Predictive value of the IGF-I generation test to predict the results of growth hormone treatment in short children.

• Conditions: Severe idiopathic short stature; MedDRA version: 14.0Level: LLTClassification code 10066333Term: Idiopathic short statureSystem Organ Class: 10028395 - Musculoskeletal and connective tissue disorders; Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11]

• Registration Number: EUCTR2010-019980-13-NL
• Lead Sponsor: Ipsen Farmaceutica b.v.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2011-07-11
• Last Update Posted: 14 October 2013

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

Prepubertal children aged 2.0-9.0 (females) or 2.0-10.0 (males) years, bone age <9 (females) or <10 (males) years, height SDS<-2.5 (for ethnically adequate references), peak GH after provocation >30 mU/L, IGF-I SDS < -2 (at least twice, one of which determined in UMCU), body mass index (BMI) SDS > -2
Are the trial subjects under 18? yes
Number of subjects for this age range: 20
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

•Has a history of hypersensitivity to growth hormone or phenol (conservative added to GH in NutropinAq), or drugs with a similar chemical structure.
•Was treated with any other Investigational Medicinal Product (IMP) within the last 30 days before study entry.
•Has any mental condition rendering the subject unable to understand the nature, scope and possible consequences of the study, and/or evidence of an uncooperative attitude.
•Has abnormal baseline findings, any other medical condition(s) or laboratory findings that, in the opinion of the Investigator, might jeopardise the subject’s safety or decrease the chance of obtaining satisfactory data needed to achieve the objectives of the study.
•Has a birth weight and/or length below -2 SDS for Swedish reference charts (2). Patients will not be excluded due to an unknown birth weight or length.
•Has a known cause of short stature, or any significant concomitant disease that is likely to interfere with growth or with the study schedule/objectives, or is a known contraindication to GH treatment such as: chromosomal abnormalities (known syndromes associated with short stature, and skeletal dysplasias); growth failure due to high doses of glucocorticosteroids, hypothyroidism, chronic illnesses; malignancy, intra-cranial tumor; chronic disease such as insulin-dependent diabetes mellitus; chronic infectious disease; chronic renal insufficiency; chronic heart failure; chronic hepatic disease; celiac disease; chronic pulmonary disease; active rheumatic disease; psychosis; neurofibromatosis; McCune Albright syndrome; dysmorphic syndromes such as Russell-Silver syndrome, Leri-Weill syndrome, achondroplasia, etc; and emotional deprivation. Hypothyroidism adequately substituted with thyroid hormone replacement therapy is not an exclusion criterion. For girls, the karyotype, to eliminate a Turner syndrome, is mandatory. In case of a positive Rappold score (> 4) (3), a SHOX defect has to be excluded.
•Has dysmorphic features suspect for chromosomal breakage syndromes.
•Has known causes of decreased IGF-I (e.g. undernutrition).
•Has an abnormal sitting height:height ratio SDS (<-2 or >+2), corrected for bone age, according to Dutch references (4).
•Has any abnormality at laboratory screening according to the Dutch consensus protocol (see appendix 3).
•Is likely to require treatment during the study with drugs that are not permitted by the study protocol (see appendix 4).
•Has active neoplasia or suspected neoplasia.
•The parents are investigator site personnel directly affiliated with the study, or are the immediate family of investigator site personnel directly affiliated with the study. Immediate family is defined as spouse, parent, child, or sibling, whether biological or legally adopted.
•Is unable or unwilling to comply with the study visits or the test schedule required by the protocol.
•Patients not affiliated to the health insurance system will not be allowed to participate in this trial.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified