Clinical trial NCT00488124: Growth Response in Short Children Suffering From a Disease With Growth Retardation and Treated With Somatropin
Official title: Evaluation of Growth Response in Short Children Suffering From a Disease With Growth Retardation and Treated With Somatropin: A Prospective, Longitudinal Non-randomised, Open, Phase II Study
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Idiopathic Short Stature
Keywords: short stature
Lead sponsor: University of Erlangen-Nürnberg Medical School
Sponsor class: OTHER
First posted: 2007-06-19
Enrollment: 120
Sex: ALL
Minimum age (years): 4
Maximum age (years): 10
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Growth hormone therapy will improve the height of short statured children with pathological conditions that lead to growth retardation. Growth hormone therapy will show an increase in height velocity \>1 SD compared to pretreatment height velocity. and the therapy will be safe.

Detailed description: Growth hormone (GH, Somatropin, e.g. Genotropin®) is approved in the treatment of children in specific indications. However, besides the benefit in approved indications, a benefit can also be achieved in other pathological conditions that lead to growth retardation. However, because of their relative low frequency and the long duration of GH studies in children, few data or only case reports on GH treatment are available in these conditions. Nevertheless, published data have suggested a benefit of GH treatment in children suffering from some of those diseases and pediatric endocrinologists sometimes request GH treatment for those children to improve growth rate. This protocol is designed to allow such children with severe growth retardation to be treated with GH and will allow those children to be carefully followed-up and finally evaluated at the end of the GH treatment period.

Eligibility criteria: Inclusion Criteria:

* Severe growth retardation (\< -2,5 height SDS and annual growth velocity (HV SDS) \< 0 SD according to Reinken (1992) and parental adjusted target height \< -1 SD according to Tanner (1986)
* Chronological age \> 4 and \< 10 years, prepubertal children; for girls: Tanner breast stage B = 1, for boys: testis volume ≤ 3 ml
* Any disease which is NOT part of the registered indications for GH treatment in Germany
* Written informed consent from both parents and from the patients if she/he is able to receive and understand the information
* GH treatment requested by an expert in pediatric endocrinology

Exclusion Criteria:

* Participation in any other clinical study
* Unable to follow the and/or comprehend the protocol ( e.g. severe mental retardation)
* Previous history of intolerance or hypersensitivity to the study drug
* History of malignancy
* Chromosomal anomalies with increased risk for malignancy

Primary outcomes:
measure: To show an improvement of height, change in height (SDS) under GH treatment one year after visit 2 (start of GH therapy).
timeFrame: one and two years of observation
Secondary outcomes:
measure: To show an increase in height velocity >1 SD compared to pretreatment height velocity, to confirm good clinical and biological safety of GH treatment in these patients (e.g. adverse events, serum IGF-I, fasting blood glucose and insulin)
timeFrame: one and two years of observation period
Other outcomes:
Locations:
city: Aachen
country: Germany
facility: Kinderklinik, Universitätsklinikum der RWTH Aachen, Pauwelsstraße 30
city: Dresden
country: Germany
facility: Klinik und Poliklinik für Kinder und Jugendliche der Technischen Universität Dresden, Fetscherstr. 74
city: Erlangen
country: Germany
facility: Klinik für Kinder und Jugendliche, Schwerpunkt Kinder-Endokrinologie und - Diabetologie, Loschgestr. 15
city: Essen
country: Germany
facility: Klinik für Päd. Hämatologie, Onkologie und Endokrinologie, Zentrum für Kinderheilkunde, der Universität Duisburg-Essen, Hufelandstrasse 55
city: Gauting
country: Germany
facility: Kinder- und Jugendärztin, Pippinplatz 4
city: Hamburg
country: Germany
facility: Endokrinologikum Hamburg, Lornsenstrasse 4 - 6
city: Hannover
country: Germany
facility: Kinderarztpraxis, Brabeckstrasse 153
city: Heidelberg
country: Germany
facility: Universitätsklinik für Kinder- und Jugendliche, Abt. Kinderheilkunde, Im Neuenheimer Feld 430
city: Homburg/Saar
country: Germany
facility: Universitätsklinik für Kinder- und Jugendmedizin, Kirrberger Strasse
city: Köln
country: Germany
facility: Klinik u. Poliklinik für Kinder und Jugendliche, Med. Einrichtungen der Universität Köln, Joseph-Stelzmann-Strasse 9
city: Leipzig
country: Germany
facility: Zentrum für Frauen und Kindermedizin, Liebigstrasse 20 a
city: Magdeburg
country: Germany
facility: Klinik für Allgemeine Pädiatrie und Neonatologie, Otto-von-Guericke-Universität Magdeburg, Leipziger Straße 44
city: Tübingen
country: Germany
facility: Klinik für Kinderheilkunde und Jugendmedizin, Sektion Pädiatrische Endokrinologie, Hoppe-Seyler-Straße 1
Study officials:
affiliation: Friedrich-Alexander-Universität Erlangen-Nürnberg
name: Helmuth-Günther Doerr, Prof. Dr.
role: STUDY_CHAIR
References: