Additional identifiers
EudraCT number
ClinicalTrials.gov number
Protocol/serial number
N0220117222
Study information
Scientific title
Acronym
Study hypothesis
Is the response to growth hormone dose dependent and what are the best markers to evaluate the response?
Ethics approval
Not provided at time of registration
Study design
Randomised dose comparison study
Primary study design
Interventional
Secondary study design
Randomised controlled trial
Trial setting
Not specified
Trial type
Treatment
Patient information sheet
Condition
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 names
Recombinant human growth hormone
Primary outcome measures
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.
Secondary outcome measures
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.
Overall trial start date
01/06/2002
Overall trial end date
30/09/2003
Reason abandoned
Eligibility
Participant 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).
Participant type
Patient
Age group
Child
Gender
Both
Target number of participants
5
Participant exclusion criteria
Not provided at time of registration
Recruitment start date
01/06/2002
Recruitment end date
30/09/2003
Locations
Countries of recruitment
United Kingdom
Trial participating centre
Division of Child Health
Sheffield
S10 2TH
United Kingdom
Sponsor information
Organisation
Department of Health (UK)
Sponsor details
Richmond House
79 Whitehall
London
SW1A 2NL
United Kingdom
Sponsor type
Government
Website
Funders
Funder type
Industry
Funder name
Sheffield Childrens Hospital NHS Trust (UK)
Alternative name(s)
Funding Body Type
Funding Body Subtype
Location
Funder name
Serono
Alternative name(s)
Funding Body Type
Funding Body Subtype
Location
Results and Publications
Publication and dissemination plan
Not provided at time of registration
Intention to publish date
Participant level data
Not provided at time of registration
Results - basic reporting
Publication summary