Clinical trial NCT00710307: Epidemiology Study on Insulin-like Growth Factor-1 in Children With Idiopathic Short Stature (EPIGROW Study)
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)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Idiopathic Short Stature
Lead sponsor: Ipsen
Sponsor class: INDUSTRY
First posted: 2008-07-04
Enrollment: 275
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Idiopathic Short Stature children

Brief summary: 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


Eligibility 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

Primary outcomes:
measure: 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
timeFrame: Day 1 for the first sample; between Day 14 and Day 45 for the second sample
Secondary outcomes:
measure: Proportion of patients with height ≤ -3.0 SDS,and height > -3.0 SDS and ≤ -2.5 SDS
timeFrame: Day 1
measure: 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
timeFrame: Day 1 and Day 14-45
measure: Proportion of patients having presented at least one historical documented clinically significant episode of hypoglycaemia
timeFrame: Before the start of the study and during the study.
measure: 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)
timeFrame: Day 1
Other outcomes:
Locations:
city: Paris
country: France
facility: Ipsen Central Contact
Study officials:
affiliation: Ipsen
name: Ipsen Medical Director
role: STUDY_DIRECTOR
References: