Overview

A Phase 2 Basket Study of Vosoritide in Children With Turner Syndrome, SHOX Deficiency and Noonan Syndrome With an Inadequate Response to Human Growth Hormone

Status:
RECRUITING

Trial end date:
2041-09-01

Target enrollment:

Participant gender:

Summary

The purpose of this basket study in children with Turner syndrome, SHOX deficiency, and Noonan syndrome is to evaluate the effect of 3 doses of vosoritide versus hGH 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.

Phase:

PHASE2

Details

Lead Sponsor:

BioMarin Pharmaceutical

Treatments:

Human Growth Hormone
vosoritide