Phase 2 Study of Vosoritide Treatment for Growth in Children With Noonan Syndrome Who Have Inadequate Growth After or During Human Growth Hormone Therapy

What this Trial Is About

Researchers are studying children with Noonan syndrome who have inadequate growth during or after treatment with human growth hormone hGH. The study aims to evaluate the effects of three different doses of vosoritide on growth, specifically looking at annualized growth velocity AGV after six months of treatment. The long-term safety and effectiveness of vosoritide at the therapeutic dose will be assessed up to when participants reach their final adult height FAH. This Phase 2, randomized, multicenter trial compares three dosing regimens of vosoritide, which is given as an injection. Participants receive one of three different doses of this experimental drug, provided as a lyophilized powder for reconstitution. The study includes both a short-term evaluation of growth changes over six months and a longer-term assessment of height and safety measures up to 15 years. During the study, children will have regular height measurements and assessments of growth velocity, body proportions, bone density, and bone age. Researchers will also monitor heart conditions, bone-related events, and quality of life using various questionnaires and physical activity scores. Blood and urine tests will track vosoritide levels and biological markers. Safety and growth will be followed every six to twelve months until participants reach their final adult height, which may take up to 15 years.

A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment