SAT-187 Efficacy And Safety Of Somapacitan In Non-GHD Adolescents With Short Stature: 26-week Results From The REAL9 Phase 3 Study
Bibliographic record
Abstract
Abstract Disclosure: A.K. Maniatis: Ascendis, Novo Nordisk, OPKO, Pfizer, Inc. M. Højby: Novo Nordisk. M.Y. Jalaludin: Novo Nordisk. A.A. Jorge: Novo Nordisk, Merck, Pfizer, Inc., Biomarin. J. Mori: Novo Nordisk, Pfizer, Inc. K. Soltysik: Novo Nordisk. R. Stawerska: Ascendis, OPKO, Novo Nordisk, Pfizer, Inc., Sandoz. A. Linglart: Novo Nordisk, Pfizer, Inc.. Short stature in children can be caused by a variety of factors. In the case of short children born small for gestational age (SGA), girls with Turner syndrome (TS), children with Noonan syndrome (NS) or idiopathic short stature (ISS), growth hormone (GH) secretion assessed by stimulation tests is usually normal, although GH sensitivity may be decreased and IGF-I impaired. Daily GH injections can improve longitudinal growth for such children, but treatment can be burdensome for patients/caregivers. Somapacitan is a long-acting reversible albumin-binding GH derivative in development for once-weekly subcutaneous (s.c.) administration in these indications. REAL9 (NCT05723835) is an interventional, multi-national, open-labelled, and uncontrolled phase 3 study, comprising a 26-week main phase followed by a first 130-week extension phase and a second 91-week extension phase to evaluate long-term safety and efficacy of somapacitan administered s.c. once-weekly (0.24 mg/kg/week) in older children and adolescents (aged >10 or >11 years for girls and boys, respectively) born SGA, or with TS, NS, or ISS. This study supports the pivotal phase 3 REAL8 study (NCT05330325) in treatment-naïve prepubertal children born SGA, or with TS, NS, or ISS. In total, the study enrolled 12 participants born SGA (4 GH-treatment-naïve), 11 girls with TS (4 GH-treatment-naïve), 13 participants with NS (6 GH-treatment-naïve), and 11 participants with ISS (2 GH-treatment-naïve). All participants completed 26-weeks of treatment with somapacitan. Across all indications, growth-related endpoints (height velocity, change in height standard deviation score (SDS), and change in height velocity SDS) showed an expected increase in treatment-naïve participants as well as maintenance in participants switching from daily GH to somapacitan. PK/PD model-derived weekly IGF-I average SDS values were in the same range as that determined for younger children in the REAL8 study. Somapacitan was well tolerated and had a safety profile similar to the well-known safety profile of daily GH. Adverse events (AEs) were either mild or moderate in severity across all indications, withno dose reductions required. One AE of injection site bruising was reported for one participant with NS as well as one participant with TS. No participants reported injection site pain with somapacitan. In conclusion, 26-weeks of treatment with somapacitan showed a safety and tolerability profile in older children and adolescents across all four indications (SGA, TS, NS, and ISS) similar to younger children. In addition, somapacitan effectively improved longitudinal growth in treatment-naïve participants and maintained growth in participants previously treated with daily GH. Presentation: Saturday, July 12, 2025
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.001 | 0.001 |
| Meta-epidemiology (narrow) | 0.001 | 0.000 |
| Meta-epidemiology (broad) | 0.002 | 0.001 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.001 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.001 | 0.000 |
| Research integrity | 0.001 | 0.002 |
| Insufficient payload (model declined to judge) | 0.006 | 0.001 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".