A Multi-center, Study Investigating a New, Oral Growth Hormone Secretagogue (GHS)(AEZS 130, Formerly Ardana (ARD)-07) as a Growth Hormone (GH) Stimulation Test in Terms of Safety and Efficacy
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 101
- 试验地点
- 11
- 主要终点
- Receiver Operating Characteristic (ROC) Analysis on Peak GH (Growth Hormon) Concentrations
研究概览
简要总结
The diagnosis of growth hormone deficiency (GHD) in adults is established by laboratory testing in patients with an appropriate clinical history of hypothalamic pituitary disease. Two tests that are considered to be gold standard tests for the diagnosis of GHD are the insulin tolerance test (ITT) and growth hormone releasing hormone (GHRH) combined with L-arginine (L-ARG). However, these tests are either bothersome (given intravenously) to the patient or are linked with side effects. Therefore, an orally available compound like AEZS-130 (formerly ARD-07), if demonstrated to be safe and providing adequate sensitivity and specificity could be a welcome alternative and/or complement to the current available tests.
The intent was to recruit 40 adult GHD (AGHD) patients and 40 healthy control subjects into this trial, but the original sponsor (Ardana Biosciences Ltd.) discontinued the study for financial reasons before this was completed. At the time of withdrawal of GHRH from the market in 2008, 42 AGHD patients and 10 normal controls had completed the study at 9 US sites. This study reactivated to complete the remaining 30 matched control subjects.
Additionally upon agreement with the FDA in a Special Protocol Assessment (SPA), 10 additional adult growth hormone deficient and their matched control were planned to be enrolled into this trial for a total treated population of approximatively 100 subjects.
详细描述
Thirty control subjects (i.e., without AGHD) were matched to the 30 AGHD patients who were not previously matched. Matching was based upon gender, age, BMI, and estrogen status for females. They received one oral dose of AEZS-130 followed by serial blood draws for growth hormone (GH), insulin-like growth factor 1 (IGF-1) and pharmacokinetic (PK) determinations. There was no cross over due to the unavailability of GHRH (Geref) in the United States. Under Amendment #4 to this protocol, 10 additional AGHD subjects were to be enrolled and matched as described above.
Furthermore, the objective of the study was changed to delete comparison with L-ARG + GHRH.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Diagnostic
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
AEZS-130 ( formerly ARD-07)
A single oral administration of AEZS-130 (0.5 mg/kg po) as Growth Hormone Stimulation Test
干预措施: AEZS-130 (formerly ARD-07) (Drug)
L-ARG+GHRH
This trial was set up as a multi-center, randomized, cross-over study investigating AEZS-130 as a Growth Hormone Stimulation Tests in terms of safety and efficacy compared to L-ARG+GHRH. When GHRH became unavailable on the US market, this comparator arm was no longer available, which was addressed by Amendment No. 3 (version 27-March-2010). Control subject enrolled under Amendment No. 3 were not randomized as there was no cross-over due to unavailability of L-ARG+GHRH. These control subjects received only AEZS-130
干预措施: L-ARG+GHRH (Drug)
结局指标
主要结局
Receiver Operating Characteristic (ROC) Analysis on Peak GH (Growth Hormon) Concentrations
时间窗: GH sampling: pre-dose and 30, 45, 60, 75, 90, 120, 150 min post-dose
The primary endpoint for each individual is the peak GH concentration following AEZS-130 (macimorelin) administration.
次要结局
- Number of Participants With Drug Related Adverse Events (AEs)(14 days)
- Classification and Regression Tree (CART) Analysis of Peak Growth Hormone (GH) Following Macimorelin Administration(GH sampling: pre-dose and 30, 45, 60, 75, 90, 120, 150 min post-dose)
- Peak Insulin-Like Growth Factor (IGF)-1 Concentration Following Treatment(15 min. before macimorelin administration and at 150 min after macimorelin administration)