Vosoritide for Short Stature in Turner Syndrome

What Was Tested

This registered phase 2 trial tested Vosoritide.

Turner syndrome (TS) is characterized by a missing whole or part of the second sex chromosome in a phenotypic female, resulting in short stature due to haploinsufficiency of the short-stature homeobox-containing (SHOX) gene.

It had 1 arm (groups): Vosoritide treatment arm (experimental).

The registry classes the interventions as drug.

Who Took Part

The registry lists 20 planned participants were the target.

It was open to women only aged from 3 years up to 11 years.

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

Examples of who could join: Stated willingness to comply with all study procedures and availability for the duration of the study. Age \>3 years 0 days AND \<10 years 364 days. Pre-pubertal defined as Tanner Stage 1 breasts in females. Patient height \<-2 SDS. All height SDS values are calculated using the CDC growth charts/data tables. Subjects must either be naïve to growth hormone or have a poor response to growth hormone therapy defined as either:.

The full record lists 9 inclusion and 10 exclusion criteria; among the exclusions: growth plate fusion - defined as a bone age via the greulich and pyle method of 13 years. these patients have limited remaining growth potential; prior or concomitant treatment with any form of estrogen, gonadotropin-releasing hormone (gnrh) analog, aromatase inhibitor or oxandrolone; history of any type of malignancy.

How the Study Was Run

Design: single-group and open-label.

Everyone knew when Vosoritide was being given (open label), which is simpler to run but leaves more room for expectations to affect what is reported.

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

It started in 2024-04-12 with completion listed for 2026-09.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Incidence of treatment-emergent adverse events (time frame: 12 months). Change from baseline in annualized growth velocity (time frame: 12 months). Change from baseline in age-sex standardized height standard deviation score (time frame: 12 months).

It also tracked 3 secondary outcomes, for example Changes in seated height ratio, Changes in arm span minus standing height and Change in Bone Age.

What This Means in Plain Language

Put simply, this is a phase 2 trial of Vosoritide in 20 people.

Its size is small, so on its own it can only give an early look.

Its status is unknown; no results are posted to the registry yet, so nothing here describes outcomes.

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