A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ATR-101 for the Treatment of Cushing's Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 4
- 试验地点
- 4
- 主要终点
- The Proportion of Subjects With Either a Normal 24-hr Urinary Free Cortisol (UFC) or a Reduction in 24-hr UFC of ≥ 50% Relative to Their Baseline Value
研究概览
简要总结
This is a Phase 2 multicenter, randomized, double-blind, placebo controlled study of ATR-101 to evaluate the efficacy and safety of orally-administered ATR-101 in adults with endogenous Cushing's syndrome. Following wash-out (if needed), all eligible subjects will enter an open-label intra-subject dose-escalation period of 8 weeks' duration, followed either by a double-blind randomized withdrawal period of 4 weeks' duration (if the subject meets randomization criteria) or by an additional open label dosing period of 4 weeks' duration (if the subject does not meet randomization criteria).It is anticipated that the overall duration of the study per subject will range from approximately 16-22 weeks.
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
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of endogenous Cushing's syndrome
- •Baseline UFC 1.3 to 10 × upper limit of normal (ULN)
- •If previous pituitary surgery, participants must be at least 3 months since surgery at the time of screening
- •BMI between 18 and 60 kg/m2, inclusive
排除标准
- •Pseudo-Cushing's syndrome, cyclic Cushing's syndrome or current iatrogenic Cushing's syndrome
- •Candidates for surgical treatment of Cushing's syndrome, unless surgery is not anticipated to occur during the study
- •Normal late night salivary cortisol or 24-hr urine free cortisol
- •Radiotherapy of the pituitary within 6 months
研究组 & 干预措施
ATR-101
During the 4-week randomized withdrawal period, eligible subjects will receive ATR-101 at the same dose level being used at the completion of the open-label dose-escalation period.
干预措施: ATR-101 (Drug)
Placebo
During the 4-week randomized withdrawal period, eligible subjects will receive a placebo that matches the same ATR-101 dose level being used at the completion of the open-label dose-escalation period.
干预措施: Placebo (Drug)
结局指标
主要结局
The Proportion of Subjects With Either a Normal 24-hr Urinary Free Cortisol (UFC) or a Reduction in 24-hr UFC of ≥ 50% Relative to Their Baseline Value
时间窗: Through Day 85
The number of subjects meeting the criterion was divided by the total number of subjects.
次要结局
- The Proportion of Subjects With a Normal 24-hr UFC(Through Day 57 and Day 85)
- The Proportion of Subjects With a Reduction in 24-hr UFC of ≥ 50% Relative to Their Baseline Value(Through Day 57 and Day 85)