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Growth Response in Short Children Suffering From a Disease With Growth Retardation and Treated With Somatropin

Phase 2
Completed
Conditions
Idiopathic Short Stature
Registration Number
NCT00488124
Lead Sponsor
University of Erlangen-Nürnberg Medical School
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.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
120
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

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Primary Outcome Measures
NameTimeMethod
To show an improvement of height, change in height (SDS) under GH treatment one year after visit 2 (start of GH therapy).one and two years of observation
Secondary Outcome Measures
NameTimeMethod
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)one and two years of observation period

Trial Locations

Locations (13)

Kinderklinik, Universitätsklinikum der RWTH Aachen, Pauwelsstraße 30

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Aachen, Germany

Klinik und Poliklinik für Kinder und Jugendliche der Technischen Universität Dresden, Fetscherstr. 74

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Dresden, Germany

Klinik für Kinder und Jugendliche, Schwerpunkt Kinder-Endokrinologie und - Diabetologie, Loschgestr. 15

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Erlangen, Germany

Klinik für Päd. Hämatologie, Onkologie und Endokrinologie, Zentrum für Kinderheilkunde, der Universität Duisburg-Essen, Hufelandstrasse 55

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Essen, Germany

Kinder- und Jugendärztin, Pippinplatz 4

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Gauting, Germany

Endokrinologikum Hamburg, Lornsenstrasse 4 - 6

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Hamburg, Germany

Universitätsklinik für Kinder- und Jugendliche, Abt. Kinderheilkunde, Im Neuenheimer Feld 430

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Heidelberg, Germany

Klinik u. Poliklinik für Kinder und Jugendliche, Med. Einrichtungen der Universität Köln, Joseph-Stelzmann-Strasse 9

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Köln, Germany

Universitätsklinik für Kinder- und Jugendmedizin, Kirrberger Strasse

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Homburg/Saar, Germany

Klinik für Allgemeine Pädiatrie und Neonatologie, Otto-von-Guericke-Universität Magdeburg, Leipziger Straße 44

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Magdeburg, Germany

Zentrum für Frauen und Kindermedizin, Liebigstrasse 20 a

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Leipzig, Germany

Kinderarztpraxis, Brabeckstrasse 153

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Hannover, Germany

Klinik für Kinderheilkunde und Jugendmedizin, Sektion Pädiatrische Endokrinologie, Hoppe-Seyler-Straße 1

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Tübingen, Germany

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