A study on the tissue responsiveness to short term exogenous growth hormone in children with idiopathic short stature in relation to their response to long-term treatment

ISRCTN	ISRCTN52337368
DOI	https://doi.org/10.1186/ISRCTN52337368
Secondary identifying numbers	NL342, NTR380

Submission date: 19/12/2005
Registration date: 19/12/2005
Last edited: 12/01/2021

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Signs and Symptoms

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Plain English summary of protocol

Not provided at time of registration

Contact information

Prof J.M. Wit
Scientific

Leiden University Medical Center
Department of Pediatrics
P.O. Box 9600
Amsterdam
2300 RC
Netherlands

Phone	+31 (0)71 5262824
Email	j.m.wit@lumc.nl

Study information

Study design	Randomised controlled trial
Primary study design	Interventional
Secondary study design	Randomised controlled trial
Study setting(s)	Other
Study type	Treatment
Scientific title	A study on the tissue responsiveness to short term exogenous growth hormone in children with idiopathic short stature in relation to their response to long-term treatment
Study acronym	Dose-response study
Study objectives	The change in biochemical parameters of bone and collagen metabolism during a short term Growth Hormone (GH) dose-response study predicts the long-term effect of GH on growth. Idiopathic short stature is partially explainable by an abnormal tissue responsiveness to GH and Insulin-like Growth Factor 1 (IGF-1). The hypotheses of this study are: 1. GH therapy in a dosage of 6 IU/mw/day administered before puberty increases height velocity, height in adolescence and final height. 2. GH administration affects puberty onset and its duration. 3. GH administration affects quality of life.
Ethics approval(s)	Not provided at time of registration
Health condition(s) or problem(s) studied	Idiopathic Short Stature (ISS)
Intervention	After randomisation, the control group did not receive treatment, and were followed yearly for growth and puberty assessment. The treatment group underwent two three month periods of GH administration (1.5 IU/m^2/day, 3.0 IU/m^2/day) with three month washout periods in between. Thereafter 6 IU/m^2/day was given until the beginning of puberty.
Intervention type	Drug
Pharmaceutical study type(s)
Phase	Not Specified
Drug / device / biological / vaccine name(s)	Growth Hormone (GH) and Insulin-like Growth Factor 1 (IGF-1).
Primary outcome measure	Height at stop of therapy (at onset of puberty) and final height.
Secondary outcome measures	1. Timing of onset of puberty 2. Duration of puberty 3. Relation between long-term growth response (dependent variable) and short-term growth response on various dosages and in vitro responsiveness of cultured skin fibroblasts to GH and IGF-1 4. Effect of GH therapy on quality of life
Overall study start date	01/01/1994
Completion date	01/01/2009

Eligibility

Participant type(s)	Patient
Age group	Child
Lower age limit	4 Years
Upper age limit	10 Years
Sex	Both
Target number of participants	40
Total final enrolment	40
Key inclusion criteria	1. Height Standard Deviation Score (SDS) less than -2 2. Prepubertal 3. Aged between four to eight in females, or four to ten in males 4. GH response to provocation tests more than 20 mU/l 5. Normal sitting height:height ratio 6. Normal screening blood tests and urinanalysis
Key exclusion criteria	Any systemic disease during childhood that limits the growth potential or may interfere with the evaluation of the effectiveness of therapy.
Date of first enrolment	01/01/1994
Date of final enrolment	01/01/2009

Locations

Countries of recruitment

Netherlands

Study participating centre

Leiden University Medical Center

Amsterdam
2300 RC
Netherlands

Sponsor information

Pfizer B.V. (The Netherlands) (Pfizer Inc, New York, USA)
Not defined

P.O. Box 37
Capelle a/d IJssel
2900 AA
Netherlands

Phone	+31 (0)10 4064200
Email	sigrid.van.den.broek@pfizer.com
"ROR"	https://ror.org/02bzf1224

Funders

Funder type

Research organisation

Netherlands Organization for Scientific Research (NWO)

No information available

Results and Publications

Intention to publish date
Individual participant data (IPD) Intention to share	No
IPD sharing plan summary	Not provided at time of registration
Publication and dissemination plan	Not provided at time of registration
IPD sharing plan

Study outputs

Output type	Details	Date created	Date added	Peer reviewed?	Patient-facing?
Results article	results	01/04/2010	12/01/2021	Yes	No

Editorial Notes

12/01/2021: The following changes have been made:
1. Publication reference added.
2. The final enrolment number has been added from the reference.
3. The NTR numbers have been added.