A Multicenter Dose-Titration Open-Label Study of Nevanimibe Hydrochloride for the Treatment of Classic Congenital Adrenal Hyperplasia
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 15
- 试验地点
- 11
- 主要终点
- Percentage of Subjects Achieving Serum 17-OHP Targets
研究概览
简要总结
This is a multicenter, intra-subject dose-titration open-label study of nevanimibe hydrochloride (HCl) for the treatment of classic congenital adrenal hyperplasia (CAH). Following a Screening Period of approximately 2-14 weeks, eligible subjects will enter a Baseline Period of approximately 2-8 weeks and then a 16-week Treatment Period. It is anticipated that the overall duration of the study per subject will range from 24-42 weeks.
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 and/or historical documentation of elevated 17-OHP
- •Chronic glucocorticoid replacement therapy for at least 6 consecutive months prior to screening
- •Stable glucocorticoid and mineralocorticoid regimen for at least 4 weeks prior to screening and throughout the treatment period of the study
排除标准
- •Nonclassic CAH
- •Other causes of adrenal insufficiency
- •HIV, hepatitis B, or hepatitis C
- •AST or ALT >2x ULN, bilirubin or serum creatinine >1.5x ULN
研究组 & 干预措施
Nevanimibe hydrochloride
Ascending dose level of oral nevanimibe hydrochloride beginning with 500 mg BID up to 2000 mg BID
干预措施: Nevanimibe hydrochloride (Drug)
结局指标
主要结局
Percentage of Subjects Achieving Serum 17-OHP Targets
时间窗: Through Day 113
The primary efficacy endpoint was the overall response rate within each cohort, defined as the percentage of patients achieving serum 17-OHP targets as follows: * Men and postmenopausal women: 17-OHP ≤ 2x ULN * Premenopausal women: * Follicular phase: 17-OHP ≤ 2x follicular phase ULN * Luteal phase: 17-OHP ≤ (2x follicular phase ULN + (luteal phase ULN - follicular phase ULN))
次要结局
未报告次要终点