A Study of the Genetic Basis of Response to Growth Hormone Treatment in Children With Idiopathic Short Stature

December 5, 2025 updated by: Novo Nordisk A/S

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'.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
43

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Brazil
      • São Pauloa, Brazil, 05403-908
        • University of Sao Paulo School of Medicine
  • Israel
      • Ashdod, Israel
        • Assuta Ashdod Division of Pediatric Endocrinology and Diabetes
  • United States
    • California
      • Orange, California, United States, 92868
        • [Legal] Children's Hospital of Orange County on behalf of CHOC Children's Hospital of Orange County
    • Maryland
      • Columbia, Maryland, United States, 21044
        • Novo Nordisk Investigational Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

N/A

Sampling Method

Non-Probability Sample

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.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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.
Other: No treatment is given
No treatment is provided to the participants as part of this study.
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.
Other: No treatment is given
No treatment is provided to the participants as part of this study.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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.
One year after growth hormone therapy

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
The enrichment of genetic variants per gene/pathway
Time Frame: One year after growth hormone therapy
Measured as count.
One year after growth hormone therapy

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Novo Nordisk A/S

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Clinical Transparency (dept. 2834), Novo Nordisk A/S

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
April 29, 2024

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
March 21, 2025

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
March 21, 2025

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
May 30, 2023

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 30, 2023

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 8, 2023

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
December 12, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 5, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
December 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • NN8640-4978
  • U1111-1285-4921 (Other Identifier: World Health Organization (WHO))

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

According to the Novo Nordisk disclosure commitment on novonordisk-trials.com

Drug and device information, study documents

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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