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

Launched by BIOMARIN PHARMACEUTICAL · Apr 19, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called vosoritide for children who are shorter than average for their age and sex, a condition known as idiopathic short stature (ISS). The goal is to see how effective vosoritide is when given in multiple doses and to compare its effects with a standard treatment called human growth hormone (hGH). The trial is currently looking for participants aged between 3 and 10 years old who meet specific height requirements, meaning they are significantly shorter than most children their age.

To be eligible for this study, children must have a height that is at least 2.25 standard deviations below what is typical for their age and sex, based on official growth charts. They also need to be in Tanner Stage 1, which is a way of assessing physical development related to puberty. However, children with known genetic conditions that cause short stature or who have previously received growth treatments cannot participate. If your child joins the trial, they will receive regular doses of vosoritide and will be closely monitored by medical professionals throughout the study. This trial hopes to provide new insights into potential treatments for children facing growth challenges.

Gender

ALL

Eligibility criteria

• 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