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

Phase 4

Completed

• Conditions: Idiopathic Short Stature
• Interventions: Drug: Somatotropin growth hormone recombinant human

• Registration Number: NCT00458263
• Lead Sponsor: Rabin Medical Center

Brief 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.

Study Timeline

• Study Start: 2006-04
• Study First Submitted: 2007-04-08
• Study First Submitted QC: 2007-04-08
• Study First Posted: 2007-04-10
• Primary Completion: 2011-05
• Study Completion: 2011-05
• Status Verified: 2011-10
• Last Update Submitted: 2013-01-01
• Last Update Posted: 2013-01-03

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 21

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
single arm	Somatotropin growth hormone recombinant human	-

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
Psychological parameters	once a year
HbA1c and IGF-1	at baseline. after 3 months and than every 6 months

Trial Locations

Locations (1)

schneider children medical center of Israel; 🇮🇱 Petach Tikva, Israel