Growth Response in Short Children Suffering From a Disease With Growth Retardation and Treated With Somatropin (ISS)
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
Idiopathic Short Stature |
Drug: somatropin (Genotropin® treatment) |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| 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 |
- To show an improvement of height, change in height (SDS) under GH treatment one year after visit 2 (start of GH therapy). [ Time Frame: one and two years of observation ] [ Designated as safety issue: No ]
- 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) [ Time Frame: one and two years of observation period ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 120 |
| Study Start Date: | November 2005 |
| Study Completion Date: | March 2011 |
| Primary Completion Date: | February 2011 (Final data collection date for primary outcome measure) |
-
Drug: somatropin (Genotropin® treatment)
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| Ages Eligible for Study: | 4 Years to 10 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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
Contacts and Locations| Germany | |
| Kinderklinik, Universitätsklinikum der RWTH Aachen, Pauwelsstraße 30 | |
| Aachen, Germany, 52074 | |
| Klinik und Poliklinik für Kinder und Jugendliche der Technischen Universität Dresden, Fetscherstr. 74 | |
| Dresden, Germany, 01307 | |
| Klinik für Kinder und Jugendliche, Schwerpunkt Kinder-Endokrinologie und - Diabetologie, Loschgestr. 15 | |
| Erlangen, Germany, 91054 | |
| Klinik für Päd. Hämatologie, Onkologie und Endokrinologie, Zentrum für Kinderheilkunde, der Universität Duisburg-Essen, Hufelandstrasse 55 | |
| Essen, Germany, 45122 | |
| Kinder- und Jugendärztin, Pippinplatz 4 | |
| Gauting, Germany, 82131 | |
| Endokrinologikum Hamburg, Lornsenstrasse 4 - 6 | |
| Hamburg, Germany, 22767 | |
| Kinderarztpraxis, Brabeckstrasse 153 | |
| Hannover, Germany, 30539 | |
| Universitätsklinik für Kinder- und Jugendliche, Abt. Kinderheilkunde, Im Neuenheimer Feld 430 | |
| Heidelberg, Germany, 69120 | |
| Universitätsklinik für Kinder- und Jugendmedizin, Kirrberger Strasse | |
| Homburg/Saar, Germany, 66421 | |
| Klinik u. Poliklinik für Kinder und Jugendliche, Med. Einrichtungen der Universität Köln, Joseph-Stelzmann-Strasse 9 | |
| Köln, Germany, 50931 | |
| Zentrum für Frauen und Kindermedizin, Liebigstrasse 20 a | |
| Leipzig, Germany, 04103 | |
| Klinik für Allgemeine Pädiatrie und Neonatologie, Otto-von-Guericke-Universität Magdeburg, Leipziger Straße 44 | |
| Magdeburg, Germany, 39112 | |
| Klinik für Kinderheilkunde und Jugendmedizin, Sektion Pädiatrische Endokrinologie, Hoppe-Seyler-Straße 1 | |
| Tübingen, Germany, 72076 | |
| Study Chair: | Helmuth-Günther Doerr, Prof. Dr. | Friedrich-Alexander-Universität Erlangen-Nürnberg |
More Information
No publications provided
| Responsible Party: | Professor Dr. med. Doerr, Childrens´ hospital |
| ClinicalTrials.gov Identifier: | NCT00488124 History of Changes |
| Other Study ID Numbers: | NRA6280030 |
| Study First Received: | June 18, 2007 |
| Last Updated: | March 29, 2011 |
| Health Authority: | Germany: Federal Institute for Drugs and Medical Devices |
Keywords provided by University of Erlangen-Nürnberg Medical School:
|
short stature |
Additional relevant MeSH terms:
|
Dwarfism Stress, Psychological Bone Diseases, Developmental Bone Diseases |
Musculoskeletal Diseases Genetic Diseases, Inborn Endocrine System Diseases Behavioral Symptoms |
ClinicalTrials.gov processed this record on May 23, 2013