A Phase 2 Study of Vosoritide in Children with Idiopathic Short Stature

Study Details

A Phase 2, Randomized, Controlled, Multicenter Study of Vosoritide in Children with Idiopathic Short Stature

Conditions:

Idiopathic Short Stature

Study ID

END24024 (BMN 111-210)

Study Description

The purpose of this study is to evaluate i) the effect of multiple doses of vosoritide and ii) the effect of the therapeutic dose of vosoritide compared to human growth hormone (hGH), in children with idiopathic short stature (ISS).

Eligibility Requirements

Key Inclusion Criteria

  1. Height assessment corresponding to a height Z-score of ≤ -2.25 SDs in reference to the general population of the same age and sex, as calculated using the Centers for Disease Control and Prevention (CDC) growth charts
  2. Tanner Stage 1, at time of signing the ICF (unless too young to stage).

Key Exclusions

  1. Known chromosomal imbalance or genetic variant causing short stature syndrome, including but not limited to Laron syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, Turner syndrome, disproportionate skeletal dysplasias, abnormal SHOX gene analysis, or Rasopathy (including Noonan syndrome)
  2. Previous treatment with a growth promoting agent

Principal Investigator

Adriana Carrillo Iregui, MD

Medical Director, Diabetes Program

More Information: Miami

Clinical Interests:
Type 1 and Type 2 diabetes mellitus, obesity, metabolic disorders, polycystic ovary syndrome (PCOS) and thyroid cancer.

More Information


This page was last updated on: 4/23/2026 1:29:25 PM