A Randomized, Open-label, Controlled Study to Assess the Efficacy and Safety of Cinacalcet HCl in Pediatric Subjects With Secondary Hyperparathyroidism and Chronic Kidney Disease Receiving Dialysis
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- Amgen
- 入组人数
- 55
- 试验地点
- 1
- 主要终点
- Percentage of Participants Who Achieved a ≥ 30% Reduction From Baseline In Mean Plasma iPTH During Weeks 11 to 15
研究概览
简要总结
The primary objective was to evaluate the efficacy of cinacalcet for reducing the plasma intact parathyroid hormone (iPTH) level by ≥ 30%.
详细描述
This was a 24-week, randomized, multicenter, open-label, controlled study. Participants were randomized into one of two treatment arms; oral administration of cinacalcet daily in addition to standard of care treatment, or standard of care alone. Randomization was stratified by age group (6 to < 12 and 12 to < 18 years of age). All participants received standard of care which could include therapy with Vitamin D sterols, calcium supplementation, and phosphate binders.
Participants in both treatment groups who completed the 20-week treatment period and those who ended the study due to study closure were eligible to enroll in an open-label extension study (20140159; NCT02341417) for further safety follow-up.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 6 - < 18 years
- •Diagnosis of SHPT with the mean of the two consecutive central laboratory iPTH values ≥ 300 pg/mL during screening
- •Corrected calcium value of ≥ 8.8 mg/dL during screening
- •Diagnosis of CKD, receiving either hemodialysis or peritoneal dialysis, for ≥ 30 days prior to screening
- •Parent or legally acceptable representative has provided written informed consent and subject has provided written assent when required by institutional guidelines
排除标准
- •History of congenital long QT syndrome, second or third degree heart block, ventricular tachyarrhythmias or other conditions associated with prolonged QT interval
- •Corrected QT interval (QTc) > 500 ms, using Bazett's formula
- •QTc ≥ 450 to ≤ 500 ms, using Bazett's formula, unless written permission to enroll is provided by the investigator after consultation with a pediatric cardiologist
- •Use of grapefruit juice, herbal medications, or potent cytochrome P450 3A4 (CYP3A4) inhibitors (eg, erythromycin, clarithromycin, ketoconazole, itraconazole)
- •Use of concomitant medications that may prolong the QTc interval (eg, ondansetron, albuterol)
研究组 & 干预措施
Cinacalcet
In addition to standard of care participants received cinacalcet at a starting dose (based on dry body weight) of 0.20 mg/kg administered once a day by mouth. Dose adjustments and withholding were based on ionized calcium levels, plasma iPTH, and corrected calcium levels.
干预措施: Cinacalcet HCl (Drug)
Standard of Care
Standard of care therapy included the use of vitamin D sterols, calcium supplementation, and phosphate binders.
干预措施: Standard of Care (Dietary Supplement)
Cinacalcet
In addition to standard of care participants received cinacalcet at a starting dose (based on dry body weight) of 0.20 mg/kg administered once a day by mouth. Dose adjustments and withholding were based on ionized calcium levels, plasma iPTH, and corrected calcium levels.
干预措施: Standard of Care (Dietary Supplement)
结局指标
主要结局
Percentage of Participants Who Achieved a ≥ 30% Reduction From Baseline In Mean Plasma iPTH During Weeks 11 to 15
时间窗: Baseline and weeks 11 to 15
Intact parathyroid hormone (iPTH) levels were measured at weeks 11 and 15; the mean value from these 2 measurements was calculated. This endpoint was the primary endpoint in the US only.
Percentage of Participants Who Achieved a ≥ 30% Reduction From Baseline in Mean Plasma Intact Parathyroid Hormone During the Efficacy Assessment Period
时间窗: Baseline and the efficacy assessment period (EAP), weeks 17 to 20
Intact parathyroid hormone (iPTH) levels were measured at weeks 17, 18, 19 and 20; the mean value from these measurements was calculated. This endpoint was specified as the the primary endpoint in all countries except the United States (US). In the US this endpoint was specified as a secondary efficacy endpoint.
次要结局
- Percentage of Participants Who Achieved a Mean iPTH ≤ 300 pg/mL (31.8 Pmol/L) During Weeks 17 to 20(Efficacy assessment period, weeks 17 to 20)
- Percent Change in iPTH From Baseline to the Mean Value During Weeks 17 to 20(Baseline and weeks 17 to 20)
- Change in Corrected Serum Calcium From Baseline to the Mean Value During Weeks 17 to 20(Baseline and weeks 17 to 20)
- Change in Serum Phosphorus From Baseline to the Mean Value During Weeks 17 to 20(Baseline and weeks 17 to 20)