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
Phase 3 – large-scale trial before approval
Conditions:
short stature homeobox containing gene deficiency (SHOX-D)
Turner Syndrome (TS)
idiopathic short stature (ISS)
small for gestational age (SGA)
Trial status: Authorised
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)
Annualized height velocity (AHV) at Week 52
Description
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)
Who can participate
Age group: 0-17 years
Gender: Female, Male
Target enrollment: 66 participants
Interventions
Norditropin FlexPro, injektionsvæske, opløsning i fyldt pen
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