Treatment of Short Stature in Girls With Turner Syndrome Using Daily Vosoritide Injections for 12 Months

What this Trial Is About

Researchers are investigating the use of vosoritide, a C-type natriuretic peptide analog, to treat short stature in pre-pubertal girls with Turner syndrome. Turner syndrome is a condition where part or all of the second sex chromosome is missing in females, causing short height due to a specific gene deficiency. This study focuses on girls who have never used growth hormone or have not responded well to it, aiming to see if vosoritide can increase their growth rate over 12 months. Participants will receive daily subcutaneous injections of vosoritide for one year, using dosing based on body weight following FDA-approved guidelines. The study includes an initial 12-month treatment phase where growth velocity and safety are monitored. If participants respond positively, they may continue with vosoritide in an extension phase until their growth stops. During the study, girls will be regularly assessed for safety and changes in height, including growth velocity and standardized height scores. Researchers will also measure other growth-related factors such as seated height ratio, arm span compared to standing height, and bone age. The study will compare growth over the treatment period to historical data from before starting vosoritide, with ongoing monitoring throughout participation.

Vosoritide for Short Stature in Turner Syndrome