GHD+ISS in prepubertal children born at term or prematurely, AGA or SGA GHD and SGA children (phase IV study) ISS children (phase III study)
Conditions
Interventions
Trade Name: Norditropin SimpleXX (R)/ Nordiflex (R)
Product Name: Norditropin Simplex (R)/ Nordiflex (R)
Pharmaceutical Form: Powder and solvent for solution for injection
Sponsors
Göteborgs Universitet
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Children, independent of their GH-secretion, with height below –2.0 SDS, (according to now used reference in Sweden, (14)) growing parallel or less to their growth channel during at least 1 year (? 3 months) of observation (i.e. no ongoing spontaneous catch up growth). _ age at start of GH treatment 3- =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: disease interacting with growth or syndrome, other than Silver-Russel Syndrome or appropriately treated hypothyroidism. _ ongoing catch-up growth before start of GH-treatment; measurements should be performed by trained persons, with the same technique during time period of at least 1 year. Definition: for inclusion growth should be ‘channel parallel’ or deviating downwards, i.e. change in height SDS during the pre-treatment year below + 0.3 SDS _ signs of foetal alcohol syndrome in the retina _ pathological body proportions, def sitting height % more than 2 SDS above or below normal. _ incapable of following the study protocol _ bad compliance during 1st year GH treatment (def: more than 24 missed injections 1st year on treatment) _ puberty (B2 or testes ³ 5ml)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Primary objective: The hypothesis is that there will be a significant (p<0.05) difference in the proportion of children who have height close to the set goal: height SDS -0.5 after 3 years of GH treatment, when comparing the group treated with an individualized dose with the group of children treated with the fixed dose. ;Secondary Objective: To investigate whether the metabolic responses* correlate with the growth response, independently of the GH dose given in the group of children treated with individualised GH doses and to compare with levels in the group with fixed dose. (*IGF-I, IGFBP-3, fasting lipids, leptin, fasting insulin). To compare growth response to the changes in muscle, bone and fat mass measured with DEXA. To study the child’s quality of life/self esteem and cognitive performance, using psychological tests and questionnaires to the child, to the guardian(s) and teacher. To study effects of GH-treatment and genes found to be related to growth, i.e. point mutations and/or polymorphisms related to spontaneous growth and/or the response to GH treatment. A serum and DNA-sample from start of treatment will be stored, for later analyses in relation to observed effect of GH-treatment. Tertiary objective: To explore growth data in order to construct a prediction model for growth response based on pretreatment data.;Primary end point(s): Height after 3 years of treatment or onset of puberty | — |
Countries
Sweden
Outcome results
None listed