Vosoritide for Selected Genetic Causes of Short Stature
Status:
Recruiting
Trial end date:
2023-06-01
Target enrollment:
Participant gender:
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.