Efficacy and safety of long-term growth hormone treatment In short children born small for gestational age above the age of 8 years
- Conditions
- Persistent short stature, small for gestational age (SGA)Pregnancy and ChildbirthSlow foetal growth and foetal malnutrition
- Registration Number
- ISRCTN18062389
- Lead Sponsor
- Dutch Growth Foundation (Netherlands)
- Brief Summary
1. 2009 results in: http://www.ncbi.nlm.nih.gov/pubmed/19158202 2. 2012 results in: http://www.ncbi.nlm.nih.gov/pubmed/22441140 3. 2013 results in: http://www.ncbi.nlm.nih.gov/pubmed/23125290 4. 2015 results in: http://www.ncbi.nlm.nih.gov/pubmed/26259134 5. 2018 results in: https://www.ncbi.nlm.nih.gov/pubmed/30137415 (added 12/09/2019)
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- All
- Target Recruitment
- 107
1. Children born with a birth length and/or weight less than -2 SD for gestational age
2. Short stature defined in prepubertal children as a height SD score below ?2.5 according to the Dutch National Growth References of 1997 or a predicted final height less than -2.5 SD score, calculated as the height at start of puberty plus 30 cm for boys and +20 cm for girls
3. Height velocity (cm/year) for chronological age less than P50 in pre-pubertal children
4. Chronological age at start of treatment: 8 years or older (boys and girls)
5. Well documented growth data from birth up to 2 years and at least 1 year before the start of the study
6. Informed consent
1. Turner syndrome in girls, known syndromes and serious dysmorphic symptoms suggestive for a syndrome that has not yet been described, except for Silver Russell Syndrome
2. Severe asphyxia (defined as Apgar score less than 3 after 5 minutes), and no serious diseases such as long-term artificial ventilation and oxygen supply, bronchopulmonary dysplasia or other chronic lung disease
3. Coeliac disease and other chronic or serious diseases of the gastrointestinal tract, heart, genito-urinary tract, liver, lungs, skeleton or central nervous system, or chronic or recurrent major infectious diseases, nutritional and/or vitamin deficiencies
4. Any endocrine or metabolic disorder such as diabetes mellitus, diabetes insipidus, hypothyroidism, or inborn errors of metabolism, except of GHD
5. Medications or interventions during the previous 6 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)
6. Use of medication that might interfere with growth during GH therapy, such as corticosteroids, sex steroids, LHRH analogue
7. Active or treated malignancy or increased risk of leukaemia
8. Serious suspicion of psychosocial dwarfism (emotional deprivation)
9. Expected non-compliance
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <br> 1. To assess the effect of doubling the GH dose from 1 to 26 mg\m^2\day versus continuation of treatment with 1 mg GH\m^2\day on final height, at onset of puberty in short SGA children who start puberty at a height above 140 cm<br> 2. To determine the dose-response effect of 1 versus 2 mg GH\m^2\day in combination with LHRH analogue treatment for 2 years on final height in short SGA children who start puberty at a height below 140 cm<br> 3. To determine before and during long-term growth hormone treatment: insulin sensitivity and body composition<br>
- Secondary Outcome Measures
Name Time Method <br> To assess the safety of GH treatment by studying the short- and long-term effects on:<br> 1. Blood pressure<br> 2. Thyroid function<br> 3. Fasting glucose and insulin and HbA1c levels<br>