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

Clalit Health Services

Status and phase: Completed; Phase 4

Conditions: Idiopathic Short Stature

Treatments: Drug: Somatotropin growth hormone recombinant human

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT00458263

rmc003515ctil

Details and patient eligibility

About

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.

Full 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.

Enrollment: 21 patients

Sex: All

Ages: 3 to 9 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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21 participants in 1 patient group

single arm

Experimental group

Treatment:

Drug: Somatotropin growth hormone recombinant human