E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Small for Gestational Age |
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E.1.1.1 | Medical condition in easily understood language |
Small for Gestational Age |
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E.1.1.2 | Therapeutic area | Body processes [G] - Metabolic Phenomena [G03] |
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The primary objective of this study was to evaluate the correlation between gene expression and catch-up growth (either spontaneous or drug-induced after one year of treatment) in SGA children. |
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E.2.2 | Secondary objectives of the trial |
A secondary objective was to evaluate the percentage of patients who were not treated, but who showed a spontaneous catch-up growth during two years of observation.
Safety objectives included the safety and tolerability of Saizen (recombinant human growth hormone, r-hGH) in SGA children. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- SGA at birth (defined as a length at birth equal or below the tenth percentile according to the Italian reference table of Bertini and Fabris)
- Age of 24 Months
- Caucasic
- Born at term (i.e. after the 37th completed week of gestation)
- Height equal or below (Group A) or up (Group B) the third percentile at the age of 24 months according to the Tanner reference table
- Sufficient GH secretion (more than 10 nanogram (ng)/milliliter (ml)) at least to one of the tests commonly used at that age (glucagon, Levo-dopa, arginine, clonidine, Growth Hormone Releasing Hormone (GHRH), GH integrated secretion)
- Normal level of Thyroid-stimulating hormone (THS), Free Triiodothyronine (FT3), Free Thyroxine (FT4), Insulin-like growth factor 1(IGF-1), insulin and haemoglobin A1c (HbA1c)
- Normal level of Immunoglobulin A (IgA)
- Children parents willing to comply with the protocol for the whole duration of the study
- A written Informed Consent before the baseline visit must be obtained from the parent(s) / legal guardian(s) |
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E.4 | Principal exclusion criteria |
- Congenital malformations (including Silver-Russel syndrome)
- Known abnormal karyotype, especially in girls
- Twins
- Severe psychomotor retardation
- Previous or ongoing treatment with anabolic steroids or r-hGH
- Treatments interfering with the immune system (including bacterial lysate)
- Severe chronic illnesses
- Autoimmune diseases |
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E.5 End points |
E.5.1 | Primary end point(s) |
Correlation Between Gene Expression Profiling and Catch-up Growth in Small for Gestational Age (SGA) Children |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
Percentage of Untreated Participants Who Showed a Spontaneous Catch-up Growth
Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs Leading to Study Drug Discontinuation |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
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E.8.2.4 | Number of treatment arms in the trial | 3 |
E.8.3 |
Will this trial be conducted at a single site globally?
| Yes |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 5 |
E.8.9.2 | In all countries concerned by the trial months | 5 |
E.8.9.2 | In all countries concerned by the trial days | 0 |