HighLiGHts - 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

Condition studied

Turner Syndrome (TS), idiopathic short stature (ISS), short stature homeobox containing gene deficiency (SHOX-D), small for gestational age (SGA)

Investigational medicinal product(s)

Eligibility

Primary endpoint

Annualized height velocity (AHV) at Week 52

Endpoint detail

AHV (Week 104), Change from baseline in height SDS (CDC-based) (Weeks 52 and 104), Change from baseline in height SDS (condition specific if applicable) (Weeks 52 and 104), Treatment-emergent adverse events (AEs), Safety labs, vital signs, physical exam, local tolerability assessments, and fundoscopy, Bone age (Weeks 52 and 104), Bone age:chronological age ratio (Weeks 52 and 104)