Clinical Trial

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

Recruiting Phase 2
View on ClinicalTrials.gov →
Summary
The purpose of this study in children with Noonan syndrome is to evaluate the effect of 3 doses of vosoritide on growth as measured by AGV after 6 months of treatment. The long-term efficacy and safety of vosoritide at the therapeutic dose will be evaluated up to FAH.
Protocol Amendment History 2 changes
notable Enrollment reduced: 54 -> 30 participants 2026-05-12
notable Trial sites expanded: 24 -> 36 locations 2026-05-12
Trial Details
NCT Number NCT06668805
Lead Sponsor BioMarin Pharmaceutical
Conditions Noonan Syndrome
Enrollment 30 participants
Start Date 2024-11-22
Primary Completion 2027-03 (estimated)
Study Completion 2041-09 (estimated)
Updated on ClinicalTrials.gov 2026-05-11