A Dose-finding Trial Evaluating the Effect and Safety of Once-weekly Treatment of Somapacitan Compared to Daily Norditropin® in Children With Short Stature Born Small for Gestational Age With no Catch-up Growth by 2 Years of Age or Older
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 62
- 试验地点
- 183
- 主要终点
- Height Velocity
研究概览
简要总结
The study compares 2 medicines used for the treatment of children who are born small and who stayed small: somapacitan given once a week (a new medicine) and Norditropin® given once a day (the medicine doctors can already prescribe).
Participants will either get somapacitan or Norditropin® - which treatment is decided by chance. Both participants and the study doctor will know which treatment the participants get. The study will last for 5 years. Participants will take either an injection once every week or once every day.
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
- 2 Years 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Pre-pubertal children, boys:
- •age between 2.5 and 11.0 years at screening.
- •testes volume below 4 ml.
- •Pre-pubertal children, girls:
- •age between 2.5 and 10.0 years at screening.
- •Tanner stage 1 for breast development (no palpable glandular breast tissue).
- •Born small for gestational age (birth length and/or weight below -2 standard deviation scores) (according to national standards).
- •Impaired height defined as at least 2.5 standard deviations below the mean height for chronological age and gender at screening according to the standards of Centers for Disease Control and Prevention at screening.
- •Impaired height velocity defined as annualized height velocity below the 50th percentile for chronological age and gender according to the standards of Prader calculated over a time span of minimum 6 months and maximum 18 months prior to screening.
- •No prior exposure to growth hormone therapy or Insulin-like Growth Factor-I (IGF-I) treatment.
排除标准
- •Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with standing height measurements.
- •Children with hormonal deficiencies including suspected or confirmed growth hormone deficiency according to local practise.
- •Current inflammatory diseases requiring systemic corticosteroid treatment for longer than 2 consecutive weeks within the last 3 months prior to screening.
- •Children requiring inhaled glucocorticoid therapy at a dose of greater than 400 μg/day of inhaled budesonide or equivalents for longer than 4 consecutive weeks within the last 12 months prior to screening.
- •Concomitant administration of other treatments that may have an effect on growth, e.g but not limited to methylphenidate for treatment of attention deficit hyperactivity disorder.
- •Diagnosis of attention deficit hyperactivity disorder.
- •Prior history or presence of malignancy including intracranial tumours.
研究组 & 干预措施
Somapacitan 0.16 mg/kg/week
Same treatment in main period (26 weeks) and extension period I (26 weeks). Dosage of Somapacitan during extension period II will be determined based on the data from main phase.
干预措施: Somapacitan (Drug)
Somapacitan 0.20 mg/kg/week
Same treatment in main period (26 weeks) and extension period I (26 weeks). Dosage of Somapacitan during extension period II will be determined based on the data from main phase.
干预措施: Somapacitan (Drug)
Somapacitan 0.24 mg/kg/week
Same treatment in main period (26 weeks) and extension period I (26 weeks). Dosage of Somapacitan during extension period II will be determined based on the data from main phase.
干预措施: Somapacitan (Drug)
Norditropin® 0.067 mg/kg/day
Same treatment in main period (26 weeks) and extension period I (26 weeks). Participants will switch to Somapacitan during extension period II. Dosage of Somapacitan during extension period II will be determined based on the data from main phase.
干预措施: Norditropin® (Drug)
Norditropin® 0.035 mg/kg/day
Same treatment in main period (26 weeks) and extension period I (26 weeks). Participants will switch to Somapacitan during extension period II. Dosage of Somapacitan during extension period II will be determined based on the data from main phase.
干预措施: Norditropin® (Drug)
结局指标
主要结局
Height Velocity
时间窗: Baseline (week 0); week 26
Height velocity (HV) was derived from height measurements taken at baseline (week 0) and week 26 visit as: HV = (height at 26 weeks visit - height at baseline)/(time from baseline to 26 weeks visit in years). The outcome measure was evaluated based on the data from in-trial observation period. In-trial observation period: from first administration and up until week 26 or last trial contact, whichever came first.
次要结局
- Change in Ratio of Bone Age Versus Chronological Age(Baseline (week 0); week 52)
- Change in Height Standard Deviation Score (HSDS)(Baseline (week 0); week 26)
- Change in Height Velocity Standard Deviation Score (HVSDS)(Baseline (week 0); week 26)
- Change in Fasting Plasma Glucose(Baseline (week 0); week 26)
- Change in Homeostatic Model Assessment for Steady State Beta Cell Function (HOMA-B)(Baseline (week 0); week 26)
- Change in Homeostatic Model Assessment for Insulin Resistance (HOMA-IR)(Baseline (week 0); week 26)
- Change in Glycated Haemoglobin (HbA1c)(Baseline (week 0); week 26)
- Change in Insulin-like Growth Factor I (IGF-I) Standard Deviation Score (SDS)(Baseline (week 0); week 26)
- Change in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) SDS(Baseline (week 0); week 26)