In short
Clinical trials are studying Somapacitan in children who need help to grow. These studies look at how well it works, how safe it is, and how it compares with daily growth hormone treatment. The target groups include children with short stature, growth hormone deficiency, small for gestational age, Turner syndrome, Noonan syndrome, and idiopathic short stature.
Key points
- Clinical trials of Somapacitan are studying children who need help to grow. The main groups include children with growth hormone deficiency, small for gestational age, Turner syndrome, Noonan syndrome, and idiopathic short stature. Most of the studies are Phase 3 trials, and one is Phase 2. The trials compare once-weekly Somapacitan with once-daily Norditropin® in several groups. The main outcome is usually height velocity, which shows how fast children grow over time, and one study also tracks adverse events.
Clinical trials overview
The available studies are interventional trials, which means researchers give a treatment and then measure the results. All listed trials are investigating Somapacitan in children with growth-related conditions, and the studies are either Phase 2 or Phase 3.
Three of the studies are authorised, and one is completed. The largest study listed has 412 participants, while the smallest has 44 participants.
Who the trials are for
The trials focus on children who need help to grow. The child groups include growth hormone deficiency, small for gestational age (born smaller than expected for pregnancy age), Turner syndrome, Noonan syndrome, and idiopathic short stature (short height with no clear cause).
Some studies include several conditions in one trial, while another study focuses only on children with SGA who did not show catch-up growth by 2 years of age or older. One study also includes children who were either new to growth hormone treatment or had already received it before.
What the studies are testing
Most trials compare once-weekly Somapacitan with once-daily Norditropin®, which is the standard treatment used in these studies. The goal is to see whether the weekly treatment works as well as the daily treatment for growth.
One Phase 3 study, REAL 9, looks mainly at safety in children with the four listed growth conditions. Safety is measured by counting adverse events, which are unwanted health problems that happen during the study.
The Phase 2 study in children born SGA also compares Somapacitan with Norditropin® and checks whether the weekly treatment improves growth over 26 weeks. The Phase 3 studies in children with short stature or growth hormone deficiency use a similar comparison but follow children longer, up to 52 weeks in two of the studies.
Main outcomes measured
The main outcome in most trials is height velocity, which means how fast a child grows in height over time. This outcome is usually measured in cm per year and helps show whether the treatment supports normal growth.
In the REAL 9 study, the main outcome is the number of adverse events from week 0 to week 26. In the other studies, the main focus is growth over time, with one study measuring from baseline to week 26 and two studies measuring from baseline to week 52.
Trial-by-trial summary
Here is a simple view of each study and what it is trying to answer.
- 2023-506927-27-00: A Phase 3 study in 412 children with SGA, Turner syndrome, Noonan syndrome, or idiopathic short stature. It compares once-weekly Somapacitan with once-daily Norditropin® and checks height velocity at week 52.
- 2022-501055-87-01: A Phase 3 safety study in 44 children with the same four conditions. It looks at adverse events over 26 weeks and includes children who are new to growth hormone treatment or not new to it.
- 2023-506830-66-00: A Phase 2 study in 62 children born SGA who did not show catch-up growth. It compares Somapacitan with Norditropin® and measures height velocity from week 0 to week 26.
- 2023-506829-11-00: A completed Phase 3 study in 200 children with growth hormone deficiency. It compares Somapacitan with Norditropin® and measures height velocity from week 0 to week 52.
Key points for patients
These trials are not testing Somapacitan in adults or in many other diseases; they are focused on children with growth problems. The main question is whether a once-weekly treatment can help children grow as well as the daily treatment used as a comparison.
Because the studies measure growth over time, they help researchers understand both the benefit and the safety of treatment in real patient groups. The results are based on the listed study phases, participant numbers, and outcomes shown in the trial data.