Idiopathic Short StatureNCT05894876
A Study of the Genetic Basis of Response to Growth Hormone Treatment in Children With Idiopathic Short Stature
An observational study of Idiopathic Short Stature, run by Novo Nordisk A/S at 4 sites in 3 countries.
Trial summary
The study doctor will collect information from participants with Idiopathic Short Stature, who were treated with growth hormone for at least a year when they were children, before they reached puberty. Read more about this study
- Who is it for?
- Ages 3 to 11 years
- Conditions the study is for
- Idiopathic Short Stature
- What is being tested?
- No treatment is given
- How many people
- 43 participants
- Where
- 4 sitesin 3 countries
- Study dates
- 29 Apr 2024to 21 Mar 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 11 years
- All sexes
- Child
Study population: 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.
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
4 sites in 3 countries.
4 sites in 3 countries
United States2 sites
Novo Nordisk Investigational Site
Columbia, Maryland, 21044
[Legal] Children's Hospital of Orange County on behalf of CHOC Children's Hospital of Orange County
Orange, California, 92868
Israel1 site
Assuta Ashdod Division of Pediatric Endocrinology and Diabetes
Ashdod
About this study
Official title: A Study of the Genetic Determinants of Response to Growth Hormone Treatment in Children With Idiopathic Short Stature
The study doctor will collect information from participants with Idiopathic Short Stature, who were treated with growth hormone for at least a year when they were children, before they reached puberty. The word "Idiopathic" refers to "unknown cause", and as such the study participants have/had short stature with no identifiable medical cause. The purpose of the study is to identify differences in the genetic characteristics of participants who responded well or poorly to growth hormone therapy. No medications or other treatments are provided to the participants by Novo Nordisk as part of this study. The study will last for up to 1 year. The participants will attend their usual doctor's appointments. If the participants are not usually visiting the clinic, they will need to do it only once as part of this study. If the participant agrees to take part in the study, they will be asked to read and sign the 'Agreement to take part form'.
- Conditions
- Idiopathic Short Stature
- Being tested
- No treatment is given
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No treatment is givenOther
No treatment is provided to the participants as part of this study.
| Study group | Role | What they receive |
|---|---|---|
| Good responders | Participants on previous treatment with growth hormone will have one study visit for taking a non-invasive biological sample. Good responders are defined as participants with a change in height Standard Deviation Score (SDS) more than (>) 1.0, corresponding to >85th percentile. Receives: Other: No treatment is given | |
| Poor responders | Participants on previous treatment with growth hormone will have one study visit for taking a non-invasive biological sample. Poor responders are defined as participants with a change in height SDS less than (<) 0.4, corresponding to <15th percentile. Receives: Other: No treatment is given |
What is being measured
The primary outcome is the main question the study is designed to answer.
The frequency of genetic variants that significantly differ between the two responder groups
Measured as odds ratio.
Measured: One year after growth hormone therapy
Secondary outcomes (1)
The enrichment of genetic variants per gene/pathway
Measured as count.
Measured: One year after growth hormone therapy
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2023
- Study start29 Apr 2024
- Primary completion21 Mar 2025
- Study completion21 Mar 2025
Why it stopped: Due to recruitment challenges, the sponsor has decided to terminate the study prematurely.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Idiopathic Short StatureStudies of the same condition, open and under way.
- More trials run by Novo Nordisk A/SThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 43 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Transparency (dept. 2834), Study director, Novo Nordisk A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05894876
- Sponsor's protocol ID
- NN8640-4978
- Other IDs
- U1111-1285-4921
- Status last verified
- Dec 2025
- First submitted
- 30 May 2023
- Last updated
- 12 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
Study at a glance
From the registry record, last updated by the sponsor on 12 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study of the Genetic Determinants of Response to Growth Hormone Treatment in Children With Idiopathic Short Stature
- ClinicalTrials.gov ID
- NCT05894876
- Acronym / sponsor ID
- NN8640-4978
- Conditions
- Idiopathic Short Stature
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Due to recruitment challenges, the sponsor has decided to terminate the study prematurely.
- Enrolment
- 43 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- 29 Apr 2024 (actual)
- Primary completion
- 21 Mar 2025 (actual)
- Study completion
- 21 Mar 2025 (actual)
- First posted
- 8 Jun 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study doctor will collect information from participants with Idiopathic Short Stature, who were treated with growth hormone for at least a year when they were children, before they reached puberty. The word "Idiopathic" refers to "unknown cause", and as such the study participants have/had short stature with no identifiable medical cause. The purpose of the study is to identify differences in the genetic characteristics of participants who responded well or poorly to growth hormone therapy. No medications or other treatments are provided to the participants by Novo Nordisk as part of this study. The study will last for up to 1 year. The participants will attend their usual doctor's appointments. If the participants are not usually visiting the clinic, they will need to do it only once as part of this study. If the participant agrees to take part in the study, they will be asked to read and sign the 'Agreement to take part form'.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Good responders | – | Other: No treatment is givenParticipants on previous treatment with growth hormone will have one study visit for taking a non-invasive biological sample. Good responders are defined as participants with a change in height Standard Deviation Score (SDS) more than (>) 1.0, corresponding to >85th percentile. |
| Poor responders | – | Other: No treatment is givenParticipants on previous treatment with growth hormone will have one study visit for taking a non-invasive biological sample. Poor responders are defined as participants with a change in height SDS less than (<) 0.4, corresponding to <15th percentile. |
Who may qualify
- Ages
- 3 to 11 years
- Sex
- All
- Age groups
- Child
- Study population
- 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.
Key inclusion criteria (12 listed)
- 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:
Key exclusion criteria (18 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No treatment is givenNo treatment is provided to the participants as part of this study. | Other | Good responders, Poor responders |
Primary outcome
The frequency of genetic variants that significantly differ between the two responder groups
Time frame: One year after growth hormone therapy
Measured as odds ratio.
1 secondary outcome
The enrichment of genetic variants per gene/pathway
Time frame: One year after growth hormone therapy
Results and safety
The study was stopped early (reason given: Due to recruitment challenges, the sponsor has decided to terminate the study prematurely.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Dec 2025.
- Individual participant dataSharing planned: Yes. According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Transparency (dept. 2834) study director
Novo Nordisk A/S
4 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Idiopathic Short Stature, run by Novo Nordisk A/S at 4 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 12 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States, Brazil, Israel. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: Due to recruitment challenges, the sponsor has decided to terminate the study prematurely.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The frequency of genetic variants that significantly differ between the two responder groups (time frame: One year after growth hormone therapy). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Dec 2025.