A Phase 3 Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children with Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders
Key facts
- Phase
- Phase 3
This trial has no sites in United States.
It has sites in 5 countries. Choose a country to see its sites.
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record
Print a page for my doctorTrial details
- Who runs it
- Ascendis Pharma Endocrinology Division A/S
- Trial code
- EU 2025-523079-44-00
- Record updated
- 25 May 2026
- Original description
To evaluate the efficacy of lonapegsomatropin as compared to somatropin in children and adolescentsRead full original descriptionShow less
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
To evaluate the efficacy of lonapegsomatropin as compared to somatropin in children and adolescents
Inclusion: Chronological age between ≥2 and <18 years, at start of screening; Naïve to growth hormone and growth hormone promoting therapies; Prepubertal; Diagnosis of TS, SHOX-D, SGA, or ISS with impaired growth or short stature, according to the following disease-specific criteria: TS or SHOX-D (Léri-Weill dyschondrosteosis) a. Diagnosis confirmed by a genetic test. NOTE: Historical test results are acceptable for proof of diagnosis. For karyotypes, a minimum of 20 cells must be counted. b. Impaired growth or short stature defined as: (i.) AHV <25th percentile over a time span of 6-16 months prior to screening utilizing a historical height properly documented in a health care setting (self-measurement record is not accepted) OR (ii.) Height <5th percentile for sex and age according to the Centers for Disease Control Growth Charts for the United States SGA without catch-up growth c. Birth weight and/or birth length < -2.0 SDS for gestational age according to the 2006 World Health Organization Child Growth Standards. For infants born premature, the Fenton Preterm Infant Growth Chart (Fenton 2013) should be used. d. Impaired growth or short stature defined as: (i.) AHV <25th percentile over a time span of 6-16 months prior to screening properly documented in a health care setting (self-measurement record is not accepted) OR (ii.) Height < -2.0 SDS for age and sex according to the 2000 Centers for Disease Control Growth Charts for the United States for children ≥ 3 years or height < -2.5 SDS for age and sex according to the for children ≥ 2 years and < 3 years ISS e. Height < -2.25 SDS for sex and age according to the Centers for Disease Control Growth Charts for the United States with no identifiable cause for short stature. f. Documented normal GH-IGF-1 axis, defined as either: (i.) IGF-1 SDS >0 at screening based on central laboratory OR (ii.) Historical documentation of normal peak GH upon stimulation test (as defined by local institution) g. 46,XX chromosome as determined by karyotype or microarray if female. For karyotypes, a minimum of 30 cells must be counted.
Exclusion: Advanced bone age X-ray by central reading defined as >20% above chronological age in months (Greulich 1959); Closed epiphyses as defined as bone age of ≥14.0 years in females or ≥16.0 years in males.