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

Sponsor

University of Erlangen-Nürnberg Medical School (Other)

Overall Status

Completed

CT.gov ID

NCT00488124

Collaborator

(none)

120

Enrollment

Locations

63.9

Duration (Months)

9.2

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Idiopathic Short Stature	Drug: somatropin (Genotropin® treatment)	Phase 2

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.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

120 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (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

Study Start Date :

Nov 1, 2005

Actual Primary Completion Date :

Feb 1, 2011

Actual Study Completion Date :

Mar 1, 2011

Outcome Measures

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). [one and two years of observation]

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) [one and two years of observation period]

Eligibility Criteria

Criteria

Ages Eligible for Study:

4 Years to 10 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Locations

	Site	City	Country	Postal Code
1	Kinderklinik, Universitätsklinikum der RWTH Aachen, Pauwelsstraße 30	Aachen	Germany	52074
2	Klinik und Poliklinik für Kinder und Jugendliche der Technischen Universität Dresden, Fetscherstr. 74	Dresden	Germany	01307
3	Klinik für Kinder und Jugendliche, Schwerpunkt Kinder-Endokrinologie und - Diabetologie, Loschgestr. 15	Erlangen	Germany	91054
4	Klinik für Päd. Hämatologie, Onkologie und Endokrinologie, Zentrum für Kinderheilkunde, der Universität Duisburg-Essen, Hufelandstrasse 55	Essen	Germany	45122
5	Kinder- und Jugendärztin, Pippinplatz 4	Gauting	Germany	82131
6	Endokrinologikum Hamburg, Lornsenstrasse 4 - 6	Hamburg	Germany	22767
7	Kinderarztpraxis, Brabeckstrasse 153	Hannover	Germany	30539
8	Universitätsklinik für Kinder- und Jugendliche, Abt. Kinderheilkunde, Im Neuenheimer Feld 430	Heidelberg	Germany	69120
9	Universitätsklinik für Kinder- und Jugendmedizin, Kirrberger Strasse	Homburg/Saar	Germany	66421
10	Klinik u. Poliklinik für Kinder und Jugendliche, Med. Einrichtungen der Universität Köln, Joseph-Stelzmann-Strasse 9	Köln	Germany	50931
11	Zentrum für Frauen und Kindermedizin, Liebigstrasse 20 a	Leipzig	Germany	04103
12	Klinik für Allgemeine Pädiatrie und Neonatologie, Otto-von-Guericke-Universität Magdeburg, Leipziger Straße 44	Magdeburg	Germany	39112
13	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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

, ,

ClinicalTrials.gov Identifier:

NCT00488124

Other Study ID Numbers:

NRA6280030

First Posted:

Jun 19, 2007

Last Update Posted:

Dec 14, 2020

Last Verified:

Dec 1, 2020

Keywords provided by , ,

short stature

Additional relevant MeSH terms:

Dwarfism

Study Results

No Results Posted as of Dec 14, 2020