海克托罗对患有慢性肾脏病 3 期和 4 期继发性甲状旁腺功能亢进但尚未接受透析的儿科患者的安全性和有效性
2026年6月8日 更新者：Sanofi
一项开放标签、随机、平行的小组研究，以评估 Hectorol®（Doxercalciferol 胶囊）在患有慢性肾脏病 3 期和 4 期伴继发性甲状旁腺功能亢进症但尚未进行透析的儿科患者中的安全性和有效性
主要目标：
评估 Hectorol® 胶囊在降低完整甲状旁腺激素 (iPTH) 升高水平方面的作用。
次要目标：
- 评估 Hectorol® 胶囊与 Rocaltrol®（骨化三醇）胶囊的安全性。
- 确定服用 Hectorol® 后 1,25-二羟基维生素 D2 的药代动力学特征。
研究概览
详细说明
每个患者的总研究持续时间将大约长达 28 周。
研究类型
介入性
注册 (实际的)
21
阶段
- 第三阶段
联系人和位置
本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。
学习地点
-
-
Biobio
-
Concepción、Biobio、智利、4070038
- Investigational Site Number : 1520004
-
-
Reg Metropolitana de Santiago
-
Santiago、Reg Metropolitana de Santiago、智利、7500539
- Investigational Site Number : 1520003
-
-
-
-
Alabama
-
Birmingham、Alabama、美国、35233
- Children's of Alabama- Site Number : 8400022
-
-
California
-
Los Angeles、California、美国、90048
- Cedars-Sinai Medical Center- Site Number : 8400033
-
Sacramento、California、美国、95817
- University of California Davis Health- Site Number : 8400005
-
-
Connecticut
-
New Haven、Connecticut、美国、06510
- Yale University School of Medicine- Site Number : 8400029
-
-
Florida
-
Miami、Florida、美国、33136
- University of Miami Hospital- Site Number : 8400006
-
Miami、Florida、美国、33155
- Nicklaus Children's Hospital - Miami - Southwest 62nd Avenue- Site Number : 8400008
-
-
Illinois
-
Chicago、Illinois、美国、60612
- Rush University Medical Center- Site Number : 8400020
-
-
Minnesota
-
Minneapolis、Minnesota、美国、55454
- M Health Fairview University of Minnesota Medical Center - West Bank- Site Number : 8400014
-
-
New Jersey
-
Hackensack、New Jersey、美国、07601
- Hackensack Meridian Health - Hackensack University Medical Center- Site Number : 8400010
-
Morristown、New Jersey、美国、07962
- Goryeb Chidlren's Hospital- Site Number : 8400016
-
-
New York
-
New Hyde Park、New York、美国、11040
- Cohen Children's Medical Center- Site Number : 8400017
-
New York、New York、美国、10029
- The Mount Sinai Hospital- Site Number : 8400007
-
-
North Carolina
-
Durham、North Carolina、美国、27710
- Duke University Medical Center- Site Number : 8400034
-
Greenville、North Carolina、美国、27858
- East Carolina University- Site Number : 8400025
-
-
Pennsylvania
-
Pittsburgh、Pennsylvania、美国、15224
- UPMC Children's Hospital of Pittsburgh- Site Number : 8400028
-
-
South Carolina
-
Greenville、South Carolina、美国、29605
- Greenville Memorial Hospital- Site Number : 8400027
-
-
Tennessee
-
Nashville、Tennessee、美国、37292
- Vanderbilt University Medical Center- Site Number : 8400024
-
-
Texas
-
Houston、Texas、美国、77030
- Texas Children's Hospital- Site Number : 8400013
-
-
Utah
-
Salt Lake City、Utah、美国、84132
- University of Utah Health Hospital- Site Number : 8400026
-
-
Virginia
-
Richmond、Virginia、美国、23219
- Virginia Commonwealth University Medical Center- Site Number : 8400009
-
-
Wisconsin
-
Marshfield、Wisconsin、美国、54449
- Marshfield Medical Center - Marshfield- Site Number : 8400001
-
-
参与标准
研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。
资格标准
适合学习的年龄
1年 至 14年 (孩子、成人)
接受健康志愿者
不
描述
纳入标准：
- 5至18岁的男性或女性。
- 体重≥15 公斤。
- 慢性肾病 (CKD) 3 期或 4 期未进行透析，定义为肾小球滤过率 (GFR) 在 15 至 59 mL/min/1.73m^2 之间 （由 Schwartz 方程建立）在第 -2 周就诊。
- 在第 -2 周就诊时，完整甲状旁腺激素 (iPTH) 值 >100 pg/mL（对于 CKD 第 3 期）或 >160 pg/mL（对于 CKD 第 4 期）。
- 签署知情同意书/同意书。
排除标准：
- 患者的血清 25-羟基维生素 D 水平
- 患者在第 -2 周就诊时校正后的钙含量≥10 mg/dL。
- 患者 13 至 18 岁儿童的血清磷 >4.5 mg/dL； >5.8 mg/dL 对于第 -2 周访视时的 5 至 12 岁儿童。
- 患者预计需要在 3 个月内进行维持性血液透析。
- 患者在基线访视前 14 天内使用过西那卡塞或维生素 D 甾醇疗法，例如骨化三醇、多西骨化醇或帕立骨化醇。
- 患者在基线（第 0 周）就诊前 12 个月内有心脏病史或活动性症状性心脏病。
- 患者目前患有慢性胃肠道疾病（即吸收不良、严重慢性腹泻、慢性溃疡性结肠炎或回肠造口术）。
- 患者目前患有原发性甲状旁腺功能亢进症或进行了甲状旁腺全切除术。
- 患者患有活动性恶性肿瘤。
- 患者无法吞咽与 Hectorol® 和 Rocaltrol® 胶囊大小相似的胶囊。
- 患者有对多西钙化醇、骨化三醇或其他维生素 D 类似物敏感或过敏的病史。
- 患者目前使用铝基或镁基粘合剂。
上述信息并非旨在包含与患者可能参与临床试验相关的所有考虑因素。
学习计划
本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。
研究是如何设计的？
设计细节
- 主要用途：治疗
- 分配：随机化
- 介入模型：并行分配
- 屏蔽：无（打开标签）
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
|
实验性的：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.
|
药物剂型：胶囊 给药途径：口服
其他名称：
|
|
有源比较器：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.
|
药物剂型：胶囊 给药途径：口服
其他名称：
|
研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Percentage of Participants Who Achieved 2 Consecutive >=30% Reductions in Intact Parathyroid Hormone From Baseline up to Week 12
大体时间：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.
|
Baseline (Day 1) up to Week 12
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Percent Change in Intact Parathyroid Hormone From Baseline to Weeks 12 and 24
大体时间：Baseline (Day 1) to Weeks 12 and 24
|
Blood samples were collected for assessment of iPTH levels.
The percentage changes from baseline iPTH, the effects over the treatment period time was explored using a mixed model for repeated measures approach (MMRM) as appropriate.
The baseline value is defined as the last available value before the first dose of study treatment.
|
Baseline (Day 1) to Weeks 12 and 24
|
|
Number of Hypercalcemia Events up to Weeks 12 and 24
大体时间：Up to Weeks 12 and 24
|
Hypercalcemia was defined as albumin corrected serum calcium >10.2 milligrams per deciliter (mg/dL).
Here, data for number of hypercalcemia events are reported.
|
Up to Weeks 12 and 24
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs)
大体时间：From first dose of study treatment (Day 1) up to 4 days after the last dose of study treatment; approximately 36 weeks
|
An adverse event (AE) was any untoward medical occurrence in a participant or clinical investigation participant who was administered a pharmaceutical product, and which did not necessarily have a causal relationship with this treatment.
An AE occurred or was detected from the date the participant signed the informed consent form, irrespective of study periods without administration of the study treatment.
TEAEs were defined as the AEs that developed, worsened or became serious during the treatment-emergent period (defined as time from administration of study treatment [Day 1] to last administration of study treatment + 4 days).
SAE: Any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event.
|
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
大体时间：Pre-dose, 1, 4, 7, and 24 hours post-dose at Week 8 or 10
|
Blood samples were collected at specified timepoints after administration of doxercalciferol (Hectorol®) to determine Cmax.
Evaluation of the 1, 25-Dihydroxyvitamin D2 concentration-time data was obtained using non-compartmental methods.
As pre-specified in protocol pharmacokinetic (PK) parameters were assessed at Week 8 or Week 10 choice was as per the schedule availability of the site and the participants.
|
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
大体时间：Pre-dose, 1, 4, 7, and 24 hours post-dose at Week 8 or 10
|
Blood samples were collected at specified timepoints after administration of doxercalciferol (Hectorol®) to determine tmax.
Evaluation of the 1, 25-Dihydroxyvitamin D2 concentration-time data was obtained using non-compartmental methods.
As pre-specified in protocol PK parameters were assessed at Week 8 or Week 10 choice was as per the schedule availability of the site and the participants.
|
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
大体时间：Pre-dose, 1, 4, 7, and 24 hours post-dose at Week 8 or 10
|
Blood samples were collected at specified timepoints after administration of doxercalciferol (Hectorol®) to determine AUC0-24h.
Evaluation of the 1, 25-Dihydroxyvitamin D2 concentration-time data was obtained using non-compartmental methods.
As pre-specified in protocol PK parameters were assessed at Week 8 or Week 10 choice was as per the schedule availability of the site and the participants.
|
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
大体时间：Pre-dose, 1, 4, 7, and 24 hours post-dose at Week 8 or 10
|
Blood samples were collected at specified timepoints after administration of doxercalciferol (Hectorol®) to determine Ctrough.
Evaluation of the 1, 25-Dihydroxyvitamin D2 concentration-time data was obtained using non-compartmental methods.
As pre-specified in protocol PK parameters were assessed at Week 8 or Week 10 choice was as per the schedule availability of the site and the participants.
|
Pre-dose, 1, 4, 7, and 24 hours post-dose at Week 8 or 10
|
合作者和调查者
在这里您可以找到参与这项研究的人员和组织。
赞助
调查人员
- 研究主任：Clinical Sciences & Operations、Sanofi
研究记录日期
这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。
研究主要日期
学习开始 (实际的)
2017年1月19日
初级完成 (实际的)
2025年6月11日
研究完成 (实际的)
2025年6月11日
研究注册日期
首次提交
2016年8月4日
首先提交符合 QC 标准的
2016年8月4日
首次发布 (估计的)
2016年8月9日
研究记录更新
最后更新发布 (实际的)
2026年7月2日
上次提交的符合 QC 标准的更新
2026年6月8日
最后验证
2026年6月1日
更多信息
与本研究相关的术语
其他相关的 MeSH 术语
其他研究编号
- LPS14314
- U1111-1178-4657 (注册表标识符：ICTRP)
计划个人参与者数据 (IPD)
计划共享个人参与者数据 (IPD)？
是的
IPD 计划说明
合格的研究人员可以请求访问患者水平的数据和相关研究文件，包括临床研究报告、带有任何修订的研究方案、空白病例报告表、统计分析计划和数据集规范。
患者层面的数据将被匿名化，研究文件将被编辑以保护试验参与者的隐私。
有关赛诺菲数据共享标准、合格研究和请求访问流程的更多详细信息，请访问：https://vivli.org
药物和器械信息、研究文件
研究美国 FDA 监管的药品
是的
研究美国 FDA 监管的设备产品
不
此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.