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

This study has been completed.
Sponsor:
Information provided by:
University of Erlangen-Nürnberg Medical School
ClinicalTrials.gov Identifier:
NCT00488124
First received: June 18, 2007
Last updated: March 29, 2011
Last verified: March 2011
  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

Resource links provided by NLM:


Further study details as provided by University of Erlangen-Nürnberg Medical School:

Primary Outcome Measures:
  • 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 ]

Secondary Outcome Measures:
  • 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)
Intervention Details:
    Drug: somatropin (Genotropin® treatment)
    0,035 mg /kg bodyweight of Somatropin per day given by subcutaneous injections through an injection device (GenotropinPEN)
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

Ages Eligible for Study:   4 Years to 10 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00488124

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
Sponsors and Collaborators
University of Erlangen-Nürnberg Medical School
Investigators
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
Bone Diseases
Bone Diseases, Developmental
Endocrine System Diseases
Genetic Diseases, Inborn
Musculoskeletal Diseases

ClinicalTrials.gov processed this record on November 20, 2014