A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Chorea Associated With Huntington Disease
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 128
- 试验地点
- 1
- 主要终点
- Change From Screening Period Baseline to Maintenance Period in the Unified Huntington's Disease Rating Scale (UHDRS) Total Maximal Chorea (TMC) Score.
研究概览
简要总结
This is a Phase 3, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, and tolerability of valbenazine to treat chorea in participants with Huntington disease.
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
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Have a clinical diagnosis of Huntington Disease (HD) with chorea
- •Be able to walk, with or without the assistance of a person or device
- •Participants of childbearing potential who do not practice total abstinence must agree to use hormonal or two forms of nonhormonal contraception (dual contraception) consistently while participating in the study until 30 days (females) or 90 days (males) after the last dose of the study drug
- •Be able to read and understand English
排除标准
- •Have a history of previously established therapy with a VMAT2 inhibitor, in the judgement of the investigator
- •Have difficulty swallowing
- •Are currently pregnant or breastfeeding
- •Have a known history of long QT syndrome, cardiac tachyarrhythmia, left bundle-branch block, atrioventricular block, uncontrolled bradyarrhythmia, or heart failure
- •Have an unstable or serious medical or psychiatric illness
- •Have a significant risk of suicidal behavior
- •Have a history of substance dependence or substance (drug) or alcohol abuse, within 1 year of screening
- •If taking antidepressant therapy, be on a stable regimen
- •Have received gene therapy at any time
- •Have received an investigational drug in a clinical study within 30 days of the baseline visit or plan to use such investigational drug (other than valbenazine) during the study
- •Have had a blood loss ≥550 milliliters (mL) or donated blood within 30 days before the baseline visit
- •Had a medically significant illness within 30 days before baseline, or any history of neuroleptic malignant syndrome
研究组 & 干预措施
Valbenazine
Capsule, administered orally once daily for 12 weeks.
干预措施: Valbenazine (Drug)
Placebo
Capsule, administered orally once daily for 12 weeks.
干预措施: Placebo (Drug)
结局指标
主要结局
Change From Screening Period Baseline to Maintenance Period in the Unified Huntington's Disease Rating Scale (UHDRS) Total Maximal Chorea (TMC) Score.
时间窗: Baseline (average of screening and Day -1), maintenance (average of Weeks 10 and 12)
The TMC is part of the motor assessment of the UHDRS and measures chorea in 7 different body parts including the face, oral-buccal-lingual region, trunk and each limb independently. The TMC score is the sum of the individual scores and ranges from 0 to 28. A decrease in TMC scores indicates improvement in chorea symptoms.
次要结局
- Percent of Clinical Global Impression of Change (CGI-C) Responders at Week 12(Week 12)
- Percent of Patient Global Impression of Change (PGI-C) Responders at Week 12(Week 12)
- Change From Baseline to Week 12 in the Quality of Life in Neurological Disorders (Neuro-QoL) Upper Extremity Function T-Score(Baseline, Week 12)
- Change From Baseline to Week 12 in the Neuro-QoL Lower Extremity Function T-Score(Baseline, Week 12)