A Multi-centre, Prospective, Open Label, Single-arm, Non-interventional Post-marketing Study to Investigate the Long-term Safety and Clinical Parameters of Sogroya® Treatment in Children With Short Stature Due to Growth Hormone Deficiency Where Epiphysial Discs Are Not Closed Under Normal Clinical Practice Conditions in Japan Special Use-results Surveillance on Long Term Use of Sogroya® in Children With Short Stature Due to Growth Hormone Deficiency (GHD) Where Epiphysial Discs Are Not Closed
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 入组人数
- 200
- 试验地点
- 84
- 主要终点
- Number of adverse reactions (AR)
研究概览
简要总结
The purpose of the study is to investigate the safety and effectiveness of Sogroya® in children with short stature due to growth hormone deficiency where epiphysial discs are not closed under real-world clinical practice in Japan. The study will last for about 1 year (at shortest) to 3 years (at longest) depending on when the participant takes part in the study. The participant will be asked to answer questionnaire(s) about how they feel about the growth hormone (GH) product treatment once during the study (at about 3 months after starting the Sogroya® treatment) and about 3 months after starting the Sogroya® treatment.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 0 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed consent obtained before any study-related activities (study-related activities are any procedure related to recording of data according to the protocol).
- •The decision to initiate treatment with commercially available Sogroya® has been made by the patient/Legally Acceptable Representative (LAR) and the treating physician before and independently from the decision to include the patient in this study. Both GH treatment naïve and non-naïve children are eligible.
- •Male or female age 0 to 18 years (exclusive) at the time of signing informed consent.
- •Diagnosis with short stature due to GHD where epiphysial discs are not closed according to local normal clinical practice.
排除标准
- •Previous participation in this study. Participation is defined as having given informed consent in this study.
- •Treatment with any investigational drug within 30 days prior to baseline (the starting date of Sogroya® treatment).
- •Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.
- •Contraindication described in approved product labelling in Japan.
- •Patients with hypersensitivity to the active substance or to any of the excipients
- •Patients with malignant tumour
- •Female patients who are either pregnant or likely to be pregnant
研究组 & 干预措施
Children with GHD
Participants will be treated with commercially available Sogroya® according to routine clinical practice at the discretion of the treating physician. Administration will be according to the approved product labelling. The decision to treat a participant with Sogroya® is made at the treating physician's discretion before and independently from the decision to include the patient in this study.
干预措施: Somapacitan (Drug)
结局指标
主要结局
Number of adverse reactions (AR)
时间窗: From baseline (week 0) to end of study (up to 156 weeks)
Measured as count of reactions.
次要结局
- Number of adverse events (AEs)(From baseline (week 0) to end of study (up to 156 weeks))
- Number of serious adverse reactions (SARs)(From baseline (week 0) to end of study (up to 156 weeks))
- Change in height velocity (HV)(Every 12 months from baseline (week 0) to end of study (up to 156 weeks))
- Change in height standard deviation score (HSDS)(Every 12 months from baseline (week 0) to end of study (up to 156 weeks))
- Number of serious adverse events (SAEs)(From baseline (week 0) to end of study (up to 156 weeks))
- Change in ratio of bone age/chronological age(Every 12 months from baseline (week 0) to end of study (up to 156 weeks))
- Growth hormone device assessment tool (G-DAT)(At 12 weeks)
- Growth hormone patient preference questionnaire (GH-PPQ)(At 12 weeks)
- Change in bone age(Every 12 months from baseline (week 0) to end of study (up to 156 weeks))
- Change in height velocity standard deviation score (HVSDS)(Every 12 months from baseline (week 0) to end of study (up to 156 weeks))
- Change in insulin-like growth factor-I standard deviation score (IGF-I SDS)(Every 12 months from baseline (week 0) to end of study (up to 156 weeks))