Assessment of Body Composition in Children Treated With Growth Hormone for the Indication of Isolated Non-acquired Growth Hormone Deficiency.
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 200
- 试验地点
- 1
- 主要终点
- Body mass index
研究概览
简要总结
Since 1985, growth hormone deficiency (GHD) in children has been the first condition treated with daily injections of recombinant human growth hormone. Noncompliance with daily growth hormone (GH) therapy is common. Several long-acting growth hormone (LAGH) treatments have recently become available for prescription in France after pivotal phase III trials demonstrated the non-inferiority of these LAGH compared to daily GH administration. To date, published data on LAGH in children are largely limited to clinical trials of GH deficiency (GHD).
Contrary to what is observed with daily GH, body mass index increases during the first year of LAGH treatment. With the Somapacitan, the observed mean body mass index (BMI) (SDS) remained within the normal range, but with an increase from -0.17 to +0.39 in the LAGH group and a decrease from -0.25 to -0.49 in the daily GH group. In the Somatrogon study, BMI increased from -0.51 to -0.08 in the somatrogon group, while it decreased from -0.44 to -0.64 in the daily GH group. This increase in BMI was transient and then normalized over the 3-year follow-up.
In June 2025, recent data from the experience of private endocrinologists in France (AFPEL) on the real-life use of somatrogon were presented at the congress of the French Society of Pediatric Endocrinology and Diabetology. They reported a +1 SD increase in BMI during the first months of treatment in a cohort of 99 children, but an improvement was observed after prolonged treatment.
However, significant and persistent weight gain was observed in some patients, with a marked increase in abdominal adiposity. Some discontinued LAGH treatment in favor of daily GH.
Longer-term, real-life data are therefore needed to better understand the changes in BMI in these children treated with LAGH.
详细描述
Since 1985, growth hormone deficiency (GHD) in children has been the first condition treated with daily injections of recombinant human growth hormone. Noncompliance with daily growth hormone (GH) therapy is common. Several long-acting growth hormone (LAGH) treatments have recently become available for prescription in France after pivotal phase III trials demonstrated the non-inferiority of these LAGH compared to daily GH administration. To date, published data on LAGH in children are largely limited to clinical trials of GH deficiency (GHD).
Contrary to what is observed with daily GH, body mass index increases during the first year of LAGH treatment. With the Somapacitan, the observed mean body mass index (BMI) (SDS) remained within the normal range, but with an increase from -0.17 to +0.39 in the LAGH group and a decrease from -0.25 to -0.49 in the daily GH group. In the Somatrogon study, BMI increased from -0.51 to -0.08 in the somatrogon group, while it decreased from -0.44 to -0.64 in the daily GH group. This increase in BMI was transient and then normalized over the 3-year follow-up.
In June 2025, recent data from the experience of private endocrinologists in France (AFPEL) on the real-life use of somatrogon were presented at the congress of the French Society of Pediatric Endocrinology and Diabetology. They reported a +1 SD increase in BMI during the first months of treatment in a cohort of 99 children, but an improvement was observed after prolonged treatment.
However, significant and persistent weight gain was observed in some patients, with a marked increase in abdominal adiposity. Some discontinued LAGH treatment in favor of daily GH.
Longer-term, real-life data are therefore needed to better understand the changes in BMI in these children treated with LAGH.
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
- 3 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients aged 3 to 17 years inclusive.
- •Confirmed diagnosis of growth hormone deficiency.
- •Indication for treatment with daily or depot growth hormone.
- •Growth hormone treatment naive or already being treated with growth hormone and wishing to change galenical.
- •Holders of parental authority and children or adolescents informed and consenting to participate in the study.
排除标准
- •BMI greater than +3 SD.
- •Multiple pituitary insufficiency.
- •Acquired growth hormone insufficiency.
- •Patient with type 1 or type 2 diabetes.
- •Known eating disorders.
