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

This study has been completed.
Sponsor:
Collaborator:
Pfizer
Information provided by (Responsible Party):
Rabin Medical Center
ClinicalTrials.gov Identifier:
NCT00458263
First received: April 8, 2007
Last updated: January 1, 2013
Last verified: October 2011
  Purpose

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.


Condition Intervention Phase
Idiopathic Short Stature
Drug: Somatotropin growth hormone recombinant human
Phase 4

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: One Arm, Open Study to Assess Biochemical Markers of Growth Response to Growth Hormone Treatment in Children With Idiopathic Short Stature

Resource links provided by NLM:


Further study details as provided by Rabin Medical Center:

Primary Outcome Measures:
  • Height [ Time Frame: every 4 months ] [ Designated as safety issue: No ]
  • Growth velocity [ Time Frame: every 4 months ] [ Designated as safety issue: No ]
  • Height at beginning of puberty [ Time Frame: At the biginning of puberty ] [ Designated as safety issue: No ]
  • Final height [ Time Frame: When acheiving final height ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Psychological parameters [ Time Frame: once a year ] [ Designated as safety issue: No ]
  • HbA1c and IGF-1 [ Time Frame: at baseline. after 3 months and than every 6 months ] [ Designated as safety issue: Yes ]

Enrollment: 21
Study Start Date: April 2006
Study Completion Date: May 2011
Primary Completion Date: May 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: single arm Drug: Somatotropin growth hormone recombinant human
daily Sub Cutaneous injections

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

Ages Eligible for Study:   3 Years to 9 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00458263

Locations
Israel
schneider children medical center of Israel
Petach Tikva, Israel, 49202
Sponsors and Collaborators
Rabin Medical Center
Pfizer
Investigators
Principal Investigator: Moshe Phillip, Prof, MD Schneider Children Medical Center
  More Information

No publications provided

Responsible Party: Rabin Medical Center
ClinicalTrials.gov Identifier: NCT00458263     History of Changes
Other Study ID Numbers: rmc003515ctil
Study First Received: April 8, 2007
Last Updated: January 1, 2013
Health Authority: Israel: Ministry of Health

Keywords provided by Rabin Medical Center:
Growth Hormone treatment
ISS
Biochemical markers
Growth response

Additional relevant MeSH terms:
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Genetic Diseases, Inborn
Endocrine System Diseases
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on July 31, 2014