NCT07536269招募中2 期
A Phase 2, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Crinecerfont in Pediatric Participants 3 Months to <4 Years of Age With Classic Congenital Adrenal Hyperplasia
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 20
- 试验地点
- 11
- 主要终点
- Number of Participants With Treatment-emergent Adverse Events (TEAE)
研究概览
简要总结
The main objective of this study is to assess the safety and tolerability of crinecerfont in pediatric participants 3 months to <4 years of age with CAH.
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
- 3 Months 至 47 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Have a medically confirmed diagnosis of classic CAH (salt wasting or simple virilizing) due to 21-hydroxylase deficiency (21-OHD)
- •Have received at least 3 months of hydrocortisone treatment before Day 1, with a stable dose for at least 4 weeks before screening.
- •Have a body weight of at least 4.5 kilograms (kg) at screening.
- •Have a newborn screen that is otherwise normal except for elevated 17-OHP or any other abnormality on newborn screen that was cleared upon evaluation by a pediatric specialist.
排除标准
- •Have a known or suspected diagnosis of any of the other forms of classic CAH.
- •Have any condition besides CAH that requires chronic daily therapy with orally administered steroids.
- •Have any other clinically significant medical condition or chronic disease.
- •Note: Other protocol-defined inclusion and exclusion criteria may apply.
研究组 & 干预措施
Crinecerfont
Experimental
Participants with CAH will receive crinecerfont.
干预措施: Crinecerfont (Drug)
结局指标
主要结局
Number of Participants With Treatment-emergent Adverse Events (TEAE)
时间窗: Day 1 up to 28 weeks
次要结局
未报告次要终点
研究者
研究点 (11)
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