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

Idiopathic Short Stature Clinical Trial

— ISS  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00458263
• Other study ID #: rmc003515ctil
• Status: Completed
• Phase: Phase 4
• First received: April 8, 2007
• Last updated: January 1, 2013
• Start date: April 2006
• Est. completion date: May 2011

Study information

• Verified date: October 2011
• Source: Rabin Medical Center
• Contact: n/a
• Is FDA regulated: No
• Health authority: Israel: Ministry of Health
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 21
• Est. completion date: May 2011
• Est. primary completion date: May 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 3 Years to 9 Years

Eligibility

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

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Dwarfism
• Idiopathic Short Stature

Intervention

Drug:
• Somatotropin growth hormone recombinant human
daily Sub Cutaneous injections

Locations

Country	Name	City	State
Israel	schneider children medical center of Israel	Petach Tikva

Sponsors (2)

Lead Sponsor	Collaborator
Rabin Medical Center	Pfizer

Country where clinical trial is conducted

Israel, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Height		every 4 months	No
Primary	Growth velocity		every 4 months	No
Primary	Height at beginning of puberty		At the biginning of puberty	No
Primary	Final height		When acheiving final height	No
Secondary	Psychological parameters		once a year	No
Secondary	HbA1c and IGF-1		at baseline. after 3 months and than every 6 months	Yes