NCT06382155

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

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

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 100 (estimated)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 3 Years – 11 Years
• Healthy volunteers: Not accepted

Summary

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)(hGH; only in the United States), in children with idiopathic short stature (ISS).

Detailed description

Following a minimum 6 month observational period in which baseline growth is assessed, participants in the vosoritide and placebo groups will complete a minimum of 6 months of randomized treatment (maximum of 6 months of placebo treatment), followed by open-label treatment with vosoritide until they reach near-final adult height, or at least 16 years of age for females or 18 years of age for males, whichever comes later. Participants randomized to the hGH group will receive open-label hGH for a minimum of 4 years.

Conditions

• Idiopathic Short Stature

Interventions

Type	Name	Description
DRUG	Vosoritide Injection	Experimental Drug Lyophilized powder for reconstitution
DRUG	Human Growth Hormone	Commercial product containing somatotropin
DRUG	Placebo	Lyophilized powder for reconstitution

Timeline

• Start date: 2024-10-21
• Primary completion: 2026-06-01
• Completion: 2036-12-01
• First posted: 2024-04-24
• Last updated: 2026-03-13

Locations

47 sites across 6 countries: United States, Australia, France, Germany, Italy, South Korea

Regulatory

• FDA-regulated drug study