A Study of the Genetic Determinants of Response to Growth Hormone Treatment in Children with Idiopathic Short Stature
A Study of the Genetic Determinants of Response to Growth Hormone Treatment in Children with Idiopathic Short Stature
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Clinical Trial Information
Trial Contact: Tilme, Linda
Trial Phone: 321.843.5278
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IRB No: 24.041.03
Protocol Abbrev: NN8640-4978
Principal Investigator: Joshua H Yang, MD
Age Group: Pediatric
Secondary Protocol No: NN8640-4978
ClinicalTrials.gov ID: NCT05894876
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Objective
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
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Key Eligibility
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 Centers 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.
