RecruitingPhase 3NCT07221851

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

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

Sponsor

Ascendis Pharma A/S

Enrollment

186 participants

Start Date

Dec 12, 2025

Study Type

INTERVENTIONAL

Conditions

Turner Syndrome Idiopathic Short Stature Short Stature Homeobox Gene Mutation Small for Gestational Age at Delivery

Summary

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. The purpose of the study is to see how well treatment with once-weekly lonapegsomatropin works compared to treatment with daily somatropin. Approximately 186 participants will be distributed equally (1:1), to receive either lonapegsomatropin for 2 years or somatropin for 1 year followed by lonapegsomatropin for 1 year. This trial will be conducted in the United States, France, Germany, Italy, Romania, Spain and South Korea.

Eligibility

Min Age: 2 YearsMax Age: 17 Years

Plain Language Summary

Simplified for easier understanding

This study is comparing a once-weekly growth hormone injection (lonapegsomatropin) to standard daily injections in children with short stature due to conditions like Turner syndrome, short-for-gestational-age (SGA), isolated short stature (ISS), or a genetic condition called SHOX deficiency. The goal is to reduce the burden of daily injections. **You may be eligible if...** - Your child is between 2 and 18 years old and prepubertal (hasn't started puberty) - They have never received growth hormone treatment before - They have one of these diagnoses confirmed by genetic testing: Turner syndrome, SHOX deficiency, born small for gestational age (SGA), or idiopathic short stature (ISS) - They are able to stand without assistance **You may NOT be eligible if...** - Your child has started puberty - Your child has previously received any growth hormone therapy - They have certain other medical conditions affecting growth or metabolism Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

COMBINATION_PRODUCTLonapegsomatropin [SKYTROFA®]

Subcutaneous injection once weekly

COMBINATION_PRODUCTSomatropin Pen Injector

Subcutaneous injection once daily