A Pivotal, Parallel-Arm, Phase 3, Open-Label, Active-controlled, Global, Multicenter, Randomized Basket Trial Investigating the Efficacy and Safety of Once-weekly Lonapegsomatropin Compared to Daily Somatropin in Prepubertal Children and Adolescents with Growth Failure or Short Stature due to Growth Hormone Sufficient Disorders ? Turner Syndrome, SHOX Deficiency, Small for Gestational Age, and Idiopathic Short Stature
ASND0047
What is the goal of the study?
This basket trial will enroll prepubertal children and adolescents with clinically diagnosed and genetically confirmed (if applicable) TS, SHOX-D, SGA, or ISS between ages of ?2 and <18 years with open growth plates. prior to randomization participants must be treatment nave never have received growth promoting therapies for short stature or growth failure. approximately 186 participants will be randomized with specific enrollment targets for each condition-specific cohort: 72 participants in ts shox-d 72 participants in iss and 42 participants in sga. within each of these three cohorts participants will be equally randomized 1:1 to receive either lonapegsomatropin or somatropin. randomization will be stratified by chronological age>< 5 years and ? 5 years) and sex. The primary efficacy endpoint of this trial is AHV at Week 52, which will be analyzed separately for each cohort, with efficacy established by meeting the non-inferiority margin defined for each cohort. Additional endpoints include additional measures of linear growth, PK and PD characterization of lonapegsomatropin, immunogenic response, safety assessments, and clinical outcome assessments.
Who can participate in the study?
Please contact the study team listed below to learn more.