Clinical trial NCT00458263: Biochemical Markers of Growth Response to Growth Hormone Treatment in Children With Idiopathic Short Stature
Official title: One Arm, Open Study to Assess Biochemical Markers of Growth Response to Growth Hormone Treatment in Children With Idiopathic Short Stature
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Idiopathic Short Stature
Keywords: Growth Hormone treatment
ISS
Biochemical markers
Growth response
Lead sponsor: Rabin Medical Center
Sponsor class: OTHER
Collaborators: name: Pfizer
First posted: 2007-04-10
Enrollment: 21
Sex: ALL
Minimum age (years): 3
Maximum age (years): 9
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Height
timeFrame: every 4 months
measure: Growth velocity
timeFrame: every 4 months
measure: Height at beginning of puberty
timeFrame: At the biginning of puberty
measure: Final height
timeFrame: When acheiving final height
Secondary outcomes:
measure: Psychological parameters
timeFrame: once a year
measure: HbA1c and IGF-1
timeFrame: at baseline. after 3 months and than every 6 months
Other outcomes:
Locations:
city: Petach Tikva
country: Israel
facility: schneider children medical center of Israel
Study officials:
affiliation: Schneider Children Medical Center
name: Moshe Phillip, Prof, MD
role: PRINCIPAL_INVESTIGATOR
References: