Phase IIIB, International, Single Group, Open Study to Define an Optimal Monitoring of IGF-1 in Children Treated With NutropinAq, Using a Novel Capillary Blood Collection Method
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Ipsen
- 入组人数
- 251
- 试验地点
- 42
- 主要终点
- Insulin-Like Growth Factor I (IGF-I) Levels Measured Using the Timed Capillary Blood Spot Samples
研究概览
简要总结
The main purpose of this study is to establish an optimal monitoring regimen in NutropinAq treated children, using newly developed capillary blood spot IGF-1 measurement technology.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children under 18 with growth failure associated with inadequate growth hormone secretion, or Turner syndrome or chronic renal insufficiency.
排除标准
- •Children with closed epiphyses
- •Children with active neoplasm
- •Children with acute critical illness
研究组 & 干预措施
NutropinAq 10 mg/2 mL (30 IU)
Patients received daily subcutaneous (s.c.) injections of NutropinAq 10 milligrams (mg)/2 milliliters (mL) for 6 months. The therapeutic daily doses administered were as follows:
- GHD patients: 0.025 - 0.035 mg/ kilogram (kg) bodyweight
- TS patients: up to 0.05 mg/kg bodyweight
- CRI patients: up to 0.05 mg/kg bodyweight
Patients visited the study clinic for a baseline visit and for 2 other visits every 3 months (Weeks 12 and 24). Additional home assessments were made at Weeks 21, 22 and 23.
The investigator determined the dose administered to each patient, and it was recommended to perform the injection in the evening.
干预措施: Somatropin (rDNA origin) (Drug)
结局指标
主要结局
Insulin-Like Growth Factor I (IGF-I) Levels Measured Using the Timed Capillary Blood Spot Samples
时间窗: At Weeks 21, 22 and 23
Fingertip capillary blood was collected using filter paper cards for the assay of capillary blood spot IGF-I in line with the monitoring recommendations of the Lawson Wilkins Paediatric Endocrine Society (LWPES) for treatment with recombinant GH therapy in children. Capillary IGF-I assays were performed by the patient at home one day per week during Weeks 21, 22 and 23 only (same week day). The samples were scheduled in the evening prior to the injection of NutropinAq and between 7:00 and 9:00 the following morning. An extended window from 6:00 to 12:00 was allowed for defining protocol deviations. The number of capillary blood spot IGF-I measurements and the optimal timing of samples to assess the IGF-I status of NutropinAq treated patients was assessed. IGF-I measurements for the morning and evening sampling are presented.
次要结局
- Assessment of IGF-I Levels: Categorised by Weekly Timing (Weeks 21-23) and Daily Timing (Morning and Evening)(At Weeks 21, 22 and 23)
- Multivariate Linear Regression Analyses to Assess Factors Affecting the Variability of IGF-I Levels: Categorised by Time of Year, Calculated Age at Enrolment and Disease Condition(Up to Week 24)
- Change From Baseline at Week 24 in the IGF-I Levels as Measured by Capillary Blood Spot Method and Serum IGF-I Assay(Baseline to Week 24)
- Change From Baseline at Week 12 and Week 24 in Insulin-Like Growth Factor Binding Protein 3 (IGFBP3) Measurements(Baseline to Week 12 and Week 24)
- Change From Baseline at Week 24 in the Auxological Parameter Calculated Height SDS(Baseline to Week 24)
- Change From Baseline at Week 24 in the Auxological Parameter Calculated Weight SDS(Baseline to Week 24)
- Assessment of IGF-I Levels: Categorised by Sex and Prepubertal Status(At Weeks 21, 22 and 23)
- Multivariate Linear Regression Analyses to Assess Factors Affecting the Variability of IGF-I Levels: Categorised by Disease Condition and Location(Up to Week 24)
- Change From Baseline at Week 24 in the Auxological Parameter Height(Baseline to Week 24)
- Change From Baseline at Week 24 in the Auxological Parameter Weight(Baseline to Week 24)
- Extent of Exposure to NutropinAq Throughout the Study(Up to Week 24)
- Change From Baseline at Week 24 in the Auxological Parameter Annualised Growth Velocity(Baseline to Week 24)
- Percentage of Patients Rating the Overall Handling of the Administration Device, NutropinAq Pen, to Assess the Acceptability and Tolerance of NutropinAq and Its Pen(At Month 5)
- Change From Baseline at Week 24 in the Auxological Parameter Annualised Growth Velocity SDS(Baseline to Week 24)
- Posology of NutropinAq at Baseline (Visit 1) Summarised as Mean Dose(Visit 1 (Baseline))