An Open-Label, Randomized, Parallel Group Study to Assess the Safety and Efficacy of Hectorol® (Doxercalciferol Capsules) in Pediatric Patients With Chronic Kidney Disease Stages 3 and 4 With Secondary Hyperparathyroidism Not Yet on Dialysis
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- Sanofi
- 入组人数
- 21
- 试验地点
- 46
- 主要终点
- Percentage of Participants Who Achieved 2 Consecutive >=30% Reductions in Intact Parathyroid Hormone From Baseline up to Week 12
研究概览
简要总结
Primary Objective:
Evaluated the effect of Hectorol® capsules in reducing elevated levels of intact parathyroid hormone (iPTH).
Secondary Objectives:
- Evaluated the safety profile of Hectorol® capsules versus Rocaltrol® (calcitriol) capsules.
- Determined the pharmacokinetic profile of 1,25-dihydroxyvitamin D2 after administration of Hectorol®.
详细描述
The total study duration per participant was approximately up to 28 weeks.
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
- 5 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female aged 5 to 18 years old.
- •Weight ≥15 kg.
- •Chronic kidney disease (CKD) Stage 3 or 4 not on dialysis, defined as glomerular filtration rate (GFR) between 15 and 59 mL/min/1.73m^2 (established by Schwartz equation) at Week -2 visit.
- •Intact parathyroid hormone (iPTH) value >100 pg/mL for CKD Stage 3 or >160 pg/mL for CKD Stage 4, at Week -2 visit.
- •Signed informed consent/assent form.
排除标准
- •The participant had a serum 25-hydroxyvitamin D level <30 ng/mL at screening.
- •The participant had a corrected calcium ≥10 mg/dL at the Week -2 visit.
- •The participant had a serum phosphorus >4.5 mg/dL for children 13 to 18 years of age; >5.8 mg/dL for children 5 to 12 years of age at the Week -2 visit.
- •The participant was anticipated to require maintenance hemodialysis within 3 months.
- •The participant used cinacalcet or vitamin D sterol therapies such as calcitriol, doxercalciferol, or paricalcitol within 14 days prior to the baseline visit.
- •The participant had a history of, or active, symptomatic heart disease within 12 months prior to the baseline (Week 0) visit.
- •The participant had a chronic gastrointestinal disease (ie, malabsorption, severe chronic diarrhea, chronic ulcerative colitis, or ileostomy).
- •The participant had primary hyperparathyroidism or has had a total parathyroidectomy.
- •The participant had an active malignancy.
- •The participant was unable to swallow a capsule in size similar to the Hectorol® and Rocaltrol® capsules.
- •The participant had a history of sensitivity or allergy to doxercalciferol, calcitriol or other vitamin D analogs.
- •The participant used aluminum or magnesium-based binders.
- •The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
研究组 & 干预措施
Hectorol
Hectorol (Doxercalciferol) was administered orally two to three times weekly dependent on participant age. A dose titration scheme was used to individualize the dose to the participant's iPTH management.
干预措施: Doxercalciferol (GZ427397) (Drug)
Rocaltrol
Rocaltrol (Calcitriol) was administered orally seven days/week. A dose titration scheme was used to individualize the dose to the participant's iPTH management.
干预措施: Calcitriol (Drug)
结局指标
主要结局
Percentage of Participants Who Achieved 2 Consecutive >=30% Reductions in Intact Parathyroid Hormone From Baseline up to Week 12
时间窗: Baseline (Day 1) up to Week 12
Blood samples were collected for assessment of iPTH levels. The percentage of participants meeting the iPTH \>=30% reduction from baseline at 2 consecutive study visits up to Week 12 was calculated. Two consecutive \>=30% reductions in iPTH from baseline up to Week 12 was defined as two consecutive 30% or greater reductions at any two consecutive measurements from baseline up to Week 12 with on-treatment strategy applied. The confidence interval (CI) was estimated using Clopper-Pearson method. The baseline value is defined as the last available value before the first dose of study treatment. Percentages are rounded off to the tenth decimal place.
次要结局
- Percent Change in Intact Parathyroid Hormone From Baseline to Weeks 12 and 24(Baseline (Day 1) to Weeks 12 and 24)
- Number of Hypercalcemia Events up to Weeks 12 and 24(Up to Weeks 12 and 24)
- Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs)(From first dose of study treatment (Day 1) up to 4 days after the last dose of study treatment; approximately 36 weeks)
- Maximum Observed Plasma Concentration (Cmax) of 1,25-Dihydroxyvitamin D2 at Week 8 or 10(Pre-dose, 1, 4, 7, and 24 hours post-dose at Week 8 or 10)
- Time to Maximum Plasma Concentration (Tmax) of 1,25-Dihydroxyvitamin D2 at Week 8 or 10(Pre-dose, 1, 4, 7, and 24 hours post-dose at Week 8 or 10)
- Area Under the Concentration-Time Curve From Time 0 to 24 Hours (AUC0-24h) of 1,25-Dihydroxyvitamin D2 at Week 8 or 10(Pre-dose, 1, 4, 7, and 24 hours post-dose at Week 8 or 10)
- Trough Plasma Concentration (Ctrough) of 1,25-Dihydroxyvitamin D2 at Week 8 or 10(Pre-dose, 1, 4, 7, and 24 hours post-dose at Week 8 or 10)