A Phase 2b, Double-Blind, Randomized Extension Study to Evaluate the Long-Term Safety and Efficacy of Votoplam in Participants With Huntington's Disease
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 144
- 试验地点
- 1
- 主要终点
- Number of Participants With Treatment-emergent Adverse Events (TEAEs)
研究概览
简要总结
The primary goal of this study is to evaluate the long-term safety and pharmacodynamic effects of votoplam in participants with HD.
详细描述
Participants who completed the Treatment Period in the parent Phase 2a Study PTC518-CNS-002-HD (NCT05358717), fulfilled the enrollment criteria, and chose to enroll in this extension study will undergo baseline evaluations and be assessed for 54 additional months.
All participants will receive active votoplam in this extension study. Participants who received votoplam in the parent Study PTC518-CNS-002-HD will continue at the same dose level they received in that study in a blinded fashion (5, 10 milligrams [mg]). Participants who received placebo in the parent Study PTC518-CNS-002-HD will be allocated to a votoplam dose level according to the same dosing group in which they were previously randomized (5, 10 mg).
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 25 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant who has completed the Treatment Period in Study PTC518-CNS-002-HD.
排除标准
- •Participants who have not previously completed the Treatment Period in Study PTC518-CNS-002-HD.
- •Note: Other inclusion and exclusion criteria may apply.
研究组 & 干预措施
Votoplam 5 mg
Participants will receive Votoplam 5 mg tablets once daily orally for 48 months.
干预措施: Votoplam (Drug)
Votoplam 10 mg
Participants will receive votoplam 10 mg tablets once daily orally for 48 months.
干预措施: Votoplam (Drug)
结局指标
主要结局
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
时间窗: Baseline up to Month 54
Blood Total Huntingtin Protein (tHTT) Levels
时间窗: Baseline up to Month 52
次要结局
- Change From Baseline in Caudate Volume as Assessed Via Volumetric Magnetic Resonance Imaging (vMRI) at Month 48(Baseline, Month 48)
- Change From Baseline in Composite Unified Huntington's Disease Rating Scale (cUHDRS) Scores at Month 48(Baseline, Month 48)
- Cerebrospinal Fluid (CSF) Mutant Huntingtin Protein (mHTT) Levels(Baseline up to Month 48)
- Blood mHTT Protein Levels(Baseline up to Month 52)