NCT04219007

Vosoritide for Selected Genetic Causes of Short Stature

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 56 (actual)
• Sponsor: Andrew Dauber · Academic / Other
• Sex: All
• Age: 3 Years – 10 Years
• Healthy volunteers: Not accepted

Summary

Short stature can be caused by a number of genetic etiologies, many of which directly affect the growth plate. The FGFR3/CNP pathway is central to growth of the chondrocyte. The study team hypothesizes that patients with selected genetic causes of short stature that interact with this pathway will benefit from treatment with vosoritide, a CNP analog, a selective NPR-B agonist which directly targets the growth plate. This study will enroll patients with short stature in selected genetic categories and will follow them for a 6 month observation period to obtain a baseline growth velocity, safety profile and quality of life assessment. Patients will then be treated with vosoritide for 12 months and will be assessed for safety monitoring and improvement in height outcomes.

Conditions

• Short Stature

Interventions

Type	Name	Description
DRUG	Vosoritide	After enrollment, subjects will be followed for a 6 month observation only period to establish a baseline height velocity as well as safety profile and quality of life assessment. Vosoritide will then be administered daily via subcutaneous injection at a dose of 15 µg/kg/day for 12 months.

Timeline

• Start date: 2020-08-04
• Primary completion: 2025-08-14
• Completion: 2035-06-01
• First posted: 2020-01-06
• Last updated: 2026-03-18

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study