Multicenter, Placebo-Controlled, Double-Blind, Randomized, Parallel-Group, Phase 2b Study to Evaluate the Efficacy and Safety of Ecopipam in Children and Adolescents With Tourette's Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 153
- 试验地点
- 70
- 主要终点
- Change From Baseline in the Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS) at Week 12
研究概览
简要总结
This study evaluates the effect of ecopipam tablets in children and adolescents in the treatment of Tourette's Syndrome (TS). Half of the participants will receive ecopipam tablets, while the other half will receive matching placebo tablets
详细描述
Multicenter, placebo-controlled, double-blind, randomized, parallel-group, Phase 2b study in pediatric subjects (aged greater than equal to 6 to less than 18 years of age) with TS. Following a 28-day Screening period and Baseline visit, eligible subjects will be randomized 1:1 to receive either ecopipam hydrochloride (HCl) or matching placebo for a 12 week treatment period. Doses will be titrated up and down from target dose of 2 mg/kg/day. Follow Up visit will be conducted after the last dose of study medication.
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, Care Provider, Investigator)
入排标准
- 年龄范围
- 6 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •≥ 6 and < 18 years of age
- •≥ 18 kg (~ 40 lbs.)
- •TS diagnosis and both motor and vocal tics that cause impairment with normal routines
- •Minimum score of 20 on the YGTSS-Total Tic Score
- •May not be taking any medications used to treat motor or vocal tics for at least 14 days prior to Baseline.
- •Effective contraception during the study and 30 days after last study dose for sexually active subjects
排除标准
- •Certain mood or psychiatric disorders (i.e., dementia, bipolar disorder, schizophrenia, major depressive disorder)
- •Unstable medical illness or clinically significant lab abnormalities
- •Risk of suicide
- •Pregnant or lactating women
- •Moderate to severe renal insufficiency
- •Hepatic insufficiency
- •Positive urine drug screen
- •Unstable doses for drugs to treat anxiety, depression, Attention Deficit Hyperactivity Disorder
- •Certain medications that would lead to drug interactions
- •Recent behavioral therapy
研究组 & 干预措施
Ecopipam HCI 2 mg/kg/day
Ecopipam HCl 12.5-, 50-, 75- and 100-mg tablets; 2 mg/kg/day target dose; oral administration daily in evenings
干预措施: Ecopipam (Drug)
Placebo
Matching Placebo tablets taken orally in the evening
干预措施: Placebo (Drug)
结局指标
主要结局
Change From Baseline in the Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS) at Week 12
时间窗: Baseline, Week 12
The YGTSS was a semi-structured clinical interview designed to measure the tic severity. This scale consisted of a tic inventory, with 5 separate rating scales to rate the severity of symptoms, and an impairment ranking. Ratings were made along 5 different dimensions on a scale of 0=none to 5=severe for motor and vocal tics, each including number, frequency, intensity, complexity, and interference. The YGTSS TTS was the summation of the severity scores of motor and vocal tics. The total tic score (TTS) ranged from 0 (none) to 50 (severe) with higher score represent more severe symptoms. A negative change from baseline indicates improvement.
次要结局
- Change From Baseline in Clinical Global Impression of Tourette Syndrome Severity (CGI-TS-S) at Week 12(Baseline, Week 12)