Why do some kids respond to growth hormone? scientists look to genetics
NCT ID NCT05894876
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study looked at children with idiopathic short stature (short stature with no known cause) who had already received growth hormone therapy for at least a year. Researchers compared the genetic profiles of those who responded well to treatment versus those who did not. The goal was to find genetic differences that might explain why some children benefit more from growth hormone. No new treatments were given as part of this study.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- What this could lead to
- If successful, this study could help doctors predict which children with idiopathic short stature will benefit from growth hormone therapy.
- What could go wrong
- The study was terminated early with only 43 participants, so results may be limited and not conclusive. It is an observational study, not a treatment trial.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Participants
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43 people
The number who actually took part.
- Started
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Apr 2024
- Finished
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Mar 2025
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
Study population will consist of participants treated with GH for at least one year when they were prepubertal ISS children and had no prior exposure to growth promoting therapy prior to initiation of GH therapy, including but not limited to growth hormone, Insulin-like Growth Factor type 1 (IGF-1) and ghrelin analogues.
- Ages
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3 to 11 years
- Sex
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Anyone
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Signed consent/parental consent and patient assent for minor children obtained before any study-related activities (study-related activities are any procedure related to recording of data according to the protocol). * Patient has been diagnosed with Idiopathic Short Stature (ISS) and received at least 1 year of GH therapy. * Patient was prepubertal at initiation of and throughout first year of GH therapy, as determined by the treating physician and patient medical records. * Patient had no prior exposure to growth promoting therapy prior to initiation of GH therapy, including but not limited to growth hormone, IGF-I and ghrelin analogues. * Age at initiation of GH therapy: * Boys: Age above or equal to 3 years and below 11.0 years. * Girls: Age above or equal to 3 years and below 10.0 years. * Impaired height prior to initiation of GH therapy defined as at least 2 standard deviations below the mean height for chronological age and sex according to local growth reference charts. In the absence of local reference charts, the standards of Centres for Disease Control and Prevention should be used. * GH deficiency has been excluded via GH stimulation test (cut point of 7 nanograms per milliliter \[ng/ml\]) or other clinical and biochemical criteria according to local clinical practice. * Patient fits within one of the following response groups: * Change in Height Standard Deviation Score (SDS) after approximately the first year (+/- 2 months) of GH therapy greater than (\>) 1.0. * Change in Height SDS after approximately the first year (+/- 2 months) of GH therapy less than (\<) 0.4. Exclusion Criteria: * Previous participation in this study. Participation is defined as having given informed consent in this study. * Mental incapacity, unwillingness or language barriers precluding adequate understanding, cooperation or informed consent. * Receipt of any investigational medicinal product within 3 months before or during the first year of GH therapy that could influence response to GH therapy. * Concomitant illness within 3 months before or during the first year of GH therapy that could (positively or negatively) influence the first year of GH therapy. Exception: Attention Deficit Hyperactive Disorder and its treatment can be included but should be recorded. * Children with suspected or confirmed growth hormone deficiency according to local practice. * Concomitant use of medication including gonadotropin-releasing hormone (GnRH) analogues, aromatase inhibitors, sex steroids, glucocorticoids or any other medication that can influence response to GH therapy. Exception: Attention Deficit Hyperactive Disorder and its treatment can be included but should be recorded. * Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with standing height measurements, such as but not limited to: * Significant spinal abnormalities including but not limited to scoliosis, kyphosis and spina bifida variants. * Any other disorder that can cause short stature such as, but not limited to, psychiatric disorders, nutritional disorders, chronic systemic illness, chronic respiratory conditions (e.g. asthma), and chronic renal disease. * Turner Syndrome (including mosaicism). * Noonan Syndrome. * Born small for gestational age (defined as birth length below -2 SDS OR birth weight below -2 SDS OR both) (according to national standards). * Extreme prematurity, defined as gestational age less than 32 weeks. * Syndromic short stature defined by the presence of significant dysmorphic features and/OR major malformations, Laron syndrome, Prader-Willi syndrome, Russell-Silver syndrome. * Significant developmental delays, autism spectrum or intellectual deficit of any degree. * Skeletal dysplasia. * Magnetic resonance imaging (MRI) result confirming pituitary structural abnormalities. * Poor adherence to GH therapy or interruption of it for any time during the first year of therapy, as judged by the treating physician.
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Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Assuta Ashdod Division of Pediatric Endocrinology and Diabetes
Ashdod, Israel
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Novo Nordisk Investigational Site
Columbia, Maryland, 21044, United States
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University of Sao Paulo School of Medicine
São Pauloa, 05403-908, Brazil
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[Legal] Children's Hospital of Orange County on behalf of CHOC Children's Hospital of Orange County
Orange, California, 92868, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Growth hormone treatment under Long-Term watch in thousands of children
- Scientists hunt for hidden genes behind unexplained short stature
- New hope for short kids: experimental drug takes on growth hormone
- New growth hormone injection aims to help short children grow taller
- Weekly shot may help short kids grow – new study underway
- New study aims to unlock growth secrets in short children