The primary objective of this study is to assess the effect of individualizing the growth hormone dose versus standard treatment with 1 mg/m2/day on adult height and the first and five year growth response. Secondly, we want to assess the long term…
ID
Source
Brief title
Condition
- Endocrine and glandular disorders NEC
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The effect of individual growth hormone therapy compared to standard treatment
with 1 mg/m2/day, assessment of long-term safety and psychosocial development.
Secondary outcome
n.a.
Background summary
Children born small for gestational age with persistent short stature can be
effectively treated with growth hormone. Several studies have described the
short-term and long-term growth response of different growth hormone dosages,
but the optimal dose for individual short SGA children has not yet been
established. De Ridder et al. developed a model to predict height at the onset
of puberty and adult height for short children born SGA who will start with
growth hormone treatment. The model developed by De Ridder et al. can be used
for prediction of adult height for an individual child. This allows a better
determination of the growth hormone dose that should be prescribed for each
individual patient. Using this prediction model to determine individual GH
dosages might make better individual treatment possible.
Study objective
The primary objective of this study is to assess the effect of individualizing
the growth hormone dose versus standard treatment with 1 mg/m2/day on adult
height and the first and five year growth response. Secondly, we want to assess
the long term safety of growth hormone therapy on insulin sensitivity and body
composition, to determine the effect of growth hormone therapy on psychosocial
development and intelligence, and to assess possible genetic causes of short
stature in children born SGA
Study design
Randomised, open labelled multicenter growth hormone trial.
Intervention
Patients are randomly assigned to one of the two GH dose regimens (1 mg/m2/day
versus individualized dose) at the start of the study.
Study burden and risks
Since June 2005 growth hormone treatment is licensed for short children born
SGA (EMEA). Studies on growth hormone treatment did not reveal any deleterious
effects of this therapy so far. Subjects will visit the local outpatient clinic
on a three monthly basis. Yearly, blood samples will be drawn and bone age will
be determined using X-rays of the left hand and wrist. These visits and
measurements are in accordance with the national guidelines on treatment of
short SGA children. A subgroup will have a two-yearly, more extensive
investigation at the Erasmus MC / Sophia.
Westzeedijk 106
Rotterdam 3016 AH
NL
Westzeedijk 106
Rotterdam 3016 AH
NL
Listed location countries
Age
Inclusion criteria
- Children born with a birth length and/or weight < -2 SDS for gestational age (Niklasson)
- Short stature defined as height SD score below *2.5 according to the Dutch National Growth References of 1997.
- Height of * 1 SD score below target height SD score (TH SDS).
- Height velocity (cm/year) for chronological age * 0 SDS in prepubertal children.
- Chronological age at start of treatment between 4 and 11 years for boys and between 4 and 9 years for girls.
- Bone age (G&P) * 13 years for girls and * 15 years for boys.
- Well documented growth data from birth up to 2 years and at least 1 year before the start of the study.
- Informed consent.
Exclusion criteria
- Syndromes (except for Silver Russell Syndrome), chromosomal abnormalities and serious dysmorphic symptoms suggestive for a syndrome that has not yet been described.
- severe psychomotor retardation according to DSM IV.
- Complicated neonatal period.
- Celiac disease and other chronic or serious diseases of the gastro-intestinal tract, heart, genito-urinary tract, liver, lungs, skeleton or central nervous system, or chronic or recurrent major infectious diseases, nutritional and/or vitamin deficiencies.
- Any endocrine or metabolic disorder, except for growth hormone deficiency (GHD).
- Genetic alterations in the IGF-I receptor gene.
- Medications or interventions during the previous 6 months that might have interfered with growth.
- Use of medication that might interfere with growth during growth hormone therapy.
- Active or treated malignancy or increased risk of leukaemia.
- Serious suspicion of psychosocial dwarfism (emotional deprivation).
- Expected non-compliance.
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2009-011155-40-NL |
| CCMO | NL27033.078.09 |