Onderzoek met mensen

Overzicht van medisch-wetenschappelijk onderzoek in Nederland

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.

Gepubliceerd: Laatst bijgewerkt:

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…

Download: PDF | XML
Ethische beoordeling
Positief advies
Status
Werving gestopt
Type aandoening
-
Onderzoekstype
Interventie onderzoek
Zet begrijpelijke taal modus aan

ID

NL-OMON24302

Bron

NTR

Verkorte titel

Dose-response study

Aandoening

Children with idiopathic short stature (ISS)

Ondersteuning

Primaire sponsor :
Pfizer (New York) (previously Pharmacia), through local representatives in the Netherlands.
Overige ondersteuning :
NWO (Netherlands Medical Research Council)

Onderzoeksproduct en/of interventie

Uitkomstmaten

Primaire uitkomstmaten

Height at stop of therapy (at onset of puberty) and final height.

Achtergrond van het onderzoek

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.

Doel van het onderzoek

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.

Onderzoeksproduct en/of interventie

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.

Publiek

Leiden University Medical Center (LUMC),
Department of Pediatrics,
P.O. Box 9600
J.M. Wit
Albinusdreef 2
Amsterdam 2300 RC
The Netherlands
+31 (0)71 5262824
j.m.wit@lumc.nl

Wetenschappelijk

Leiden University Medical Center (LUMC),
Department of Pediatrics,
P.O. Box 9600
J.M. Wit
Albinusdreef 2
Amsterdam 2300 RC
The Netherlands
+31 (0)71 5262824
j.m.wit@lumc.nl

Belangrijkste voorwaarden om deel te mogen nemen (Inclusiecriteria)

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.

Belangrijkste redenen om niet deel te kunnen nemen (Exclusiecriteria)

Any systemic disease during childhood that limits the growth potential or may interfere with the evaluation of the effectiveness of therapy.

Opzet

Type
:
Interventie onderzoek
Onderzoeksmodel
:
Parallel
Blindering
:
Open / niet geblindeerd
Controle
:
Geneesmiddel

Deelname

Nederland
Status
:
Werving gestopt
(Verwachte) startdatum :
Aantal proefpersonen :
40
Type :
Werkelijke startdatum

Positief advies
Datum :
Soort :
Eerste indiening

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 NL342
NTR-old NTR380
Ander register : N/A
ISRCTN ISRCTN52337368

Samenvatting resultaten

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.<br> Kamp, G.A., Wit, J.M. High-dose growth hormone therapy in idiopathic short stature. Horm Res 1998;49 (suppl 2):67-72. 238. <br>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. <br>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. <br>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. <bR>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. <br>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