2023-504628-24-00招募中2 期
A Phase 2b, Double-Blind, Randomized Extension Study to Evaluate the Long-Term Safety and Efficacy of PTC518 in Participants With Huntington’s Disease
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 172
- 试验地点
- 16
- 主要终点
- Evaluate blood tHTT protein levels over time
研究概览
简要总结
Safety - Evaluate the long-term safety of PTC518 in participants with Huntington’s disease (HD) Efficacy - Evaluate the pharmacodynamic effects of PTC518 through the reduction in blood total huntingtin (tHTT) levels
研究设计
- 分配方式
- Randomized
- 主要目的
- Study design
- 盲法
- Double (Analyst, Monitor, Investigator, Subject)
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •Ambulatory male or female participant aged 25 years and older, inclusive, who has completed the Treatment Period in Study PTC518-CNS-002-HD
- •Participant is willing and able to provide informed consent and comply with all protocol requirements
- •Women of childbearing potential (WOCBP) must agree to use highly effective methods of contraception during dosing and for 6 months after stopping the study medication.
- •Sexually active and fertile males must use a condom during intercourse while taking study drug and for 6 months after stopping study drug and should neither father a child nor donate sperm in this period. A condom is required to be used also by vasectomized men in order to prevent potential delivery of the drug via seminal fluid.
排除标准
- •Individuals are not eligible to participate in this study if they have not previously completed the Treatment Period in Study PTC518-CNS-002-HD.
结局指标
主要结局
Evaluate blood tHTT protein levels over time
Evaluate blood tHTT protein levels over time
Safety profile as characterized by TEAEs, laboratory abnormalities, vital signs, physical examination, C-SSRS
Safety profile as characterized by TEAEs, laboratory abnormalities, vital signs, physical examination, C-SSRS
次要结局
- Change in caudate volume as assessed by vMRI over time
- Change in cUHDRS scores over time
- Evaluate CSF mHTT protein levels over time
- Evaluate blood mHTT protein levels over time
研究者
Novartis Pharma Arzneimittel GmbH
Scientific
Novartis Pharma AG
研究点 (16)
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