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

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

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.

Study Overview

• Status: Completed
• Conditions: Idiopathic Short Stature
• Intervention / Treatment: Drug: somatropin (Genotropin® treatment)

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 Type: Interventional
• Enrollment (Anticipated): 120
• Phase: Phase 2

Contacts and Locations

Study Locations

• Germany
  • Aachen, Germany, 52074
    • Kinderklinik, Universitätsklinikum der RWTH Aachen, Pauwelsstraße 30
  • Dresden, Germany, 01307
    • Klinik und Poliklinik für Kinder und Jugendliche der Technischen Universität Dresden, Fetscherstr. 74
  • Erlangen, Germany, 91054
    • Klinik für Kinder und Jugendliche, Schwerpunkt Kinder-Endokrinologie und - Diabetologie, Loschgestr. 15
  • Essen, Germany, 45122
    • Klinik für Päd. Hämatologie, Onkologie und Endokrinologie, Zentrum für Kinderheilkunde, der Universität Duisburg-Essen, Hufelandstrasse 55
  • Gauting, Germany, 82131
    • Kinder- und Jugendärztin, Pippinplatz 4
  • Hamburg, Germany, 22767
    • Endokrinologikum Hamburg, Lornsenstrasse 4 - 6
  • Hannover, Germany, 30539
    • Kinderarztpraxis, Brabeckstrasse 153
  • Heidelberg, Germany, 69120
    • Universitätsklinik für Kinder- und Jugendliche, Abt. Kinderheilkunde, Im Neuenheimer Feld 430
  • Homburg/Saar, Germany, 66421
    • Universitätsklinik für Kinder- und Jugendmedizin, Kirrberger Strasse
  • Köln, Germany, 50931
    • Klinik u. Poliklinik für Kinder und Jugendliche, Med. Einrichtungen der Universität Köln, Joseph-Stelzmann-Strasse 9
  • Leipzig, Germany, 04103
    • Zentrum für Frauen und Kindermedizin, Liebigstrasse 20 a
  • Magdeburg, Germany, 39112
    • Klinik für Allgemeine Pädiatrie und Neonatologie, Otto-von-Guericke-Universität Magdeburg, Leipziger Straße 44
  • Tübingen, Germany, 72076
    • Klinik für Kinderheilkunde und Jugendmedizin, Sektion Pädiatrische Endokrinologie, Hoppe-Seyler-Straße 1

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 years to 10 years (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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

Outcome Measure	Time Frame
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

Collaborators and Investigators

• Sponsor: University of Erlangen-Nürnberg Medical School
• Investigators: Study Chair: Helmuth-Günther Doerr, Prof. Dr., Friedrich-Alexander-Universität Erlangen-Nürnberg

Study record dates

Study Major Dates

• Study Start: November 1, 2005
• Primary Completion (ACTUAL): February 1, 2011
• Study Completion (ACTUAL): March 1, 2011

Study Registration Dates

• First Submitted: June 18, 2007
• First Submitted That Met QC Criteria: June 18, 2007
• First Posted (ESTIMATE): June 19, 2007

Study Record Updates

• Last Update Posted (ACTUAL): December 14, 2020
• Last Update Submitted That Met QC Criteria: December 10, 2020
• Last Verified: December 1, 2020

More Information

Terms related to this study

• Keywords: short stature
• Additional Relevant MeSH Terms: Endocrine System Diseases; Genetic Diseases, Inborn; Musculoskeletal Diseases; Bone Diseases; Bone Diseases, Developmental; Dwarfism
• Other Study ID Numbers: NRA6280030