A Well-Controlled, Fixed-Dose Study of TEV-50717 (Deutetrabenazine) for the Treatment of Tics Associated With Tourette Syndrome
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 158
- 试验地点
- 56
- 主要终点
- Change From Baseline in the TTS of the YGTSS at Week 8 Between High-Dose TEV-50717-Treated Participants and Placebo-Treated Participants
研究概览
简要总结
Standard placebo-controlled, double-blind study design (TEV-50717 [low dose and high dose] vs. placebo in a 1:1:1 ratio) was chosen to determine whether study drug treatment results in a statistically significant effect on the tics in participants with TS.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 6 Years 至 16 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant weighs at least 44 pounds (20 kg) at baseline.
- •Participant meets the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5™) diagnostic criteria for TS and, in the opinion of the investigator, participant, and parent/legal guardian, the participant's active tics are causing distress or impairment.
- •Participant has a TTS of 20 or higher on the YGTSS at screening and baseline.
- •Participant is able to swallow study medication whole.
- •-Additional criteria apply, please contact the investigator for more information
排除标准
- •Participant has a neurologic disorder other than TS that could obscure the evaluation of tics.
- •The participant 's predominant movement disorder is stereotypy (coordinated movements that repeat continually and identically) associated with autism spectrum disorder.
- •Participant has clinically significant depression at screening or baseline.
- •Participant has a history of suicidal intent or related behaviors within 2 years of screening
- •Participant has a history of a previous actual, interrupted, or aborted suicide attempt.
- •Participant has a first-degree relative who has completed suicide.
- •Participant has a confirmed diagnosis of bipolar disorder, schizophrenia, or another psychotic disorder.
- •Participant has received Comprehensive Behavioral Intervention for Tics for TS or Cognitive Behavioral Therapy for obsessive-compulsive disorder (OCD) within 4 weeks of screening.
- •Participant has received treatment with deep brain stimulation, transmagnetic stimulation, or transcranial direct current stimulation within 4 weeks of the screening visit for reduction of tics.
- •Participant has a history of torsades de pointes, congenital long QT syndrome, bradyarrhythmias, or uncompensated heart failure.
- •Participant has participated in an investigational drug or device study and received investigational medicinal product (IMP)/intervention within 30 days or 5 drug half-lives of baseline, whichever is longer.
- •Participant is a pregnant or lactating female, or plans to be pregnant during the study.
- •-Additional criteria apply, please contact the investigator for more information
研究组 & 干预措施
TEV-50717 Low-Dose
TEV-50717 tablets BID up to 36 mg/day orally for a total of 8 weeks
干预措施: TEV-50717 (Drug)
TEV-50717 High-Dose
TEV-50717 tablets twice daily (BID) up to 48 milligrams (mg)/day orally for a total of 8 weeks
干预措施: TEV-50717 (Drug)
TEV-50717 High-Dose
TEV-50717 tablets twice daily (BID) up to 48 milligrams (mg)/day orally for a total of 8 weeks
干预措施: Placebo (Drug)
TEV-50717 Low-Dose
TEV-50717 tablets BID up to 36 mg/day orally for a total of 8 weeks
干预措施: Placebo (Drug)
Placebo
Placebo matched to TEV-50717 for a total of 8 weeks
干预措施: Placebo (Drug)
结局指标
主要结局
Change From Baseline in the TTS of the YGTSS at Week 8 Between High-Dose TEV-50717-Treated Participants and Placebo-Treated Participants
时间窗: Baseline, Week 8
YGTSS rating scale is a semi-structured clinician rating instrument that provides an evaluation of the number, frequency, intensity, complexity, and interference of motor and phonic tics. YGTSS is composed of 11 items: 5 items for motor tic severity, 5 items for vocal tic severity, and 1 item for impairment. Each item for motor tic severity and vocal is rated on a 6-point scale (0 for none to 5 to severe). MTSS is the sum of the 5 items for motor tic severity and VTSS is the sum of the 5 items for vocal tic severity. TTS is the sum of MTSS and VTSS, ranges from 0 (none/absent) to 50 (severe). Higher scores indicate greater severity/worse outcome. Least square (LS) mean and standard error (SE) was calculated using mixed-model repeated-measures (MMRM) with treatment group, week (3 levels: Weeks 2, 4, and 8), and the treatment group by week interaction as fixed effects; and baseline TTS, region, and age group at baseline (2 levels: 6 to 11 years, 12 to 16 years) as covariates.
次要结局
- Change From Baseline in the TS-PGII Score at Week 8 Between Low-Dose TEV-50717-Treated Participants and Placebo-Treated Participants(Baseline, Week 8)
- Change From Baseline in the Tourette Syndrome-Clinical Global Impression (TS-CGI) Score at Week 8 Between High-Dose TEV-50717-Treated Participants and Placebo-Treated Participants(Baseline, Week 8)
- Change From Baseline in the TTS of the YGTSS at Week 8 Between Low-Dose TEV-50717-Treated Participants and Placebo-Treated Participants(Baseline, Week 8)
- Change From Baseline in the TS-CGI Score at Week 8 Between Low-Dose TEV-50717-Treated Participants and Placebo-Treated Participants(Baseline, Week 8)
- Change From Baseline in the Tourette Syndrome-Patient Global Impression of Impact (TS-PGII) Score at Week 8 Between High-Dose TEV-50717-Treated Participants and Placebo-Treated Participants(Baseline, Week 8)
- Change From Baseline in the Children's Depression Inventory Second Edition (CDI-2; Parent Version and Self-reported Version) Total Score at Week 9(Baseline, Week 9)
- Change From Baseline in the Child and Adolescent Gilles de la Tourette Syndrome - Quality of Life (C&A-GTS-QOL) Activities of Daily Living (ADL) Subscale Score at Week 8 Between High-Dose TEV-50717-Treated Participants and Placebo-Treated Participants(Baseline, Week 8)
- Change From Baseline in the C&A-GTS-QOL ADL Subscale Score at Week 8 Between Low-Dose TEV-50717-Treated Participants and Placebo-Treated Participants(Baseline, Week 8)
- Number of Participants at Baseline and Week 9 With Any Suicidal Ideation or Suicidal Behavior According to the Columbia Suicide Severity Rating Scale (C-SSRS)(Baseline, Week 9)