A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of NBI-98854 in Pediatric Subjects With Tourette Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 98
- 主要终点
- Change From Baseline to Week 6 in the Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS)
研究概览
简要总结
Phase 2, double-blind, placebo-controlled study to assess the safety and efficacy of NBI-98854 administered once daily (qd) for a total of 6 weeks of treatment. This study will enroll approximately 90 male and female pediatric subjects clinically diagnosed with Tourette Syndrome.
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 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Have a clinical diagnosis of Tourette Syndrome (TS)
- •Have at least moderate tic severity
- •Have TS symptoms that impair school, occupational, and/or social function
- •If using maintenance medication(s) for TS or TS spectrum diagnoses (e.g. obsessive-compulsive disorder [OCD], Attention-Deficit Hyperactivity Disorder [ADHD]), be on stable doses
- •Be in good general health
- •Adolescent subjects (12 to 17 years of age) must have a negative urine drug screen for amphetamines, barbiturates, benzodiazepine, phencyclidine, cocaine, opiates, or cannabinoids and a negative alcohol screen
- •Subjects of childbearing potential who do not practice total abstinence must agree to use hormonal or two forms of nonhormonal contraception (dual contraception) consistently during the screening, treatment and follow-up periods of the study
排除标准
- •Have an active, clinically significant unstable medical condition within 1 month prior to screening
- •Have a known history of long QT syndrome or cardiac arrhythmia
- •Have a known history of neuroleptic malignant syndrome
- •Have a cancer diagnosis within 3 years prior to screening (some exceptions allowed)
- •Have an allergy, hypersensitivity, or intolerance to VMAT2 inhibitors
- •Have a blood loss ≥250 mL or donated blood within 30 days prior to screening
- •Have a known history of substance dependence, substance (drug) or alcohol abuse
- •Have a significant risk of suicidal or violent behavior
- •Have initiated Comprehensive Behavioral Intervention for Tics (CBIT) during the screening period or at baseline or plan to initiate CBIT during the study
- •Have received an investigational drug within 30 days before screening or plan to use an investigational drug (other than NBI-98854) during the study
研究组 & 干预措施
Dose Group 2
Administered once daily for 6 weeks
干预措施: NBI-98854 (Drug)
Placebo
Administered once daily for 6 weeks
干预措施: Placebo (Drug)
Dose Group 1
Administered once daily for 6 weeks
干预措施: NBI-98854 (Drug)
结局指标
主要结局
Change From Baseline to Week 6 in the Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS)
时间窗: Baseline, Week 6
The YGTSS is designed to rate the overall severity of motor and phonic tic symptoms across a range of dimensions: number, frequency, intensity, complexity, and interference. The YGTSS was administered by the investigator (or qualified designee) using a computer-based structured clinical interview. The TTS is the sum of the 5 motor tic items and the 5 phonic (vocal) tic items and ranges from 0 to 50, with higher scores representing greater severity
次要结局
- Clinical Global Impression of Tourette Syndrome (CGI-TS) - Improvement Score at Week 6(Week 6)
- Change From Baseline to Week 6 in the YGTSS Global Tic Severity Score(Baseline, Week 6)
- Change From Baseline to Week 6 in the Rush Video-based Tic Rating Scale (RTRS) Total Score(Baseline, Week 6)
- Change From Baseline to Week 6 in the YGTSS Impairment Score(Baseline, Week 6)
- Participants Who Are a CGI-TS-Improvement Responder at Week 6(Week 6)
- Participants Who Are a YGTSS TTS Responder at Week 6(Baseline, Week 6)
- Change From Baseline to Week 6 in the Premonitory Urge for Tics Scale (PUTS) Total Score(Baseline, Week 6)
- Change From Baseline to Week 6 in the Clinical Global Impression of Tics (CGI-Tics) - Severity Score(Baseline, Week 6)