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.

NCT06668805

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

Recruiting
PHASE2Ages 3–11InterventionalTreatment
BioMarin Pharmaceutical
~30 participants
Updated 2026-08-24 on ClinicalTrials.gov
What's tested:Vosoritide Injection

At a glance

Recruiting sites
33 of 41 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Change from baseline in Annualized Growth Velocity (AGV)
Measured over At 6 months
Noonan Syndrome
41 sites across 37 states
California2
New York2
Texas2
Paris2
Colorado1
Delaware1
District of Columbia1
Florida1
  • Medical Director, MD, PhD · STUDY_DIRECTOR · BioMarin Pharmaceutical

Opens a ready-to-send draft in your own email app — review before sending.

  • Change from baseline in Annualized Growth Velocity (AGV)At 6 months