Biochemical Markers of Growth Response to Growth Hormone Treatment in Children With Idiopathic Short Stature
Condition: Idiopathic Short Stature
MeSH terms: Dwarfism
Study type: Interventional
Phase: Phase 4

Summary: 
      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: 
      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:

        1. To determine axiological and biochemical markers for growth response

        2. To assess the period of time necessary to determine the parameters which will
           differentiate between responders and non-responders

      Inclusion criteria:

        1. Ages 3 to <9 years

        2. Short stature with height >2.25 Standard Deviation below the mean

        3. Prepubertal (Tanner stage I) at commencement of trial

        4. Peak Growth Hormone above 10ng/ml in at least one provocative test for Growth Hormone
           secretion

        5. Signing Informed consent forms

      Exclusion criteria:

        1. Intra Uterine Growth Retardation

        2. Growth retardation associated with malignancy, severe chronic disease, genetic syndromes
           and endocrine disorders

        3. Diabetes

        4. Treatment with any medical product which may interfere with Growth Hormone effects

      Methods:

        1. 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.

        2. 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

        3. 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.
    

Intervention name(s): Somatotropin growth hormone recombinant human
Intervention type(s): Drug
Intervention description(s): daily Sub Cutaneous injections
Intervention MeSH terms: Hormones
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Growth Hormone treatment; ISS; Biochemical markers; Growth response