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

NCT00710307 | Completed

• 1 other identifier: 2-79-52800-001
• Study Type: observational
• Target: 275
• Locations: 1 country
• Sites: 1

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

Conditions

Idiopathic Short Stature

Outcome Measures

Primary Outcomes (1)

• 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

  Day 1 for the first sample; between Day 14 and Day 45 for the second sample

Secondary Outcomes (4)

• Proportion of patients with height ≤ -3.0 SDS,and height > -3.0 SDS and ≤ -2.5 SDS

  Day 1

• 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

  Day 1 and Day 14-45

• Proportion of patients having presented at least one historical documented clinically significant episode of hypoglycaemia

  Before the start of the study and during the study.

• 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)

  Day 1

Interventions

Blood sampling PROCEDURE

Blood samples will be collected at visit 1 (day 1) and at visit 2 (day 14 to 45).

Genetic analysis GENETIC

The sample for genetic analyses may be taken at Visit 1. The blood sample volume will be 6 mL.

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Idiopathic Short Stature children

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Ipsen: lead

Study Sites (1)

Ipsen Central Contact

Paris, France

Location

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.

MeSH Terms

Interventions

Blood Specimen Collection; Genetic Testing

Intervention Hierarchy (Ancestors)

Specimen Handling; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Punctures; Surgical Procedures, Operative; Investigative Techniques; Genetic Techniques; Genetic Services; Health Services; Health Care Facilities Workforce and Services; Diagnostic Services; Preventive Health Services

Study Officials

• Ipsen Medical Director

  Ipsen

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 2, 2008
• First Posted: July 4, 2008
• Study Start: October 1, 2008
• Primary Completion: January 1, 2010
• Study Completion: January 1, 2010
• Last Updated: January 15, 2019

Record last verified: 2019-01

Locations

FR (1)