NCT06455059

Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vosoritide in Children With Hypochondroplasia

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 80 (estimated)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 3 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

The intent and design of this Phase 3 study is to assess vosoritide as a therapeutic option for the treatment of children with hypochondroplasia (HCH).

Detailed description

This is a Phase 3 randomized, stratified, placebo-controlled, double-blind multicenter study to evaluate the effect of 52 weeks of daily vosoritide administration on annualized growth velocity (AGV) in participants with HCH. Eligible participants with documented HCH confirmed by genetic testing will roll over from Study 111-902 and enter the 111-303 study. Participants will be randomly assigned to one of two treatment groups: Placebo or Vosoritide. The route of administration is subcutaneous injection, and the frequency of administration is daily.

Conditions

• Hypochondroplasia

Interventions

Type	Name	Description
DRUG	Vosoritide	Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily.
DRUG	Placebo	Subcutaneous injection of recommended dose of placebo

Timeline

• Start date: 2024-06-17
• Primary completion: 2026-08-01
• Completion: 2026-08-01
• First posted: 2024-06-12
• Last updated: 2026-01-20

Locations

23 sites across 9 countries: United States, Australia, Canada, France, Germany, Italy, Japan, Spain, United Kingdom

Regulatory

• FDA-regulated drug study