|- candidate number||9430|
|- NTR Number||NTR2895|
|- ISRCTN||ISRCTN wordt niet meer aangevraagd.|
|- Date ISRCTN created|
|- date ISRCTN requested|
|- Date Registered NTR||11-mei-2011|
|- Secondary IDs||NL34670.068.10 / 10-1-092; CCMO / METC MUMC|
|- Public Title||Metabole effecten van groeihormoon.|
|- Scientific Title||Predictive diagnostic study on the short-term metabolic effects of rhGH treatment in growth hormone deficiency and small for gestational age children.|
|- ACRONYM||Metabolic effects of growth hormone.|
|- hypothesis||The hypothesis is that rhGH treatment in children with GHD or SGA results within 6 weeks in a change of metabolism recognizable as an increase of total energy expenditure (TEE). These change in metabolism can be used as a predictor of growth response in the first year of treatment. |
|- Healt Condition(s) or Problem(s) studied||Growth hormone deficiency, Growth hormone|
|- Inclusion criteria||1. All children scheduled for growth hormone treatment, fulfilling the next criteria:|
A. Children born small for gestational age without catch up growth;
B. Children born with a birth length and/or weight < -2 SDS for gestational age (Niklasson);
C. Short stature defined as height SDS below -2.5 according to the Dutch National Growth References of 1997 and height of ≥ 1.3 SDS below target height SDS.
2. Children with growth hormone deficiency:
A. GHD is confirmed in all patients who during an arginine and clonidine provocation test show a peak GH level of < 20 mU/l.
3. Informed consent;
4. Age ≥ four years.
|- Exclusion criteria||1. Children with a chronological or bone age greater than 8 years for girls and 10 years for boys, because of the influence of puberty;|
2. Children younger that 4 years of age;
3. Children with syndromes or diseases that influence growth otherwise than GDH or SGA;
4. Expected non-compliance based on earlier knowledge over the patient by the opinion of the endocrinologist.
|- mec approval received||no|
|- multicenter trial||no|
|- Type||Single arm|
|- planned startdate ||1-jul-2011|
|- planned closingdate||1-jul-2013|
|- Target number of participants||30|
|- Primary outcome||The relation between short time effects of rhGH treatment on total body water (TBW) and total energy expenditure (TEE) in growth hormone deficient and small for gestational age children and the long-term change in height SDS after one year.|
|- Secondary outcome||What effect has GH on a number of metabolic risk parameters that are typical parameters for the metabolic syndrome in adults?|
|- Timepoints||Total body water (TBW), total energy expenditure (TEE), basal metabolic rate (BMR) and physical activity level (PAL) measurements are performed over a 2-wk period using the doubly labeled water (DLW) method before and during 6 weeks of GH treatment. Markers of metabolic risk factors will be determined during routine blood controls. Baseline characteristics of growth patterns, blood pressure, BMI and waist circumference are collected every three months during routine controls.|
Furthermore, the measurements will be linked with the anthropometric parameters of each individual assembling a prognostic growth profile, therefore the children will be followed during one year of treatment to evaluate the change in height standard deviation score (SDS).
|- Trial web site||N/A|
|- CONTACT FOR PUBLIC QUERIES||MD. D.A. Schott|
|- CONTACT for SCIENTIFIC QUERIES||MD. D.A. Schott|
|- Sponsor/Initiator ||Maastricht University Medical Center (MUMC+)|
(Source(s) of Monetary or Material Support)
|Novo Nordisk Farma B.V. |
|- Brief summary||Short stature is a frequently seen problem for the paediatric endocrinologist. As the most common endocrine cause the diagnosis growth hormone deficiency (GHD) is stated. To diagnose GHD is troublesome, because of the paucity of biological endpoints. Momentarily, GHD is confirmed in children by means of growth hormone provocation tests, but the outcome of these endocrine tests is not discriminative and does not adequately predict the effect of therapy on growth. Besides its growth-promoting effect, growth hormone (GH) also influences metabolism. The changes in metabolism might be useful as a predictor of the growth effect.
There seems to be an association between the disturbance of the growth hormone axis and several features of the metabolic syndrome (MS). The MS is characterized as a cluster of metabolic abnormalities that strongly increase the risk of cardiovascular disease and type II diabetes mellitus in adulthood. It is known that both GH and insulin-like growth factor-I (IGF-I) reduces these cardiovascular risk factors and has beneficial effects on body composition by reducing fat mass and increasing muscle mass. Beside the GHD children also children born small for gestational age (SGA) seem to benefit from rhGH treatment.
The study design is a predictive diagnostic study monitoring the metabolic effects and efficacy of rhGH in GHD and SGA subjects. Total body water (TBW), total energy expenditure (TEE), basal metabolic rate (BMR) and physical activity level (PAL) measurements are performed over a 2-wk period using the doubly labeled water (DLW) method before and during GH treatment. Markers of metabolic risk factors will be determined during routine blood controls. Baseline characteristics of growth patterns, blood pressure, BMI and waist circumference are collected every three months during routine controls. Furthermore, the measurements will be linked with the anthropometric parameters of each individual assembling a prognostic growth profile, therefore the children will be followed during one year of treatment to evaluate the change in height standard deviation score (SDS). |
|- Main changes (audit trail)|
|- RECORD||11-mei-2011 - 18-mei-2011|