Observational Study to Understand Growth Patterns in Children with Idiopathic Short Stature Aged 2 to 16 Years

What this Trial Is About

Researchers are conducting an observational study to collect baseline growth data in children diagnosed with Idiopathic Short Stature ISS. The study aims to better understand growth patterns by measuring various growth-related factors over time in this pediatric population aged between 2 and 16 years, with a focus on children experiencing significant short stature. This long-term study is sponsored by BioMarin Pharmaceutical to help characterize how children with ISS grow compared to typical growth standards. Participants will be observed without receiving any study treatments. Growth measurements such as annualized growth velocity, height Z-score, standing height, body mass index BMI, and BMI Z-score will be collected every six months. The study will also record medical events and explore relationships between genetic variants and growth velocity. This observational approach allows tracking changes over time without intervention. Families will be involved in regular assessments every six months for up to 15 years. These evaluations include physical growth measurements and monitoring of medical events related to short stature. Data from historic growth hormone hGH stimulation tests and treatment status will also be considered. The studys primary outcomes focus on changes in growth metrics, while secondary outcomes assess medical event rates and genetic associations. Participation requires ongoing cooperation for scheduled measurements and consent from parents or guardians.

A Study to Assess Growth in Children With Idiopathic Short Stature