A randomised dose comparison study of recombinant human growth hormone effects on metabolism markers in children with growth hormone (GH) deficiency

ISRCTN	ISRCTN44127724
DOI	https://doi.org/10.1186/ISRCTN44127724
Protocol serial number	N0220117222
Sponsor	Department of Health (UK)
Funders	Sheffield Childrens Hospital NHS Trust (UK), Serono

Submission date: 12/09/2003
Registration date: 12/09/2003
Last edited: 15/10/2014

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Nutritional, Metabolic, Endocrine

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Record updated in last year

Plain English summary of protocol

Not provided at time of registration

Contact information

Dr NP Wright
Scientific

Division of Child Health
Sheffield Children's Hospital
Western Bank
Sheffield
S10 2TH
United Kingdom

Phone	+44 (0)114 222 2000

Study information

Primary study design	Interventional
Study design	Randomised dose comparison study
Secondary study design	Randomised controlled trial
Scientific title
Study objectives	Is the response to growth hormone dose dependent and what are the best markers to evaluate the response?
Ethics approval(s)	Not provided at time of registration
Health condition(s) or problem(s) studied	Nutritional, Metabolic, Endocrine: Growth hormone deficiency
Intervention	Patients will attend a screening visit (combined with the usual visit to teach child and parent how to inject GH and familiarise them with the pen) for collection of informed consent (patients and parent/guardian). Demographic data, medical history, auxology, pubertal development and concomitant medication details will have been collected in outpatients. All these data are routinely collected as part of the normal clinical process. Randomisation to one of three dose regimes will then take place. At entry to the study biological samples will be collected - 10 to 12 ml of blood and 24 h urine collection. These will currently be an additional investigation. Further assessment of auxological data and pubertal staging will take place after 3 months. Repeat biological samples (10 to 12 ml of blood and 24 h urine collection) will be collected. Venesection routinely takes place after 3 months treatment for clinical reasons to facilitate monitoring of insulin-like growth factor (IGF-1).
Intervention type	Drug
Phase	Not Applicable
Drug / device / biological / vaccine name(s)	Recombinant human growth hormone
Primary outcome measure(s)	Measurements: Blood samples will be sent to central laboratories for analysis. Parameters to be analysed are as follows: Glucose, HbA1C, insulin, total cholesterol, triglycerides, high density lipoproteins (HDL) and low density lipoprotein (LDL) cholesterol; bone-specific isoenzymes: calcaemia, phosphoraemia, alkaline phosphatase; markers of bone formation and resorption: osteocalcin, N-telopeptide, C-telopeptide, total deoxypyridinoline and type 3 procollagen; insulin-like growth factor 1 (IGF-1), insulin-like growth factor binding protein 3 (IGF-BP3), ALS, IGF-BP1; dehydroepiandrosterone sulphate (DHEA-S), testosterone, antimullerian hormone (AMH) (boys only); free thyroxine (FT4), leptin; parathyroid hormone (PTH) and vitamin D (25OH-D). Evaluation of primary efficacy endpoint: This is an investigational study with a principal objective of identifying primary endpoints from a battery of biological markers for later use in a second study. Consequently this study does not have any pre-specified primary outcome measures.
Key secondary outcome measure(s)	Evaluation of secondary efficacy endpoints: For each of the biological markers, an appropriate parametric or non-parametric statistical analysis will be employed to investigate differences between dose groups at the 3-month assessment while adjusting for appropriate co-variates.
Completion date	30/09/2003

Eligibility

Participant type(s)	Patient
Age group	Child
Sex	All
Target sample size at registration	5
Key inclusion criteria	Recruitment and number of subjects: Maximum recruitment of five pre-pubertal newly diagnosed GH-deficient patients in whom a clinical decision is made that they would benefit from treatment with GH and who wish to take part in study (subject to inclusion/exclusion criteria in accordance with protocol).
Key exclusion criteria	Not provided at time of registration
Date of first enrolment	01/06/2002
Date of final enrolment	30/09/2003

Locations

Countries of recruitment

United Kingdom
England

Study participating centre

Division of Child Health

Sheffield
S10 2TH
United Kingdom

Results and Publications

Individual participant data (IPD) Intention to share	No
IPD sharing plan summary	Not provided at time of registration
IPD sharing plan