GH treatment of short, small-for-gestational-age children has a beneficial effect on linear growth.
ID
Bron
Verkorte titel
Aandoening
1. Small for gestational age (SGA);
2. Intrauterine growth retardation (IUGR).
Ondersteuning
Onderzoeksproduct en/of interventie
Uitkomstmaten
Primaire uitkomstmaten
1. To assess the effect of GH therapy on:<br>
a. linear growth;<br>
b. bone maturation;<br>
c. pubertal development;<br>
d. final height;<br>
in children with IUGR and no catch-up growth.
Achtergrond van het onderzoek
Multicentred, double-blind, randomized, two-arm trial comparing two dose regimens of Norditropin® (a 2-year initial trial 14/NL and the trial extensions 20/NL (2-years) and 21/NL (till final height). In trial 14/NL, children were randomized to receive GH at either 3 IU (~1mg)/m2/day or 6 IU (~2mg)/m2/day for a 2-year treatment period. The children were stratified by age (3.00-5.99 years; 6.00-8.99 years; 9.00-10.99 years) and by their plasma 24-hour GH profile (normal GH insufficient, unknown).
Subjects who completed this trial continued in the trial extension 20/NL, and continued treatment, without interruption, in double-blind fashion at the dose level at which they were originally randomised.
Eleven older children who did not meet the criteria on age and puberty were included in a separate protocol. These children were treated according to protocol addendum GHRETARD/BPD/16/NL. Trial conduct in 16/NL was the same as that for 14/NL with the exception that all children received GH at 6 IU (~2mg)/m2/day. After two years of treatment, these children were allowed to continue in trial extension 20/NL.
Doel van het onderzoek
GH treatment of short, small-for-gestational-age children has a beneficial effect on linear growth.
Onderzoeksopzet
N/A
Onderzoeksproduct en/of interventie
Growth hormone treatment in either 3 or 6 IU (~1 or 2 mg)/m2/day (randomized double-blind dose-response trial).
Publiek
P.O. Box 2060
A.C.S. Hokken-Koelega
Dr. Molewaterplein 60
Rotterdam 3000 CB
The Netherlands
+31 (0)10 4636744
a.hokken@erasmusmc.nl
Wetenschappelijk
P.O. Box 2060
A.C.S. Hokken-Koelega
Dr. Molewaterplein 60
Rotterdam 3000 CB
The Netherlands
+31 (0)10 4636744
a.hokken@erasmusmc.nl
Belangrijkste voorwaarden om deel te mogen nemen (Inclusiecriteria)
1. Birth length
2. Uncomplicated neonatal period, defined as no signs of:
a. severe asphyxia (Apgar score <3 after 5 minutes);
b. complicated sepsis neonatorum;
c. long-term complicated respiratory ventilation (for instance, bronchopulmonary dysplasia or pneumothorax);
3. No catch-up growth defined as obtaining a height of >= P3 (Roede), within the first two years of life or at a later stage;
4. Height velocity (HV) (cm/year) for chronological age <= P50 (Tanner);
5. Chronological age at start of treatment: girls: 3.00 to 8.99 years; boys: 3.00 to 10.99 years;
6. Prepubertal signs as defined by 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 start of treatment;
8. Written informed consent from child and/or parents/guardians.
Belangrijkste redenen om niet deel te kunnen nemen (Exclusiecriteria)
1. Any endocrine or metabolic disorder (such as diabetes mellitus, diabetes insipidus, hypothyroidism, or inborn errors of metabolism);
2. Disorders of the genito-urinary tract, cardio-pulmonary or gastro-intestinal tract, or nervous system, nutritional and/or vitamin deficiencies;
3. Chromocomal abnormalities or signs of a syndrome, except for Silver-Russel syndrome;
4. Chondrodysplasia;
5. Hydrocephalus;
6. Subjects with active malignant diseases or with increased risk of leukaemia;
7. Serious suspicion of psychosocial dwarfism (emotional deprivation);
8. Previous anabolic sex steroid or GH therapy.
Opzet
Deelname
Opgevolgd door onderstaande (mogelijk meer actuele) registratie
Geen registraties gevonden.
Andere (mogelijk minder actuele) registraties in dit register
Geen registraties gevonden.
In overige registers
| Register | ID |
|---|---|
| NTR-new | NL1008 |
| NTR-old | NTR1037 |
| Ander register | : |
| ISRCTN | ISRCTN wordt niet meer aangevraagd |
Samenvatting resultaten
2. Sas, T., de Waal, W., Mulder, P., Houdijk, M., Jansen, M., Reeser, M. & Hokken-Koelega, A. (1999) Growth hormone treatment in children with short stature born small for gestational age: 5-year results of a randomized, double-blind, dose-response trial. J Clin Endocrinol Metab, 84, 3064-3070;<br>
3. Sas, T., Mulder, P. & Hokken-Koelega, A. (2000) Body composition, blood pressure, and lipid metabolism before and during long-term growth hormone (GH) treatment in children with short stature born small for gestational age either with or without GH deficiency. J Clin Endocrinol Metab, 85, 3786-3792;<br>
4. Sas, T.C., Gerver, W.J., De Bruin, R., Mulder, P.G., Cole, T.J., De Waal, W. & Hokken-Koelega, A.C. (2000) Body proportions during 6 years of GH treatment in children with short stature born small for gestational age participating in a randomised, double-blind, dose-response trial. Clin Endocrinol (Oxf), 53, 675-681;<br>
5. Sas, T., Mulder, P., Aanstoot, H.J., Houdijk, M., Jansen, M., Reeser, M. & Hokken-Koelega, A. (2001) Carbohydrate metabolism during long-term growth hormone treatment in children with short stature born small for gestational age. Clin Endocrinol (Oxf), 54, 243-251;<br>
6. van Pareren, Y., Mulder, P., Houdijk, M., Jansen, M., Reeser, M. & Hokken-Koelega, A. (2003) Effect of discontinuation of growth hormone treatment on risk factors for cardiovascular disease in adolescents born small for gestational age. J Clin Endocrinol Metab, 88, 347-353;<br>
7. Van Pareren, Y., Mulder, P., Houdijk, M., Jansen, M., Reeser, M. & Hokken-Koelega, A. (2003) Adult height after long-term, continuous growth hormone (GH) treatment in short children born small for gestational age: results of a randomized, double-blind, dose-response GH trial. J Clin Endocrinol Metab, 88, 3584-3590;<br>
8. van Pareren, Y.K., Duivenvoorden, H.J., Slijper, F.S., Koot, H.M. & Hokken-Koelega, A.C. (2004) Intelligence and psychosocial functioning during long-term growth hormone therapy in children born small for gestational age. J Clin Endocrinol Metab, 89, 5295-5302;<br>
9. Bannink, E.M., van Pareren, Y.K., Theunissen, N.C., Raat, H., Mulder, P.G. & Hokken-Koelega, A.C. (2005) Quality of life in adolescents born small for gestational age: does growth hormone make a difference? Horm Res, 64, 166-174;<br>
10. Bannink, E.M., van Doorn, J., Mulder, P.G. & Hokken-Koelega, A.C. (2007) Free/Dissociable Insulin-Like Growth Factor (IGF)-I, Not Total IGF-I, Correlates with Growth Response during Growth Hormone Treatment in Children Born Small for Gestational Age. J Clin Endocrinol Metab, 92, 2992-3000.