to the treatment groups by chance and neither the participants nor the research staff who interact with them will know the allocation.
The following treatment cycle will investigate the safety and tolerability of two-sided treatment with NT 201 (botulinum toxin) (Open Label Bilateral Period). All participants will receive NT 201 treatment.
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
- •- Score of ≥ 2 (at least 1 cm tremor amplitude) in at least two out of three maneuvers of test item 4 (upper limb tremor) of the TETRAS performances scale confirmed by an independent TETRAS expert by means of video assessment.
排除标准
- •History or presence of day-to-day fluctuations in ET which would jeopardize meaningful tremor assessment over time, e.g. severe tremor on one day and minimal or no tremor on another day.
- •Other neurological signs, such as dystonia, ataxia, or parkinsonism, which in the judgment of the investigator could interfere with the ET diagnosis and/or assessment of ET in ULs.
- •Tremor types other than ET
研究组 & 干预措施
Placebo
Unilateral Treatment Period (1 treatment cycle): subjects to receive unilateral intramuscular placebo injection into muscles of the motor-dominant upper limb.
Open Label Bilateral Treatment Period (1 treatment cycle): subjects to receive bilateral intramuscular injection of NT 201 (130-165 units per arm) into muscles of both upper limbs.
干预措施: NT 201 (Drug)
NT 201 (IncobotulinumtoxinA, Xeomin)
Unilateral Treatment Period (1 treatment cycle): subjects to receive unilateral intramuscular injection of NT 201 (130-165 units) into muscles of the motor-dominant upper limb.
Open Label Bilateral Treatment Period (1 treatment cycle): subjects to receive bilateral intramuscular injection of NT 201 (130-165 units) into muscles of both upper limbs.
干预措施: NT 201 (Drug)
Placebo
Unilateral Treatment Period (1 treatment cycle): subjects to receive unilateral intramuscular placebo injection into muscles of the motor-dominant upper limb.
Open Label Bilateral Treatment Period (1 treatment cycle): subjects to receive bilateral intramuscular injection of NT 201 (130-165 units per arm) into muscles of both upper limbs.
干预措施: Placebo (Drug)
结局指标
主要结局
Change from study baseline to Week 6 in maximum tremor amplitude measurement at wrist level during the unilateral treatment period
时间窗: From baseline to week 6
Unilateral Treatment Period: Change From Baseline to Week 6 in Maximum Tremor Amplitude of the Wrist
时间窗: Baseline up to Week 6
TremorTek kinematic analytic investigational device is combination of computer software and wearable movement sensors that allows to collect motion data when placed on individual's arm to quantify tremor in various muscle groups that impact shoulder, elbow, wrist. This assessment system was used to measure maximum angular tremor amplitude at wrist of injected limb (unit: degrees). Angular tremor amplitude was measure of tremor severity. Reduction of maximum angular tremor amplitude at wrist of injected limb represented tremor improvement.
次要结局
- Subject´s Global Impression of Change Scale [GICS] score of motor dominant UL at Week 6(Week 6)
- Change from study baseline to Week 6 in TETRAS ADL Functional Impact score(From baseline to week 6)
- Investigator´s Global Impression of Change Scale [GICS] score of motor dominant UL at Week 6(Week 6)
- Incidence of treatment-emergent AEs [TEAEs] related to treatment(From baseline to week 36)
- Change from study baseline to Week 6 in TETRAS Activities of Daily Living [ADL] UL score(From baseline to week 6)
- Change from Cycle 2 baseline to Week 6 in TETRAS Performance subscale score(Week 24 to week 36)
- Change from Cycle 2 baseline to Week 6 in TETRAS Performance dominant UL score(Week 24 to week 36)
- Change from study baseline to Week 6 in the Essential Tremor Rating Assessment Scale [TETRAS] Performance dominant upper limb [UL] score(From baseline to week 6)
- Change from Cycle 2 baseline to Week 6 in TETRAS ADL UL score(Week 24 to week 36)
- Change from Cycle 2 baseline to Week 6 in TETRAS ADL Functional Impact score(Week 24 to week 36)
- Unilateral Treatment Period: Change From Baseline to Week 6 in the Essential Tremor Rating Assessment Scale (TETRAS) Performance Dominant Upper Limb (UL) Score as Assessed by Investigator(Baseline up to Week 6)
- Unilateral Treatment Period: Change From Baseline to Week 6 in the TETRAS Activities of Daily Living (ADL) UL Score(Baseline up to Week 6)
- Unilateral Treatment Period: Change From Baseline to Week 6 in TETRAS ADL Functional Impact Score(Baseline up to Week 6)
- Bilateral Treatment Period: Change From Cycle 2 Baseline to Week 6 in TETRAS Performance Dominant UL Score(Baseline of Cycle 2 up to Week 6 (Cycle length = 12 weeks))
- Bilateral Treatment Period: Change From Cycle 2 Baseline to Week 6 in TETRAS Performance Subscale Score(Baseline of Cycle 2 up to Week 6 (Cycle length =12 weeks))
- Bilateral Treatment Period: Change From Cycle 2 Baseline to Week 6 in TETRAS ADL UL Score(Baseline of Cycle 2 up to Week 6 (Cycle length = 12 weeks))
- Bilateral Treatment Period: Change From Cycle 2 Baseline to Week 6 in TETRAS ADL Functional Impact Score(Baseline of Cycle 2 up to Week 6 (Cycle length = 12 weeks))
- Percentage of Subjects With At-least One Treatment Related Treatment-emergent Adverse Events (TEAEs)(Unilateral Treatment Period: From first injection in unilateral treatment period up to re-injection in Bilateral Treatment Period (Week 24); Bilateral Treatment Period: From re-injection in Bilateral Treatment Period at Week 24 up to end of study(Week 36))
- Unilateral Treatment Period: Subject's Global Impression of Change Scale (GICS) Score of Motor-dominant UL at Week 6(Week 6)
- Unilateral Treatment Period: Investigator's GICS Score of Motor-dominant UL at Week 6(Week 6)