A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Dyskinesia Due to Cerebral Palsy
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 86
- 试验地点
- 39
- 主要终点
- Change in the Total Maximal Chorea (TMC) Score of the Unified Huntington Disease Rating Scale (UHDRS) from Baseline to the Average of the Week 12 and Week 14 assessments
研究概览
简要总结
The primary objective of this study is to evaluate the efficacy of valbenazine versus placebo on improving chorea in pediatric and adult participants who have dyskinesia due to cerebral palsy (DCP) with choreiform movements.
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 70 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Medically confirmed diagnosis of DCP (that is, a hyperkinetic movement disorder due to cerebral palsy [CP]) with choreiform movements.
- •Medical conditions are stable and expected to remain stable throughout the study.
排除标准
- •Participants will be excluded from the study if they meet any of the following criteria:
- •Are pregnant or breastfeeding.
- •Have a clinical diagnosis or history of dyskinesia due to condition other than CP.
- •Have inability to swallow soft foods, unless medications can be administered via gastrostomy/gastrojejunostomy tube.
- •Have any suicidal behavior or suicidal ideation in the year prior to screening or on Day
- •Is a substance abuser of any compound.
- •Known history of long QT syndrome or cardiac tachyarrhythmia, or clinically significant electrocardiogram (ECG) abnormalities.
研究组 & 干预措施
Placebo
Placebo dosed for 14 weeks (double-blind treatment period) followed by open-label valbenazine treatment.
Participants may opt to enter an open-label valbenazine treatment extension phase.
干预措施: Valbenazine (Drug)
Placebo
Placebo dosed for 14 weeks (double-blind treatment period) followed by open-label valbenazine treatment.
Participants may opt to enter an open-label valbenazine treatment extension phase.
干预措施: Placebo (Drug)
Valbenazine
Valbenazine dosed for 14 weeks (double-blind treatment period) followed by open-label valbenazine treatment.
Participants may opt to enter an open-label valbenazine treatment extension phase.
干预措施: Valbenazine (Drug)
结局指标
主要结局
Change in the Total Maximal Chorea (TMC) Score of the Unified Huntington Disease Rating Scale (UHDRS) from Baseline to the Average of the Week 12 and Week 14 assessments
时间窗: Baseline, Week 12 and Week 14
次要结局
- Change in the Clinical Global Impression of Severity (CGI-S) Score from Baseline to Week 14(Baseline, Week 14)
- Change in the Movement Disorders - Childhood Rating Scale (MD-CRS) Part I Score from Baseline to the Average of the Week 12 and Week 14 Assessments(Baseline, Week 12 and Week 14)
- Change in the Total Maximal Dystonia (TMD) Score of the UHDRS from Baseline to the Average of the Week 12 and Week 14 Assessments(Baseline, Week 12 and Week 14)
- Patient Global Impression of Improvement (PGI-I) Score at Week 14(Week 14)
- Caregiver Global Impression of Improvement (CaGI-I) Score at Week 14(Week 14)
- Clinical Global Impression of Improvement (CGI-I) Score at Week 14(Week 14)
- Goal Attainment Score at Week 14 Using the Goal Attainment Scale (GAS)(Week 14)
- Change in Pain Assessment from Baseline to Week 14 Using the Faces Pain Scale-Revised (FPS-R)(Baseline, Week 14)
- Change in the UHDRS Total Motor Score (TMS) from Baseline to the Average of the Week 12 and Week 14 Assessments(Baseline, Week 12 and Week 14)