Phase 3 Study Comparing Once-Weekly Lonapegsomatropin to Daily Somatropin in Children and Adolescents Aged 2 to 17 With Growth Failure or Short Stature Due to Turner Syndrome, SHOX Deficiency, SGA, or Idiopathic Short Stature

What this Trial Is About

Researchers are evaluating the effectiveness and safety of once-weekly lonapegsomatropin compared to daily somatropin in children and adolescents aged 2 to under 18 years with short stature or growth failure due to conditions such as Turner Syndrome, SHOX deficiency, small for gestational age, or idiopathic short stature. This phase 3 basket trial includes participants with confirmed diagnoses and open growth plates to study growth hormone sufficient disorders. Participants will be randomly assigned to one of two groups. One group will receive lonapegsomatropin injections once weekly for two years. The other group will receive daily somatropin injections for one year followed by lonapegsomatropin once weekly for the second year. Both treatments are given by subcutaneous injection, and the study compares the annual growth velocity and other growth-related measures over 52 and 104 weeks. During the trial, participants will be closely monitored with regular assessments including growth measurements, bone age evaluations, and safety checks for treatment-related side effects. The main outcome is the annualized height velocity after 52 weeks, with additional measurements at 104 weeks. The study includes genetic confirmation when relevant and requires ongoing monitoring for adverse events. Participation lasts up to two years with evaluations throughout to assess treatment impact and safety.

Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders