Condition category
Nutritional, Metabolic, Endocrine
Date applied
12/09/2003
Date assigned
12/09/2003
Last edited
15/10/2014
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

Contact information

Type

Scientific

Primary contact

Dr NP Wright

ORCID ID

Contact details

Division of Child Health
Sheffield Children's Hospital
Western Bank
Sheffield
S10 2TH
United Kingdom
+44 (0)114 222 2000

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

http://www.doh.gov.uk

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

Publication citations

Additional files

Editorial Notes