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

Actively Recruiting

Phase 3

Age: 2Years - 17Years

All Genders

NCT07221851

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

Led by Ascendis Pharma A/S · Updated on 2026-04-21

186

Participants Needed

Research Sites

167 weeks

Total Duration

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

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

Who Can Participate

Age: 2Years - 17Years

All Genders

Eligibility Criteria

You may qualify if you...

Chronological age between 2 and under 18 years at screening start
No previous treatment with growth hormone or growth hormone promoting therapies
Prepubertal status
Ability to stand without help
Diagnosis of Turner Syndrome or SHOX deficiency confirmed by genetic test with impaired growth or short stature defined by specific height or height velocity criteria
Diagnosis of small for gestational age without catch-up growth defined by birth weight/length below -2.0 SDS and specific height or height velocity criteria
Diagnosis of idiopathic short stature with height below -2.25 SDS and normal growth hormone-insulin-like growth factor 1 axis
For females with idiopathic short stature, 46,XX chromosome confirmed by karyotype or microarray
Stable and adequate doses of hormone replacement therapy for any hormone deficiencies other than growth hormone for at least 4 weeks before and during screening
Written informed consent signed by parent(s) or legal guardian(s); assent by participant as required

You will not qualify if you...

Advanced bone age X-ray more than 20% above chronological age
Closed growth plates defined by bone age 14 years or older in females or 16 years or older in males
Current diagnosis of diabetic retinopathy
Untreated moderate or severe sleep apnea
Prader Willi syndrome with severe obesity, severe upper airway obstruction, or severe respiratory problems
Signs or symptoms of intracranial hypertension or active proliferative retinopathy
Uncontrolled hypo- or hyperthyroidism
Uncontrolled diabetes mellitus with HbA1c above 7.5%
History of gastrointestinal inflammatory conditions, HIV, radiation exposure, skeletal dysplasias, growth hormone deficiency, or cardiothoracic surgery
Significant liver or kidney problems including low kidney function
Undiagnosed or uncontrolled high blood pressure
Treatment with agents that affect growth or growth hormone action including sex steroids and ADHD stimulants
High dose inhaled glucocorticoids for more than 28 consecutive days in the past year
Female who is pregnant, plans pregnancy, or breastfeeding
Participation in another interventional trial with investigational compounds within 90 days or concurrently
Any condition making compliance unlikely or increasing risk
For Turner Syndrome: presence of Y chromosome material without gonadectomy, less than 10% 45,X mosaicism, or significant heart problems affecting growth
For small for gestational age: chromosomal abnormalities, significant gene mutations or syndromes affecting growth, skeletal dysplasias
For idiopathic short stature: conditions causing disproportionate short stature, chromosomal abnormalities, significant gene mutations or syndromes affecting growth

AI-Screening