Multicenter Interventional Study: Somatrogon Impact on Outcomes in Naive Small for Gestational Age or Idiopathic Short Stature Pediatric Patients Compared With Daily Growth Hormone
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 254
- 试验地点
- 33
- 主要终点
- Annual Height Velocity
研究概览
简要总结
This study is a randomized, open-label, active controlled, parallel group study comparing the efficacy and safety of once weekly Somatrogon to daily Growth Hormone (Genotropin) in pre-pubertal children with short stature either born Small for Gestational Age (SGA) or with Idiopathic Short Stature (ISS). The planned study duration is 12 months with a screening period of up to 30 days. The study will consist of two groups: 140 children with SGA who are naïve to GH treatment will be randomized 1:1 to receive either Somatrogon or Genotropin for 12 months. A second group will include 114 children with ISS who are naïve to GH treatment who will be randomized 1:1 to receive either Somatrogon or Genotropin for 12 months.
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 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of SGA or ISS. SGA, defined as born with a birth weight and/or length <-2 SDS below the mean for gestational age. ISS, defined as height < -2 SDS for age and gender without evidence of GHD
- •Females aged ≥3 years and <9 years. Males aged ≥3 years and <11 years
- •Pre-pubertal- Tanner stage 1 for breasts and testes.
- •A bone age of not more than chronological age recorded in previous 8 weeks.
- •Current height < -2 SDS for age and gender.
- •Participants using hormonal replacement therapy(s) must be on an optimized and stable treatment regimen (hormone levels within normal ranges on screening) for at least three months prior to screening
- •Participants who are willing and able to comply with all scheduled visits, treatment plan, laboratory tests, and other study procedures.
排除标准
- •History of cancer, radiation therapy or chemotherapy.
- •History of GHD.
- •Children who are small due to malnutrition, defined as a Z score of weight for height and/or BMI below -2 for age, according to national standards.
- •History of HIV-positive, acquired immune deficiency syndrome (AIDS), hepatitis B, hepatitis C, or tuberculosis.
- •Microcephaly (Head Circumference < -2 SDS)
- •Any chronic disease or diagnosis, likely to affect growth, including but not limited to gastrointestinal disorder, celiac disease, untreated thyroid disease, diabetes mellitus and metabolic disorders.
- •Known or suspected skeletal dysplasias
- •Known or suspected chromosomal abnormalities
- •IGF-1 >2 SDS
- •Any disorder or condition which, in the opinion of the investigator, might jeopardize participant's safety or compliance with the protocol
- •Prior exposure to growth promoting therapy
- •Current use of any prohibited concomitant medication(s): Any rhGH or growth-promoting therapy, Any therapy that affects appetite or weight, Psychiatric medications associated with weight changes and/or diabetes, excluding medications used to treat ADHD, Any androgen or estrogen therapy including over the counter supplements, Systemic corticosteroids (inhaled or oral) exceeding the doses: Inhaled: > 400 μg/day of inhaled budesonide or equivalent. Oral: > 8 mg/m2/day of oral hydrocortisone or equivalent.
- •Previous administration with an investigational drug within 90 days.
- •Fasting blood glucose >126 mg/dL
- •Renal impairment
- •Hepatic dysfunction.
- •Known hypersensitivity to the components of the study intervention
研究组 & 干预措施
Somatrogon
Somatrogon will be administered subcutaneously (s.c) once weekly using a multi dose disposable prefilled pen for single patient use intended for SC self injection.
干预措施: Somatrogon (Drug)
Genotropin
Genotropin will be administrated subcutaneously (s.c) daily using Genotropin Pen growth hormone delivery devices or Genotropin two chamber cartridges (will be supplied in their primary commercial packaging)
干预措施: Genotropin (Drug)
结局指标
主要结局
Annual Height Velocity
时间窗: • Annualized HV after 12 months of treatment
Annual Height Velocity in cm. Annual Height Velocity at 12 months is based on the difference between the heights at 12 months and baseline.
次要结局
- height SDS(3, 6, 9 and 12 months)
- Bone maturation(at screening and after 12 months)
- Insulin-like Growth Factor-1 (IGF-1)(Screening and after 3, 6, 9 and 12 months)
- Insulin-like Growth Factor-1 (IGF-1) Standard Deviation Score (SDS)(Baseline and at 12 months)
- change in health-related quality of life(baseline and after 12 months)
- Height Velocity over time(HV over 3, 6 and 9 months)
- height SDS(3, 6, 9 and 12 months)
- Insulin-like Growth Factor-1 (IGF-1) Standard Deviation Score (SDS)(Baseline and at 12 months)
- change in health-related quality of life(baseline and after 12 months)
- Bone maturation(at screening and after 12 months)
- Insulin-like Growth Factor-1 (IGF-1)(Screening and after 3, 6, 9 and 12 months)