A study on the Tissue Responsiveness to short term exogenous GH in children with idiopathic short stature in relation to their reponse to long-term treatment.|
|- candidate number||1598|
|- NTR Number||NTR380|
|- Date ISRCTN created||19-dec-2005|
|- date ISRCTN requested||28-okt-2005|
|- Date Registered NTR||12-sep-2005|
|- Secondary IDs||N/A |
|- Public Title||A study on the Tissue Responsiveness to short term exogenous GH in children with idiopathic short stature in relation to their reponse to long-term treatment.|
|- Scientific Title||A study on the Tissue Responsiveness to short term exogenous GH in children with idiopathic short stature in relation to their reponse to long-term treatment.|
|- ACRONYM||Dose-response study|
|- hypothesis||The change in biochemical parameters of bone and colleagen metabolism during a shortterm GH dose-response study predicts the long-term effect of GH on growth. Idiopathic short stature is partially explainable by an abnormal tissue responsiveness to GH and IGF-I. GH theray in a dosage of 6 IU//mw.day administered before puberty increases height velocity, height in adolescence and final height. GH administration affects puberty onset and its duration. GH administration affects quality of life. |
|- Healt Condition(s) or Problem(s) studied||Idiopathic Short Stature (ISS)|
|- Inclusion criteria||40 children. Height SDS<-2, prepubertal, age 4-8 (F) or 4-10 (M), GH response to provocation tests >20 mU/l, normal sitting height.height ratio, normal screening blood tests and urinanalysis.|
|- Exclusion criteria||Any systemic disease during childhood that limits the growth potential or may interfere with the evaluation of the effectiveness of therapy.|
|- mec approval received||yes|
|- multicenter trial||yes|
|- planned startdate ||1-jan-1994|
|- planned closingdate||1-jan-2009|
|- Target number of participants||40|
|- Interventions||After randomisation, the control group did not receive treatment, and were followed yearly for growth and puberty assessment. |
The treatment group underwent two 3 months periods of GH administration (1.5 IU/m2.d, 3.0 IU/m2.d) with 3 months washout periods in between. Thereafter 6 IU/m2.d was given until the beginning of puberty.
|- Primary outcome||Height at stop of therapy (at onset of puberty) and final height. |
|- Secondary outcome||1. Timing of onset of puberty; |
- duration of puberty;
2. Relation between long-term growth response (dependent variable) and short-term growth response on various dosages and in vitro responsiveness of cultured skin fibroblasts to GH and IGF-I;
3. Effect of GH therapy on quality of life.
|- Trial web site||N/A|
|- status||inclusion stopped: follow-up|
|- CONTACT FOR PUBLIC QUERIES||Prof. Dr. J.M. Wit|
|- CONTACT for SCIENTIFIC QUERIES||Prof. Dr. J.M. Wit|
|- Sponsor/Initiator ||Pfizer B.V. (Pfizer Inc, New York)|
(Source(s) of Monetary or Material Support)
|Netherlands Organization for Scientific Research (NWO)|
|- Publications||Kamp, G.A., Rekers-Mombarg L.T.M., Wit, J.M. Does GH treatment affect pubertal timing in children with idiopathic short stature? Highlights 1997;5:12-15. 205.|
Kamp, G.A., Wit, J.M. High-dose growth hormone therapy in idiopathic short stature. Horm Res 1998;49 (suppl 2):67-72. 238.
Rekers-Mombarg, L.T.M., Kamp, G.A., Massa, G.G., Wit, J.M. Influence of growth hormone treatment on pubertal timing and pubertal growth in children with idiopathic short stature. J Pediatr Endocrinol Metab 1999;12 (5):611-622 287.
Theunissen N.C.M., Kamp, G.A., Koopman, H.M., Zwinderman, K.A.H., Vogels, M.A., Wit, J.M. Quality of life and self esteem in children treated for idiopathic short stature. J.Pediatrics, 2002;140:507-15 288.
Kamp, G.A. Waelkens, J.J.J., De Muinck Keizer-Schrama, S.M.P.F., Delemarre-van de Waal, H.A., Verhoeven-Wind, L., Wit, J.M. High dose growth hormone treatment induces acceleration of skeletal maturation and an earlier onset of puberty in children with idiopathic short stature. Arch Dis Child, 2002;87:215-220289.
Kamp, G.A., Ouwens, D.M., Hoogerbrugge, C.M., Zwinderman, A.H., Maassen, J.A., Wit, J.M. Skin fibroblasts of children with idiopathic short stature show an increased mitogenic response to IGF-I and secrete more IGFBP-3 compared to normal children. Clin Endocrinol 2002;56:439-447 294.
Kamp, G.A. , Zwinderman, A.H., Doorn, J. van, Hackeng, W., Frölich, M., Schönau, E., Wit, J.M. Biochemical markers of growth hormone (GH) sensitivity in children with idiopathic short stature: individual capacity of IGF-I generation after high dose GH treatment determines the growth response to GH. Clin Endocrinol 2002; 57:315-325
|- Brief summary||The predictive power of the diagnostic phase (dose-response relationship, in vitro responsiveness of skin fibroblasts) is limited. GH accelerates puberty onset and duration. No clear effect on quality of life was found. |
|- Main changes (audit trail)|
|- RECORD||12-sep-2005 - 6-mrt-2006|
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