A Study of the Genetic Basis of Response to Growth Hormone Treatment in Children With Idiopathic … (NCT05894876) | Clinical Trial Compass
TerminatedNot Applicable
A Study of the Genetic Basis of Response to Growth Hormone Treatment in Children With Idiopathic Short Stature
Stopped: Due to recruitment challenges, the sponsor has decided to terminate the study prematurely.
United States, Brazil, Israel43 participantsStarted 2024-04-29
Plain-language 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. 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'.
Who can participate
Age range
3 Years – 11 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:
* 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 t…
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.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
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.
What they're measuring
1
The frequency of genetic variants that significantly differ between the two responder groups