A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of NBI-827104 in Subjects With Essential Tremor
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 31
- 试验地点
- 1
- 主要终点
- Change from Baseline to Day 28 of Each Treatment Period in Amplitude at Peak Frequency of Postural Tremor
研究概览
简要总结
The main objective of this study is to evaluate the efficacy, safety, and tolerability of NBI-827104 in adults with essential tremor.
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)
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female subjects, 18 to 75 years of age, inclusive at screening.
- •Body mass index (BMI) between 18 and 35 kg/m², inclusive, at screening.
- •Diagnosis of Essential Tremor (inclusive of Essential Tremor plus) as defined by the Movement Disorders Society Consensus Criteria for Tremor.
- •Confirmation of bilateral upper limb action tremor in the absence of overt dystonia, ataxia, or parkinsonism.
- •History of onset of tremor before 65 years of age.
- •Tremor Performance score of ≥ 2 on at least 2 of the 6 upper limb manoeuvres (Item 4) on the TETRAS Performance Subscale and a total TETRAS Performance score ≥15 at screening.
- •All women of childbearing potential and all males must practice effective contraception during the study and be willing and able to continue contraception for at least 90 days after their last dose of study drug.
排除标准
- •Evidence of any acute (at screening or prior to first dose) or chronic disease or condition that could interfere with, or for which the treatment might interfere with, the conduct of the study, or that would pose an unacceptable risk to the subject in the opinion of the investigator.
- •Have direct or indirect trauma to the nervous system within 3 months preceding the onset of tremor.
- •Have known history of other medical or neurological conditions that may cause or explain subject's tremor.
- •Have had prior magnetic resonance guided focused ultrasound or surgical intervention (e.g., deep brain stimulation, ablative thalamotomy or gamma knife thalamotomy).
- •Have a history or evidence of clinically significant cardiac conduction abnormality, uncontrolled bradyarrhythmia, heart failure, or long QT syndrome.
研究组 & 干预措施
Sequence 1
In treatment period 1 participants will receive increasing doses of NBI-827104 for 28 days. After a 14-day washout period, participants will receive matching placebo for 28 days in treatment period 2.
干预措施: NBI-827104 (Drug)
Sequence 1
In treatment period 1 participants will receive increasing doses of NBI-827104 for 28 days. After a 14-day washout period, participants will receive matching placebo for 28 days in treatment period 2.
干预措施: Placebo (Drug)
Sequence 2
In treatment period 1 participants will receive matching placebo for 28 days. After a 14-day washout period, participants will receive increasing doses of NBI-827104 for 28 days in treatment period 2.
干预措施: NBI-827104 (Drug)
Sequence 2
In treatment period 1 participants will receive matching placebo for 28 days. After a 14-day washout period, participants will receive increasing doses of NBI-827104 for 28 days in treatment period 2.
干预措施: Placebo (Drug)
结局指标
主要结局
Change from Baseline to Day 28 of Each Treatment Period in Amplitude at Peak Frequency of Postural Tremor
时间窗: Baseline and Day 28 of each treatment period
次要结局
- Change from Baseline in TETRAS Activities of Daily Living (ADL) Score(Baseline up to Day 28 of each treatment period)
- Clinical Global Impression of Change (CGI-C)(Up to Day 28 of each treatment period)
- Change from Baseline in Essential Tremor Rating Assessment Scale (TETRAS) Performance Score(Baseline up to Day 28 of each treatment period)