What is lonapegsomatropin studying for growth failure or short stature?

Lonapegsomatropin is being studied to see how well it works compared to daily somatropin in children and adolescents with growth hormone sufficient disorders causing short stature or growth failure. The research aims to evaluate its safety and effectiveness in helping improve growth. This trial is sponsored by Ascendis Pharma A/S and conducted worldwide.

Who may be eligible for this growth failure clinical trial?

Children and adolescents aged 2 to under 18 years diagnosed with Turner Syndrome, SHOX deficiency, small for gestational age, or idiopathic short stature may be eligible. Participants must be prepubertal, growth hormone treatment-naïve, and able to stand without assistance. Please review eligibility criteria for more details.

Is there any cost to participate in this growth failure clinical trial?

Study-related care, including the study drugs and all trial visits, is provided at no cost. However, personal travel or other expenses may vary and are not guaranteed to be covered. Participants should consider these factors when deciding to join the trial.

Actively Recruiting

Phase 3

Age: 2Years - 17Years

All Genders

ID07221851

A Phase 3 Randomized Trial Comparing Weekly Lonapegsomatropin to Daily Somatropin in Children and Adolescents With Growth Failure Due to Turner Syndrome, SHOX Deficiency, Small for Gestational Age, or Idiopathic Short Stature

Led by Ascendis Pharma A/S · Updated on 2026-05-29

186

Participants Needed

Research Sites

56 weeks

Total Duration

AI-Summary

What this Trial Is About

Researchers are investigating how well once-weekly lonapegsomatropin works compared to daily somatropin in prepubertal children and adolescents aged 2 to under 18 years who have clinically diagnosed and genetically confirmed conditions such as Turner Syndrome, SHOX deficiency, small for gestational age, or idiopathic short stature. The study focuses on children with open growth plates and growth failure related to these growth hormone sufficient disorders. This phase 3, randomized, open-label, global basket trial aims to evaluate the safety and effectiveness of these treatments for growth improvement.

CONDITIONS

Brief 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 prior treatment with growth hormone or growth hormone promoting therapies
Prepubertal status
Ability to stand without assistance
Diagnosis of Turner Syndrome, SHOX deficiency, small for gestational age, or idiopathic short stature with impaired growth or short stature confirmed by genetic tests and specific growth criteria
Stable and adequate hormone replacement therapy for other hormone deficiencies if applicable for at least 4 weeks before and during screening
Written informed consent from parent(s) or legal guardian(s) and assent from participant as required

You will not qualify if you...

Advanced bone age more than 20% above chronological age
Closed growth plates defined by bone age thresholds (≥14 years in females or ≥16 years in males)
Current diagnosis of diabetic retinopathy
Untreated moderate or severe sleep apnea
Prader Willi syndrome with severe obesity or respiratory impairment
Signs of intracranial hypertension or active proliferative retinopathy
Uncontrolled thyroid disease
Uncontrolled diabetes (HbA1c >7.5%)
History or diagnosis of gastrointestinal inflammatory conditions, HIV, radiation exposure, other skeletal dysplasias, growth hormone deficiency, or cardio-thoracic surgery affecting growth
Significant liver or kidney problems
Undiagnosed or uncontrolled high blood pressure
Treatment with agents affecting growth or growth hormone action, including sex steroids and ADHD stimulants
High dose inhaled glucocorticoids for more than 28 consecutive days within 12 months
Female pregnancy, planned pregnancy, or breastfeeding
Participation in another interventional clinical trial within 90 days prior or concurrent
Conditions that may impair compliance or increase risk with study treatments
For Turner Syndrome: presence of Y chromosome material without gonadectomy, less than 10% 45,X mosaicism, significant cardiovascular dysfunction
For Small for Gestational Age: chromosomal abnormalities or syndromes affecting growth, congenital skeletal abnormalities
For Idiopathic Short Stature: conditions causing disproportionate short stature or known growth disorders such as Turner syndrome or skeletal dysplasias

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - Up to 2 years (104 weeks) depending on assigned treatment arm

Participants receive weekly or daily subcutaneous injections of growth hormone treatments as assigned to improve growth.

Regular visits for treatment monitoring throughout the treatment period