Vosoritide for Noonan Syndrome and Inadequate Growth
This study is looking at Vosoritide Injection in children with Noonan Syndrome who aren't growing enough, even with human growth hormone (hGH) treatment. The goal is to see how Vosoritide affects growth rate over 6 months. You might be able to join if you are between 3 and 11 years old (females) or 3 and 12 years old (males), have a confirmed diagnosis of Noonan Syndrome, and your height is below average. The study aims to understand the short-term effects of different doses of Vosoritide and its long-term impact on adult height. The current status of this study is unclear, and it plans to enroll 30 participants.
- Study design
- This is a Phase 2, randomized, multicenter study. It will compare the effects of three different dosing regimens of Vosoritide.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- The study will evaluate the long-term efficacy and safety of vosoritide up to final adult height (FAH).
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
At a glance
Conditions
Where it's being run
41 sites across 37 statesStudy leadership
- Medical Director, MD, PhD · STUDY_DIRECTOR · BioMarin Pharmaceutical
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
What this trial measures
- Change from baseline in Annualized Growth Velocity (AGV)At 6 months