Efficacy and safety of growth hormone treatment in short children born small for gestational age; effects of growth hormone levels on growth, insulin sensitivity and body compositio
- Conditions
- Small for gestational age (SGA), children with persistent short staturePregnancy and ChildbirthSlow foetal growth
- Registration Number
- ISRCTN65230311
- Lead Sponsor
- Erasmus Medical Center (The Netherlands)
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- All
- Target Recruitment
- 157
1. Children born with a birth length and/or weight <2 standard devations (SD) for gestational age (Usher McLean)
2. Neonatal period without signs of severe asphyxia (defined as Apgar score <3 after five minutes), and no serious diseases such as long-term artificial ventilation and oxygen supply, bronchopulmonary dysplasia or other chronic lung diseases
3. Short stature defined as a height SD score below 2.5 according to the Dutch National Growth references of 1997
4. Height velocity (cm/year) for chronological age
5. Chronological age at the start of treatment: 3.00 - 7.99 years (boys and girls)
6. Prepubertal signs defined as Tanner stage 1 or testicular volume <4 ml
7. Well documented growth data from birth up to two years and at least one year before the start of the study
8. Both growth hormone deficient and growth hormone insufficient patients
9. Informed consent
1. Chromosomal disorders, known syndromes and serious dysmorphic symptoms suggestive for a syndrome that has not yet been described, except for Silver Russell syndrome
2. Coeliac disease and other chronic or serious diseases of the gastrointestinal tract, heart, genitourinary tract, liver, lungs, skeleton, central nervous system, metabolic disease, chronic or recurrent major infectious diseases, nutritional and/or vitamin deficiencies
3. Any endocrine or metabolic disorder such as diabetes mellitus, diabetes insipidus, hypothyroidism, or inborn errors of metabolism, except for growth hormone deficiency (GHD)
4. Use of medications or interventions at this moment or during the previous six months that might have interfered with growth, such as corticosteroids (including high dose of corticosteroid inhalation), sex steroids, growth hormone, or major surgery (particularly of the spine or extremities)
5. Active malignancy or increased risk of leukaemia
6. Serious suspicion of psychosocial dwarfism (emotional deprivation)
7. Expected non-compliance
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 1. To determine before, during and after treatment termination of long-term growth hormone treatment:<br>a. Insulin sensitivity (via frequent sampling intravenous glucose tolerance test)<br>b. Body composition: in relation to each other and baseline serum GH levels during an overnight GH profile and in relation with six months of treatment with two different GH doses<br>2. To assess the long-term efficacy of biosynthetic GH treatment in a dose of 3 IU/m^2/day on final height and other various auxological parameters
- Secondary Outcome Measures
Name Time Method To assess the safety of GH treatment by studying the short- and long-term effects on:<br>a. Blood pressure<br>b. Thyroid function <br>c. Fasting glucose, insulin and haemoglobin HbA1c (HbA1c) levels