Idiopathic Short StaturePhase 2ISSNCT00488124
Growth Response in Short Children Suffering From a Disease With Growth Retardation and Treated With Somatropin
A Phase 2 study testing somatropin (Genotropin® treatment) in people with Idiopathic Short Stature, run by University of Erlangen-Nürnberg Medical School at 13 sites in Germany.
Trial 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. Read more about this study
- Who is it for?
- Ages 4 to 10 years
- Conditions the study is for
- Idiopathic Short Stature
- What is being tested?
- somatropin (Genotropin® treatment)
- Type of treatment
- Injection or infusion
- How many people
- 120 participants
- Where
- 13 sitesGermany
- Study dates
- Nov 2005to Mar 2011
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
13 sites in 1 country.
13 sites in 1 country
Germany13 sites
Kinderklinik, Universitätsklinikum der RWTH Aachen, Pauwelsstraße 30
Aachen, 52074
Klinik u. Poliklinik für Kinder und Jugendliche, Med. Einrichtungen der Universität Köln, Joseph-Stelzmann-Strasse 9
Cologne, 50931
Klinik und Poliklinik für Kinder und Jugendliche der Technischen Universität Dresden, Fetscherstr. 74
Dresden, 01307
Klinik für Kinder und Jugendliche, Schwerpunkt Kinder-Endokrinologie und - Diabetologie, Loschgestr. 15
Erlangen, 91054
Klinik für Päd. Hämatologie, Onkologie und Endokrinologie, Zentrum für Kinderheilkunde, der Universität Duisburg-Essen, Hufelandstrasse 55
Essen, 45122
Kinder- und Jugendärztin, Pippinplatz 4
Gauting, 82131
Endokrinologikum Hamburg, Lornsenstrasse 4 - 6
Hamburg, 22767
Kinderarztpraxis, Brabeckstrasse 153
Hanover, 30539
Universitätsklinik für Kinder- und Jugendliche, Abt. Kinderheilkunde, Im Neuenheimer Feld 430
Heidelberg, 69120
Universitätsklinik für Kinder- und Jugendmedizin, Kirrberger Strasse
Homburg/Saar, 66421
Zentrum für Frauen und Kindermedizin, Liebigstrasse 20 a
Leipzig, 04103
Klinik für Allgemeine Pädiatrie und Neonatologie, Otto-von-Guericke-Universität Magdeburg, Leipziger Straße 44
Magdeburg, 39112
Klinik für Kinderheilkunde und Jugendmedizin, Sektion Pädiatrische Endokrinologie, Hoppe-Seyler-Straße 1
Tübingen, 72076
About this study
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
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.
Read the 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.
- Conditions
- Idiopathic Short Stature
- Being tested
- somatropin (Genotropin® treatment)
- Keywords
- short stature
- Medical subject headings
- Dwarfism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
somatropin (Genotropin® treatment)Drug
0,035 mg /kg bodyweight of Somatropin per day given by subcutaneous injections through an injection device (GenotropinPEN)
What is being measured
The primary outcome is the main question the study is designed to answer.
To show an improvement of height, change in height (SDS) under GH treatment one year after visit 2 (start of GH therapy).
Measured: one and two years of observation
Secondary outcomes (1)
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)
Measured: one and two years of observation period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jun 2007 (estimated)
- Study startNov 2005
- Primary completionFeb 2011
- Study completionMar 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Idiopathic Short StatureStudies of the same condition, open and under way.
- More trials run by University of Erlangen-Nürnberg Medical SchoolThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 120 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Erlangen-Nürnberg Medical School
- Sponsor type
- University, hospital or other
- Investigators
- Helmuth-Günther Doerr, Prof. Dr., Study chair, Friedrich-Alexander-Universität Erlangen-Nürnberg
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00488124
- Sponsor's protocol ID
- NRA6280030
- Acronym
- ISS
- Status last verified
- Dec 2020
- First submitted
- 18 Jun 2007
- Last updated
- 14 Dec 2020
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00488124
- Acronym / sponsor ID
- ISS / NRA6280030
- Conditions
- Idiopathic Short Stature
- Keywords
- short stature
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- University of Erlangen-Nürnberg Medical SchoolUniversity, hospital or other
- Study start
- Nov 2005
- Primary completion
- Feb 2011 (actual)
- Study completion
- Mar 2011 (actual)
- First posted
- 19 Jun 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 4 to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| somatropin (Genotropin® treatment)0,035 mg /kg bodyweight of Somatropin per day given by subcutaneous injections through an injection device (GenotropinPEN) | Drug | – |
Primary outcome
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
1 secondary outcome
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Dec 2020.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Helmuth-Günther Doerr, Prof. Dr. study chair
Friedrich-Alexander-Universität Erlangen-Nürnberg
13 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing somatropin (Genotropin® treatment) in people with Idiopathic Short Stature, run by University of Erlangen-Nürnberg Medical School at 13 sites in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Dec 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Dec 2020.