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| ID | Type | Description | Link |
|---|---|---|---|
| 2025-A01871-48 | Other Identifier | ID RCB number |
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| Name | Class |
|---|---|
| URC-CIC Paris Descartes Necker Cochin | OTHER |
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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.
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.
The study concerns 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 or already being treated with growth hormone and wishing to change galenical. Auxological measurements and with the Zmetric impedance meter will be carried out at each visit of the child's usual follow-up, from the day of the start of growth hormone treatment, for 3 years.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
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| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Auxological measurements and impedancemetry | Other | Auxological measurements and with the Zmetric impedance meter will be carried out at each visit of the child's usual follow-up, from the day of the start of growth hormone treatment, for 3 years. |
| Measure | Description | Time Frame |
|---|---|---|
| Body mass index | 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. | At the start of treatment or new galenical, 3 months later, at 6 months, 12 months, 18, 24, 30 and 36 months |
| Measure | Description | Time Frame |
|---|---|---|
| Amount of muscle mass | Description of the body composition of children treated with GH for the indication of isolated growth hormone deficiency before the start of treatment or the change of the galenical and during 3 years. Amount of muscle mass in kg, in %, in kg/m2. | At the start of treatment or new galenical, 3 months later, at 6 months, 12 months, 18, 24, 30 and 36 months |
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Inclusion Criteria:
Exclusion Criteria:
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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 or already being treated with growth hormone and wishing to change galenical.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Michel MD, PhD POLAK, MD, PhD | Contact | 01 44 49 48 02 | +33 | michel.polak@aphp.fr |
| Hélène Morel | Contact | 01 71 19 63 46 | +33 | helene.morel@aphp.fr |
| Name | Affiliation | Role |
|---|---|---|
| Michel MD, PhD POLAK, MD, PhD | Assistance Publique - Hôpitaux de Paris | Principal Investigator |
| Dinane MD SAMARA-BOUSTANI, MD | Assistance Publique - Hôpitaux de Paris | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hôpital Necker-Enfants Malades | Recruiting | Paris | 75015 | France |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31780780 | Background | Kopchick JJ, Berryman DE, Puri V, Lee KY, Jorgensen JOL. The effects of growth hormone on adipose tissue: old observations, new mechanisms. Nat Rev Endocrinol. 2020 Mar;16(3):135-146. doi: 10.1038/s41574-019-0280-9. Epub 2019 Nov 28. | |
| 39261416 | Background | de Fries Jensen L, Antavalis V, Odgaard-Jensen J, Rossi A, Pietropoli A, Hojby M. Efficacy and Safety of Somapacitan Relative to Somatrogon and Lonapegsomatropin in Pediatric Growth Hormone Deficiency: Systematic Literature Review and Network Meta-analysis. Adv Ther. 2024 Nov;41(11):4098-4124. doi: 10.1007/s12325-024-02966-y. Epub 2024 Sep 11. |
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| Auxological measurements and impedancemetry | Other | Auxological measurements and with the Zmetric impedance meter will be carried out at each visit of the child's usual follow-up, from the day of the change of the galenical of the growth hormone treatment, for 3 years. |
|
| Amount of fat mass | Description of the body composition of children treated with GH for the indication of isolated growth hormone deficiency before the start of treatment or the change of the galenical and during 3 years. Amount of fat mass in kg, in %, in kg/m2. | 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 | Description of the body composition of children treated with GH for the indication of isolated growth hormone deficiency before the start of treatment or the change of the galenical and during 3 years. Bone mineral content in %. | 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 | Description of the body composition of children treated with GH for the indication of isolated growth hormone deficiency before the start of treatment or the change of the galenical and during 3 years. Amount of extracellular and intracellular water (in L). | 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 | Description of the body composition of children treated with GH for the indication of isolated growth hormone deficiency before the start of treatment or the change of the galenical and during 3 years. Hydration of fat-free mass (in %). | 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 | Description and evolution of health parameters from the patient's usual care during 3 years :
| At the start of treatment or new galenical, 3 months later, at 6 months, 12 months, 18, 24, 30 and 36 months |