Epidemiology Study on Insulin-like Growth Factor-1 in Children With Idiopathic Short Stature (EPIGROW Study)

This study has been completed.
Sponsor:
Information provided by:
Ipsen
ClinicalTrials.gov Identifier:
NCT00710307
First received: July 2, 2008
Last updated: September 9, 2010
Last verified: September 2010
  Purpose

The purpose of the protocol is to describe the distribution of IGF-1 deficiency in the studied population of Idiopathic Short Children without Growth Hormone Deficiency or any other identified cause of short stature and not treated with recombinant Growth Hormone or IGF-1


Condition Intervention Phase
Idiopathic Short Stature
Procedure: Blood sampling
Genetic: Genetic analysis
Phase 4

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Cross-Sectional
Official Title: Descriptive, Cross-sectional and Prospective Epidemiology Study, on the Identification of Insulin-like Growth Factor-1 Status in Idiopathic Short Stature Children (EPIGROW Study)

Resource links provided by NLM:


Further study details as provided by Ipsen:

Primary Outcome Measures:
  • Proportion of patients with a mean of the two basal IGF-1 measurements ≤-2.0 SDS, > -2.0 SDS and below 0 SDS, ≥ 0.0 SDS [ Time Frame: Day 1 for the first sample; between Day 14 and Day 45 for the second sample ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Proportion of patients with height ≤ -3.0 SDS,and height > -3.0 SDS and ≤ -2.5 SDS [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
  • Description of mean basal IGF-1 and IGFBP-3 levels, and basal ALS and prolactin levels in patients with height ≤ -3.0 SDS, and height > -3.0 SDS and ≤ -2.5 SDS [ Time Frame: Day 1 and Day 14-45 ] [ Designated as safety issue: No ]
  • Proportion of patients having presented at least one historical documented clinically significant episode of hypoglycaemia [ Time Frame: Before the start of the study and during the study. ] [ Designated as safety issue: No ]
  • Identification of candidate genes and/or DNA aberrations or changes potentially associated with short stature. DNA regions identified during the genome-wide scan will be further mapped at higher resolution (DNA-sequencing) [ Time Frame: Day 1 ] [ Designated as safety issue: No ]

Biospecimen Retention:   Samples With DNA

Whole blood will be collected. The blood sample will be kept as long as necessary and for a maximum of 5 years.


Enrollment: 275
Study Start Date: October 2008
Study Completion Date: January 2010
Primary Completion Date: January 2010 (Final data collection date for primary outcome measure)
Intervention Details:
    Procedure: Blood sampling
    Blood samples will be collected at visit 1 (day 1) and at visit 2 (day 14 to 45).
    Genetic: Genetic analysis
    The sample for genetic analyses may be taken at Visit 1. The blood sample volume will be 6 mL.
  Eligibility

Ages Eligible for Study:   2 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Idiopathic Short Stature children

Criteria

Inclusion Criteria:

  • Short children, height ≤ -2.5 SDS
  • Age ≥ 2 years
  • With at least one normal or elevated peak GH response to a stimulatory test (peak GH ≥ 7 ng/mL) at the time of the study and/or at a given time-point during the last 12 months
  • Pre-pubertal
  • Signed Informed Consent, including agreement to have blood samples taken for hormonal measurement and genetic analysis, by both parents or by Legally Acceptable Representatives when applicable and the child when applicable

Exclusion Criteria:

  • The following identified causes of short stature:
  • GH-deficient short stature
  • Other endocrine causes (hypothyroidism, Cushing's syndrome, parathyroid or vitamin D disorders, hypogonadism)
  • Identified syndromes with genetic abnormalities (including Turner, Noonan and Russell-Silver syndromes)
  • Chronic diseases including malnutrition, coeliac disease, chronic inflammation, muscular dystrophy, thalassaemia, blood disorders, severe liver or kidney disease and severe cyanotic heart disease
  • Chronic diseases requiring treatment with chronically administered corticosteroids
  • Skeletal dysplasia
  • Psychosocial short stature
  • Patients having received irradiation, including total body irradiation
  • Patients currently on GH or IGF-1 therapy or having received GH or IGF-1 therapy in the last 12 months
  • Patients likely to require GH, IGF-1 or chronic corticosteroid treatment during the study
  • Any mental condition that prevents both parents or Legally Acceptable Representatives and the child when applicable from understanding the nature, scope and possible consequences of the study, or any evidence of an uncooperative attitude
  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: NCT00710307

Locations
France
Ipsen Central Contact
Paris, France
Sponsors and Collaborators
Ipsen
Investigators
Study Director: Pacale Dutailly Ipsen
  More Information

No publications provided

Responsible Party: Pascale Dutailly, Ipsen
ClinicalTrials.gov Identifier: NCT00710307     History of Changes
Other Study ID Numbers: 2-79-52800-001
Study First Received: July 2, 2008
Last Updated: September 9, 2010
Health Authority: Belgium: Federal Agency for Medicinal Products and Health Products

Additional relevant MeSH terms:
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Genetic Diseases, Inborn
Endocrine System Diseases
Mitogens
Mitosis Modulators
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on September 18, 2014