Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatro… (NCT07221851) | Clinical Trial Compass
RecruitingPhase 3
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
United States, France, Germany186 participantsStarted 2025-12-12
Plain-language 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.
Who can participate
Age range
2 Years – 17 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Chronological age between ≥2 and \<18 years, at start of screening.
. Naïve to growth hormone and growth hormone promoting therapies.
. Prepubertal.
. Able to stand without assistance.
. Diagnosis of TS, SHOX-D, SGA, or ISS with impaired growth or short stature, according to the following disease-specific criteria:
. 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.
. Impaired growth or short stature defined as:
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1This trial compares a weekly injection of lonapegsomatropin to daily growth hormone shots — given how hard it can be to stick to daily injections, is the weekly schedule something my child might genuinely benefit from, and does my doctor think that convenience trade-off is worth discussing for our situation?
2Since this is a Phase 3 trial, there's already some earlier safety data on lonapegsomatropin — can my doctor walk me through what is currently known about its safety profile compared to the daily growth hormone my child might otherwise receive?
3The trial is specifically for conditions like Turner Syndrome, Short Stature Homeobox gene mutation, idiopathic short stature, and being born small for gestational age — can my doctor confirm which of these applies to my child and whether that diagnosis is well-established enough to consider a trial like this?
4The main thing this trial is measuring is how many centimeters per year a child grows — can my doctor explain what my child's current growth rate looks like and how that compares to what would be expected from standard daily growth hormone treatment?
5Before we consider this trial, is standard daily growth hormone therapy already an approved and available option for my child's specific condition, and would my doctor recommend trying that first rather than enrolling in a research study?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
. If on hormone replacement therapies for any hormone deficiencies other than growth hormone (e.g., adrenal, thyroid), must be on adequate and stable doses for ≥4 weeks prior to and throughout screening.
Exclusion criteria
. 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.
. Current clinical diagnosis of diabetic retinopathy
. Any diagnosis or presence at screening of the following:
. Untreated moderate or severe sleep apnea as determined by formal (local) read of an inpatient or at-home sleep study.
. Prader Willi syndrome with severe obesity, history of severe upper airway obstruction, or severe respiratory impairment.
. Signs/symptoms of intracranial hypertension, active proliferative retinopathy.