Phase 2 Study of the Safety and Efficacy of CORT125134 in the Treatment of Endogenous Cushing's Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 35
- 主要终点
- Percentage of Participants With One or More Severe (≥Grade 3) Adverse Events
研究概览
简要总结
Cushing's syndrome is a relatively rare disorder caused by prolonged exposure to high levels of the glucocorticoid hormone cortisol. Cushing's syndrome may result from elevated endogenous or exogenous sources of cortisol. Endogenous Cushing's syndrome resulting from cortisol overproduction by the adrenal glands is the subject of this protocol.
Patients with exogenous Cushing's syndrome, which develops as a side effect of chronic administration of high doses of glucocorticoids, were not eligible for enrollment in this study.
The purpose of this study was to evaluate the safety and efficacy of CORT125134 for treatment of endogenous Cushing's syndrome. The multicenter study was conducted in the United States and in Europe.
详细描述
This was a Phase 2, open-label study with two dose groups, each with a two-step dose escalation, designed to evaluate the safety and efficacy of CORT125134 for the treatment of endogenous Cushing's syndrome. CORT125134 was administered orally once daily for 16 weeks with dose escalations occurring every 4 weeks.
Pharmacokinetics (PK) profiles were generated at every dose level. A data review committee reviewed PK and safety data and recommended the final plan for dose escalation in Group 2.
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
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Has a confirmed diagnosis of endogenous Cushing's syndrome.
- •Requires medical treatment of hypercortisolemia.
- •Meets at least one of the following criteria:
- •Has type 2 diabetes mellitus.
- •Has impaired glucose tolerance.
- •Has hypertension.
排除标准
- •Has non-endogenous source of hypercortisolemia
- •Has uncontrolled, clinically significant hypothyroidism or hyperthyroidism
- •Has poorly controlled hypertension
- •Has Stage ≥ 4 renal failure
研究组 & 干预措施
Group 1: Low-dose Group
100 mg/day for 4 weeks in Period 1, then 150 mg/day for 4 weeks in Period 2, then 200 mg/day for 4 weeks in Period 3. There was no washout between treatment periods. Period 3 was followed by a 4-week follow-up period. Per-protocol, Group 1 did not participate in treatment Period 4.
干预措施: CORT125134 (Drug)
Group 2: High-dose Group
250 mg/day for 4 weeks in Period 1, then 300 mg/day for 4 weeks in Period 2, then 350 mg/day for 4 weeks in Period 3, then 400 mg/day for 4 weeks in Period 4. There was no washout between treatment periods. Period 4 was followed by a 4-week follow-up period.
干预措施: CORT125134 (Drug)
结局指标
主要结局
Percentage of Participants With One or More Severe (≥Grade 3) Adverse Events
时间窗: Group 1: up to Week 16; Group 2: up to Week 20
All treatment-emergent adverse events with Common Terminology Criteria for Adverse Events (CTCAE) ≥Grade 3 (severe) were recorded and summarized.
Percentage of Participants With One or More Adverse Events
时间窗: Group 1: up to Week 16; Group 2: up to Week 20
All treatment-emergent adverse events were recorded and summarized.
次要结局
- Percentage of Participants With Hypertension Who Experience Improvement in Blood Pressure Following Treatment With CORT125134(Group 1: Week 12 or last observation; Group 2: Week 16 or last observation)
- Percentage of Participants With IGT / T2DM Who Experienced a ≥25% Reduction in AUCglucose Following Treatment With CORT125134(Before and 0.5, 1, 1.5, and 2 hours after a glucose drink at Week 12 or last observation (Group 1) or Week 16 or last observation (Group 2))