NCT07126262

A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Vosoritide in Infants and Young Children With Hypochondroplasia, Aged 0 to < 36 Months

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 60 (estimated)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 0 Months – 36 Months
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the safety and efficacy of daily administration of vosoritide in participants with HCH aged 0 to \< 36 months over a 52-week period.

Detailed description

Study 111-212 is a Phase 2, randomized, double-blind, placebo-controlled, multicenter study to assess the safety and efficacy of vosoritide versus placebo in infants and young children with HCH. Eligible participants with documented HCH confirmed by genetic testing will be randomized in a 1:1 ratio to receive vosoritide or placebo. Participants will receive study treatment daily for 52 weeks by subcutaneous (SC) injection, followed by a 2-week safety follow-up visit. Vosoritide dosing will follow a weight-band regimen.

Conditions

• Hypochondroplasia

Interventions

Type	Name	Description
DRUG	Vosoritide	The vosoritide dose administered will be based on the participant's weight and will follow the weight-band dosing regimen approved for ACH
DRUG	Placebo	Subcutaneous injection of recommended dose of placebo

Timeline

• Start date: 2025-07-30
• Primary completion: 2028-06-30
• Completion: 2028-06-30
• First posted: 2025-08-17
• Last updated: 2026-03-31

Locations

26 sites across 7 countries: United States, Australia, France, Germany, Italy, Japan, United Kingdom

Regulatory

• FDA-regulated drug study