A Phase 2 Study to Evaluate the Safety, Efficacy and Pharmacokinetics of SPR001 (Tildacerfont) in Children Aged 2 to 17 Years With Congenital Adrenal Hyperplasia (CAH)
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 67
- 试验地点
- 1
- 主要终点
- Number of Participants With Treatment-emergent Adverse Event (TEAE) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0
研究概览
简要总结
An investigation of the safety and efficacy of tildacerfont in participants with CAH.
详细描述
This is a Phase 2 open-label study with up to 10 cohorts that will evaluate the safety, efficacy, and PK of different tildacerfont dosing regimens potentially up to 200mg QD for 12 weeks in children with classic CAH, and up to 400mg BID for 4 weeks in children and adults with classic CAH.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female subjects aged 2+
- •Diagnosis of CAH due to 21-hydroxylase deficiency (OHD) and/or elevated 17- hydroxyprogesterone (OHP) requiring ongoing GC replacement since diagnosis
- •Stable dose of GC replacement for at least 1 month prior to screening
排除标准
- •History of bilateral adrenalectomy or hypopituitarism
- •Clinically significant unstable medical conditions, illness, or chronic diseases
- •History of active bleeding disorders
研究组 & 干预措施
Cohort 1: Age 11-17 Treatment with Tildacerfont
50 mg daily for 12 consecutive weeks.
干预措施: Tildacerfont (Drug)
Cohort 2: Age 11-17 Treatment with Tildacerfont
200 mg daily for 12 consecutive weeks.
干预措施: Tildacerfont (Drug)
Cohort 3: Age 2-10 Treatment with Tildacerfont
50, 100, or 200 mg daily for 12 consecutive weeks.
干预措施: Tildacerfont (Drug)
Cohort 4: Age >/= 18 Treatment with Tildacerfont
200 mg twice daily for 4 consecutive weeks.
干预措施: Tildacerfont (Drug)
Cohort 5: Age >/= 18 Treatment with Tildacerfont
300 or 400 mg twice daily for 4 consecutive weeks.
干预措施: Tildacerfont (Drug)
Cohort 6: Age 11-17 Treatment with Tildacerfont
200 mg twice daily for 4 consecutive weeks.
干预措施: Tildacerfont (Drug)
Cohort 7: Age 2-10 Treatment with Tildacerfont
200 mg twice daily for 4 consecutive weeks.
干预措施: Tildacerfont (Drug)
Cohort 8: Age 11-17 Treatment with Tildacerfont
300 or 400 mg twice daily for 4 consecutive weeks.
干预措施: Tildacerfont (Drug)
Cohort 9: Age 2-10 Treatment with Tildacerfont
300 or 400 mg twice daily for 4 consecutive weeks.
干预措施: Tildacerfont (Drug)
结局指标
主要结局
Number of Participants With Treatment-emergent Adverse Event (TEAE) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0
时间窗: 12 weeks
To evaluate safety of tildacerfont in participants with CAH as measured by number of subjects with adverse events following dosing by CTCAE version 5.0
次要结局
- Proportion of Participants Who Achieve a Reduction in Androstenedione (A4) or Reduction in Glucocorticoid (GC) Dosing(12 weeks)
- Proportion of Participants With Elevated Baseline A4 Who Achieve a Reduction in A4(4 weeks)
- Proportion of Participants With Elevated Baseline A4 Who Achieve a Reduction in A4 Who Achieve A4 Normalization(4 weeks or 12 weeks)