A Phase 2, Multicenter Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 10
- 试验地点
- 6
- 主要终点
- Number of Participants With Reduction of 17-hydroxyprogesterone to </= 2 Times the Upper Limit of Normal at Any Time Following 2 Weeks of Dosing With ATR-101
研究概览
简要总结
This is a Phase 2 multicenter, single-blind, multiple dose study to evaluate the safety and efficacy of orally administered ATR-101 in subjects with classic congenital adrenal hyperplasia (CAH). Treatment duration will range from a minimum of approximately 2 months to 6 months per subject. A subject may receive a minimum of one dose level or up to a maximum of 5 dose levels, in sequentially increasing dose strengths. Each dose level will last 28 days.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •Documented historical diagnosis of classic CAH due to 21-hydroxylase deficiency based on: Documented genetic mutation in the CYP21A2 enzyme consistent with a diagnosis of classic CAH, or historical documentation of elevated 17-hydroxyprogesterone
- •Biochemical marker of disease status of 17-hydroxyprogesterone ≥ 4 times the upper limit of normal
- •Chronic glucocorticoid replacement therapy for at least 6 consecutive months
- •Stable glucocorticoid and mineralocorticoid regimen for at least 1 month
排除标准
- •Non-classic CAH
- •Other causes of adrenal insufficiency
- •Surgery within the previous 3 months prior to screening or planned surgery during study participation
- •History of active cancer requiring medical or surgical therapy within the past 6 months
研究组 & 干预措施
ATR-101
Ascending dose levels of ATR-101 beginning with 125 mg by mouth twice per day up to 1000 mg twice per day.
干预措施: ATR-101 (Drug)
结局指标
主要结局
Number of Participants With Reduction of 17-hydroxyprogesterone to </= 2 Times the Upper Limit of Normal at Any Time Following 2 Weeks of Dosing With ATR-101
时间窗: Evaluated at baseline and day 15 of each dose level. Each subject will have up to 5 dose levels.
17-hydroxyprogesterone was measured predose in the morning at the beginning and end of each dose level.
次要结局
未报告次要终点