A Multicenter, Double-Blind, Placebo-Controlled, Randomized Withdrawal Study to Evaluate the Safety and Maintenance of Efficacy of Ecopipam in Children, Adolescents and Adults With Tourette's Disorder
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 216
- 试验地点
- 190
- 主要终点
- Time From Randomization to Relapse in Participants Greater Than and Equal to (>=) 6 and Less Than (<) 18 Years During the Double-Blind R/WD
研究概览
简要总结
This Phase 3 multicenter study evaluates the maintenance of efficacy, safety and tolerability of ecopipam tablets in children, adolescents and adults in the treatment of Tourette's Disorder (TD). The study includes an open-label period followed by a double-blind, placebo-controlled, randomized withdrawal period.
详细描述
Following a 28-day Screening period and Baseline visit, eligible subjects will be enrolled into the open-label Stabilization period comprised of a 4-week Titration phase to achieve a target steady-state dose of 1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam HCl) followed by an 8-week open-label Maintenance phase. Responders to ecopipam will be randomized to ecopipam 1.8 mg/kg/day (2 mg/kg/day ecopipam HCl) or placebo in a 1:1 fashion and enter the double-blind Randomized-Withdrawal (R/WD) period at Week 12. During the 12-week R/WD period, any subject meeting Relapse criteria will be withdrawn from blinded study medication and complete end of study assessments including safety follow up visits.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
盲法说明
This includes an open-label period followed by a double-blind, placebo-controlled, randomized withdrawal period.
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •≥ 6 years of age
- •≥ 18 kg (~ 40 lbs.)
- •TD diagnosis and both motor and vocal tics that cause impairment with normal routines
- •Minimum score of 20 on the YGTSS-R 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
- •Previous exposure to ecopipam
- •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
1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam HCl)
Ecopipam 11.2, 22.4, 33.6, 44.8, 67.2 and 89.6 mg tablets (containing 12.5, 25, 37.5, 50, 75 and 100 mg ecopipam HCl, respectively); 1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam HCl) target dose; oral administration daily in evenings.
干预措施: Ecopipam Hydrochloride (Drug)
Placebo during R/WD Phase
Matching Placebo tablets during R/WD period taken orally in the evening.
干预措施: Ecopipam Hydrochloride (Drug)
结局指标
主要结局
Time From Randomization to Relapse in Participants Greater Than and Equal to (>=) 6 and Less Than (<) 18 Years During the Double-Blind R/WD
时间窗: From randomization at Week 12 through the end of the double-blind R/WD period (up to Week 24)
Time to relapse defined as a loss of \>=50 percent (%) of the improvement experienced on the Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS) from Baseline to the last visit in the Open-Label Stabilization Period (Week 12), or initiation of additional medications to treat symptoms of Tourette's Disorder (TD), or requirement of hospitalization for worsening symptoms of TD in participants between the ages of \>= 6 and \<18 years for ecopipam compared to those receiving placebo during the double-blind, R/WD period. The YGTSS was a clinician-completed rating scale used to quantify overall tic severity in participants with TD as well as specific subdomains of tic number, frequency, intensity, complexity and interference. Each of these subdomains was scored, on a 0 to 5 scale, separately for motor and vocal tics and then summed across both motor and vocal tics to yield a total tic score ranging from 0 to 50. Higher scores represented more severe symptoms.
次要结局
- Time From Randomization to Relapse in All Participants During the Double-Blind R/WD Period(From randomization at Week 12 through the end of the double-blind R/WD period (up to Week 24))