Vosoritide for Selected Genetic Causes of Short Stature

What Was Tested

This registered phase 2 trial tested Vosoritide.

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.

It had 1 arm (groups): Genetic Short Stature (experimental).

The registry classes the interventions as drug.

Who Took Part

56 actual participants took part.

It was open to people of any sex aged from 3 years up to 10 years.

Healthy volunteers were not accepted; participants needed the condition being studied.

The full record lists 11 inclusion and 9 exclusion criteria; among the exclusions: prior treatment with a gnrh analog, aromatase inhibitor or oxandrolone; history of any type of malignancy; chronic medical condition known to affect growth including but not limited to:.

How the Study Was Run

The set-up was single-group and open-label.

Being open-label, the Vosoritide study did not mask who received what.

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2020-08-04 with completion listed for 2025-08-14.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Incidence of Treatment Emergent Adverse Events [Safety and Tolerability] (time frame: 12 months). Incidence of Symptomatic Hypotension events [Safety and Tolerability] (time frame: 12 months). Change from Baseline in annualized growth velocity (time frame: 12 months).

It also tracked 3 secondary outcomes, for example Changes in the seated height ratio as a measure of body proportions, Changes in the difference between arm span and height from baseline and Changes from Baseline in bone age/chronological age.

What This Means in Plain Language

Put simply, this is a phase 2 trial of Vosoritide in 56 people.

Its status is active not recruiting; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the Vosoritide compound page, all human trials.