Offered by: Nemours Children's
Location: Delaware Valley
Growth Hormone Treatment for Children With Noonan and Turner Syndromes
What is the trial about?
A study comparing the effect and safety of once weekly dosing of somapacitan with daily Norditropin® as well as evaluating long-term safety of somapacitan in a basket study design in children with short stature either born small for gestational age or with Turner syndrome, Noonan syndrome or idiopathic short stature
We are doing this study to compare two medicines for the treatment of these children who need help to grow. Norditropin® is an FDA approved brand of growth hormone given once a day and is a medicine doctors can already prescribe to promote growth in children with significant short stature. Somapacitan is a long-acting preparation of growth hormone attached to a chemical that makes it last longer so that it can be given once a week, this is the new medicine being tested in this study. Somapacitan is investigational and as such has not yet been approved for these indications. We want to find out if Somapacitan given weekly can make your children grow at least as well as Norditropin given daily.
Who can participate?
Children with TS, or NS aged >2 years and 26 weeks to <10 years of age.
What is involved?
Your child’s participation in the study will last about 3 years. This includes:
- 1-2 weeks at the start − to assess whether your child can take part in the study.
- In the first year your child will be taking either Somapacitan or Norditropin®.
- Children taking Somapacitan for the first year, will continue to take Somapacitan for another 2 years.
- Children taking Norditropin® for the first year, will then switch to treatment with Somapacitan and will be treated with this medicine for the next 2 years.
- Approximately 30 days after your child’s last dose of study medicine — we will check on your child’s general health during a visit.