研究组 & 干预措施
Patients with isolated non-acquired growth hormone deficiency naive to growth hormone treatment
All children in the Department of Pediatric Endocrinology, Gynecology and Diabetology at Necker Enfants Malades University Hospital treated with growth hormone, with the daily form or the delayed form, for the indication of isolated non-acquired growth hormone deficiency, naive to growth hormone treatment.
干预措施: Auxological measurements and impedancemetry (Other)
Patients with isolated non-acquired growth hormone deficiency treated with growth hormone
All children in the Department of Pediatric Endocrinology, Gynecology and Diabetology at Necker Enfants Malades University Hospital treated with growth hormone, with the daily form or the delayed form, for the indication of isolated non-acquired growth hormone deficiency, already being treated with growth hormone and wishing to change galenical.
干预措施: Auxological measurements and impedancemetry (Other)
结局指标
主要结局
Body mass index
时间窗: At the start of treatment, 3 months later, at 6 months, 12 months, 18, 24, 30 and 36 months
Body mass index calculation at each visit of the child's usual follow-up, from the day of the start of growth hormone treatment during 3 years.
Body mass index
时间窗: At the start of treatment or new galenical, 3 months later, at 6 months, 12 months, 18, 24, 30 and 36 months
Body mass index calculation at each visit of the child's usual follow-up, from the day of the start of growth hormone treatment or the change of the galenical and during 3 years.
Body mass index
时间窗: At the start of treatment, 3 months later, at 6 months, 12 months, 18, 24, 30 and 36 months
Body mass index calculation at each visit of the child's usual follow-up, from the day of the start of growth hormone treatment during 3 years.
次要结局
- Amount of muscle mass(At the start of treatment, 3 months later, at 6 months, 12 months, 18, 24, 30 and 36 months)
- Amount of fat mass(At the start of treatment, 3 months later, at 6 months, 12 months, 18, 24, 30 and 36 months)
- Amount of extracellular and intracellular water(At the start of treatment, 3 months later, at 6 months, 12 months, 18, 24, 30 and 36 months)
- Hydration of fat-free mass(At the start of treatment, 3 months later, at 6 months, 12 months, 18, 24, 30 and 36 months)
- Amount of muscle mass(At the start of treatment or new galenical, 3 months later, at 6 months, 12 months, 18, 24, 30 and 36 months)
- Amount of fat mass(At the start of treatment or new galenical, 3 months later, at 6 months, 12 months, 18, 24, 30 and 36 months)
- Bone mineral content(At the start of treatment or new galenical, 3 months later, at 6 months, 12 months, 18, 24, 30 and 36 months)
- Amount of extracellular and intracellular water(At the start of treatment or new galenical, 3 months later, at 6 months, 12 months, 18, 24, 30 and 36 months)
- Hydration of fat-free mass(At the start of treatment or new galenical, 3 months later, at 6 months, 12 months, 18, 24, 30 and 36 months)
- Description of the efficacy and tolerance of growth hormone treatment(At the start of treatment or new galenical, 3 months later, at 6 months, 12 months, 18, 24, 30 and 36 months)
- Amount of muscle mass(At the start of treatment, 3 months later, at 6 months, 12 months, 18, 24, 30 and 36 months)
- Amount of fat mass(At the start of treatment, 3 months later, at 6 months, 12 months, 18, 24, 30 and 36 months)
- Bone mineral content(At the start of treatment, 3 months later, at 6 months, 12 months, 18, 24, 30 and 36 months)
- Amount of extracellular and intracellular water(At the start of treatment, 3 months later, at 6 months, 12 months, 18, 24, 30 and 36 months)
- Hydration of fat-free mass(At the start of treatment, 3 months later, at 6 months, 12 months, 18, 24, 30 and 36 months)
- Description of the efficacy and tolerance of growth hormone treatment(At the start of treatment, 3 months later, at 6 months, 12 months, 18, 24, 30 and 36 months)