Biochemical Markers of Growth Response to Growth Hormone Treatment in Children With Idiopathic Short Stature

Study Overview
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Brief Summary
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One arm, open, prospective, intervention study to assess biochemical markers of growth response to Growth Hormone treatment in 20 Children, aged 3-9 years old, with idiopathic short stature. All participants will be treated with Growth Hormone during the first year of the study (and then in accordance with the local ethic requirement, to supply drug which is not approved for the indication used in the study, for additional 3 years) and then will be followed up for the next 3 years. The impact of Growth Hormone therapy on clinical laboratory parameters that are indicative of the growth response will be assessed by collecting blood and urine samples during the 4 years study period. The primary endpoints are measurements of height and growth velocity during the year of Growth Hormone treatment, the height at the beginning of puberty and final height. Secondary endpoints are psychological parameters, assessed by questionnaires.

Detailed Description
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One arm, open prospective intervention study to assess biochemical markers of growth response to Growth Hormone treatment in 20 children, aged 3-9 years old, with idiopathic short stature. Objectives: To determine axiological and biochemical markers for growth response To assess the period of time necessary to determine the parameters which will differentiate between responders and non-responders Inclusion criteria: Ages 3 to <9 years Short stature with height >2.25 Standard Deviation below the mean Prepubertal (Tanner stage I) at commencement of trial Peak Growth Hormone above 10ng/ml in at least one provocative test for Growth Hormone secretion Signing Informed consent forms Exclusion criteria: Intra Uterine Growth Retardation Growth retardation associated with malignancy, severe chronic disease, genetic syndromes and endocrine disorders Diabetes Treatment with any medical product which may interfere with Growth Hormone effects Methods: All participants will be treated with Growth Hormone during the first year of the study (and then in accordance with the local ethic requirement, to supply drug which is not approved for the indication used in the study, for additional 3 years) and then will be followed up for the next 3 years. The impact of Growth Hormone therapy on clinical laboratory parameters that are indicative of the growth response will be assessed by collecting blood and urine samples during the 4 years study period.Samples will be test for biochemical markers of bone formation and resorption The primary endpoints are measurements of height and growth velocity during the year of Growth Hormone treatment, the height at the beginning of puberty and final height. Secondary endpoints are psychological parameters, assessed by questionnaires.

Official Title
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One Arm, Open Study to Assess Biochemical Markers of Growth Response to Growth Hormone Treatment in Children With Idiopathic Short Stature

Conditions
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Idiopathic Short Stature

Intervention / Treatment
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* Drug: Somatotropin growth hormone recombinant human


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Ages 3 to <9 years Short stature with height >2.25 Standard Deviation below the mean Prepubertal (Tanner stage I) at commencement of trial Peak Growth Hormone above 10ng/ml in at least one provocative test for Growth Hormone secretion Signing informed consent forms Exclusion Criteria: Intra Uterine Growth Retardation Growth retardation associated with malignancy, severe chronic disease, genetic syndromes and endocrine disorders Diabetes Treatment with any medical product which may interfere with Growth Hormone

Ages Eligible for Study
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Minimum Age: 3 Years
Maximum Age: 9 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: single arm<br> | Drug: Somatotropin growth hormone recombinant human<br>* daily Sub Cutaneous injections<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Height |  | every 4 months | 
| Growth velocity |  | every 4 months | 
| Height at beginning of puberty |  | At the biginning of puberty | 
| Final height |  | When acheiving final height | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Psychological parameters |  | once a year | 
| HbA1c and IGF-1 |  | at baseline. after 3 months and than every 6 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Growth Hormone treatment, ISS, Biochemical markers, Growth response