A Non-interventional, Observational, Registry-based Study to Investigate Long-term Safety and Clinical Parameters of Somapacitan Treatment in Paediatric Patients With Growth Hormone Deficiency During Routine Clinical Practice
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 入组人数
- 500
- 试验地点
- 1
- 主要终点
- Number of adverse drug reactions
研究概览
简要总结
The aim of this study is to look at the long-term safety and clinical parameters of somapacitan in paediatric participants with growth hormone deficiency under routine clinical practice conditions. The study population will include 400 paediatric growth hormone deficient participants from the Global Registry for Novel Therapies in Rare Bone and Endocrine Conditions (GloBE-Reg) treated with once-weekly somapacitan and fulfilling the eligibility criteria of the study. The total duration of the study is planned to 10 years consisting of a 5-year recruitment period in the GLoBE-Reg followed by a 5-year follow-up period.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
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
- •Treated with commercially available somapacitan according to local practice at the discretion of the physician.
- •Primary confirmed diagnosis of growth hormone deficiency as per local practice.
- •Male or female below 18 years of age at the time of signing informed consent in the GLoBE-Reg.
排除标准
- •Participants with active malignancy or in treatment for active pre-existing malignancy.
研究组 & 干预措施
Participants with Growth Hormone Deficieny
The study is non-interventional as there are no interventions involved and decision to treat participants with commerically available somapacitan will be made at the treating physician's discretion prior to, and independently from, the decision to include the participants in the GLoBE-Reg registry. Novo Nordisk will not provide any products for included participants during the conduct of the study.
干预措施: Somapacitan (Drug)
结局指标
主要结局
Number of adverse drug reactions
时间窗: From baseline (week 0) to end of study (up to 10 years)
Measured as count of events.
次要结局
- Number of medication errors (incorrect dose administration)(From baseline (week 0) to end of study (up to 10 years))
- Number of participants with incident neoplasm(From baseline (week 0) to end of study (up to 10 years))
- Number of participants with incident diabetes mellitus type 2(From baseline (week 0) to end of study (up to 10 years))
- Height velocity(From baseline (week 0) to end of study (up to 10 years))
- Change in height velocity standard deviation score (HVSDS)(From baseline (week 0) to end of study (up to 10 years))
- Change in height standard deviation score (HSDS)(From baseline (week 0) to end of study (up to 10 years))
- Change in insulin-like growth factor I (IGF-I) standard deviation score (SDS)(From baseline (week 0) to end of study (up to 10 years))
- Change in bone age (measured as years)(From baseline (week 0) to end of study (up to 10 years))
- Change in bone age (measured as months)(From baseline (week 0) to end of study (up to 10 years))
- Participants reaching near adult height(at 10 year)
- Change in height SDS in participants reaching near adult height(From baseline (week 0) to end of study (up to 10 years))