NCT02781727已完成3 期
A Multicenter, Phase 3, Randomized, Open-label, Active-controlled, Parallel-group Trial Investigating the Safety, Tolerability, and Efficacy of TransCon hGH Administered Once a Week Versus Standard Daily hGH Replacement Therapy Over 52 Weeks in Prepubertal Children With Growth Hormone Deficiency (GHD)
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 162
- 试验地点
- 55
- 主要终点
- Annualized Height Velocity at 52 Weeks for Weekly Lonapegsomatropin and Daily hGH Treatment Groups
研究概览
简要总结
A 52 week trial of TransCon hGH, a long-acting growth hormone product, versus human growth hormone therapy. TransCon hGH will be given once-a-week, human growth hormone (hGH) will be given daily. Approximately 150 prepubertal, hGH-treatment naïve children (males and females) with GHD will be included. Randomization will occur in a 2:1 ratio (TransCon hGH : Genotropin). This is a global trial that will be conducted in Armenia, Australia, Belarus, Bulgaria, Georgia, Greece, Italy, New Zealand, Poland, Romania, Russia, Turkey, Ukraine, and the United States.
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
- 3 Years 至 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Prepubertal children with GHD (either isolated or as part of a multiple pituitary hormone deficiency) in Tanner stage 1 (Tanner 1982) aged:
- •Boys: 3-12 years, inclusive
- •Girls: 3-11 years, inclusive
- •Impaired height (HT) defined as at least 2.0 standard deviations (SD) below the mean height for chronological age and sex (HT SDS ≤ -2.0) according to the 2000 CDC Growth Charts for the United States Methods and Development, available at http://www.cdc.gov/growthcharts/
- •Diagnosis of GHD confirmed by 2 different GH stimulation tests, defined as a peak GH level of ≤10 ng/mL, determined with a validated assay
- •Bone age (BA) at least 6 months less than chronological age
- •Baseline IGF-1 level of at least 1 SD below the mean IGF-1 level standardized for age and sex (IGF-1 SDS ≤-1)
- •Written, signed informed consent of the parent(s) or legal guardian(s) of the subject and written assent of the subject (if the subject is able to read, understand, and sign)
排除标准
- •Children with a body weight below 12 kg
- •Prior exposure to recombinant hGH or IGF-1 therapy
- •Children with past or present intracranial tumor growth as confirmed by a sellar MRI scan (with contrast) at Screening (MRI results from up to 6 months prior to Screening may be accepted)
- •Children with psychosocial dwarfism
- •Children with idiopathic short stature
- •History or presence of malignant disease; any evidence of present tumor growth
- •Closed epiphyses
- •Major medical conditions and/or presence of contraindication to hGH treatment
- •Participation in any other trial of an investigational agent within 3 months prior to Screening
研究组 & 干预措施
TransCon hGH
Experimental
Once weekly subcutaneous injection of TransCon hGH
干预措施: Once weekly subcutaneous injection of TransCon hGH (Drug)
human growth hormone (Genotropin)
Active Comparator
Once daily subcutaneous injection of Genotropin
干预措施: Once daily subcutaneous injection of Genotropin (Drug)
结局指标
主要结局
Annualized Height Velocity at 52 Weeks for Weekly Lonapegsomatropin and Daily hGH Treatment Groups
时间窗: 52 weeks
Annualized height velocity (AHV) at 52 weeks for weekly lonapegsomatropin (TransCon hGH) and daily hGH treatment groups
次要结局
- Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability](52 Weeks)
- Annualized Height Velocity Over 52 Weeks for Weekly Lonapegsomatropin and Daily hGH Treatment Groups(Week 5, Week 13, Week 26, Week 39 and Week 52)
- Number of Participants With Treatment Emergent Anti-hGH Binding Antibody Formation(Start of study treatment through Week 52)
- Change in Height Standard Deviation Score Over 52 Weeks for the Weekly Lonapegsomatropin and Daily hGH Treatment Groups(Week 5, Week 13, Week 26, Week 39 and Week 52)
- Average IGF-1 Standard Deviation Score Over 52 Weeks for the Weekly Lonapegsomatropin and Daily hGH Treatment Groups(Week 13, Week 26, Week 39, and Week 52)
研究者
研究点 (55)
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