A Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Assess Tolerability, Safety, Pharmacokinetics and Effect of AZP2006 on Cerebrospinal Fluid Biomarkers in 36 Patients With Progressive Supranuclear Palsy
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 36
- 试验地点
- 3
- 主要终点
- The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
研究概览
简要总结
A phase 2 study to assess tolerability, safety, pharmacokinetics and effect of AZP2006 at different doses versus placebo on cerebrospinal fluid biomarkers in 36 patients with progressive supranuclear palsy. The patient study duration is 29 weeks including a washout period.
详细描述
This is a randomized, double blind, placebo controlled, parallel group study comparing 2 doses of AZP2006 (60 mg Once daily [QD] during the 12-week treatment period or 80 mg for 10 days followed by 50 mg QD) with placebo in 36 men and women aged ≥40 years and ≤80 years and diagnosed with probable or possible PSP according to Movement Disorder Society PSP (MDS-PSP) criteria.
Patients were assessed for eligibility at baseline. Eligible patients were randomized on
Day -1 in a 1:1:1 ratio into one of the following 3 study intervention groups based on a randomization scheme with blocks stratified by center using an interactive web response system (IWRS):
- 60 mg AZP2006/day during the 12-week treatment period;
- 80 mg AZP2006/day for 10 days followed by 50 mg AZP2006/day;
- Placebo.
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
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 40 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female patients with probable or possible PSP
- •Patients must be stable with their medication for at least 30 days prior to the inclusion visit.
排除标准
- •Any history of clinically significant head trauma or cerebrovascular disease or recent history of substance abuse or alcohol abuse and deemed to be clinically significant by the Investigator.
- •History of deep brain stimulator (DBS) surgery other than sham surgery for DBS clinical study.
研究组 & 干预措施
60mg/day/84 days
Patients randomized in this arm will receive 60 mg of study investigational drug AZP2006 once daily during 84 days.
干预措施: AZP2006 oral solution (Drug)
80mg/day/10 days followed by 50mg/day/74 days
Patients randomized in this arm will receive 80 mg of study investigational drug AZP2006 once daily during 10 days followed by 50 mg of study investigational drug AZP2006 once daily during the next 74 days.
干预措施: AZP2006 oral solution (Drug)
Placebo/84 days
Patients randomized in this arm will receive placebo solution once daily during 84 days.
干预措施: Placebo oral solution (Drug)
结局指标
主要结局
The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
时间窗: From Day 1 of Day 84 (12 weeks)
To determine the Ctrough of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks
Number and percentage of patients who prematurely discontinue from the study due to adverse events (AEs)
时间窗: From Day 1 to Day 180
Incidence in pourcentage of treatment-emergent adverse events observed directly by investigator and adverse event spontaneously reported by the patient using concise medical terminology
次要结局
未报告次要终点