E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Short stature in children born small for gestational age without catch up growth |
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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 |
To evaluate 1. Different growth hormone doses (low, high, and IGF-I titrated dose) on short and long term height velocity, final height and insulin sensitivity in short children (Height <-2.5SDS) born SGA (Birth weight <-2SDS).
2. Auxological, biochemical, and genetic predictors of growth response and insulin resistance in short SGA GH treated children.
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E.2.2 | Secondary objectives of the trial |
To evaluate long-term effects and safety of childhood GH treatment in SGA children as evaluated in adulthood (5 and 10 years after attainment of final height).
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
•Small for gestational age (BW < - 2SD below mean according to standard references) •Gestational age at birth more than 28 weeks •Short at 4 years of age (Height SDS <-2.5 according to standard references) •Short for parental height (HSDS > 1 SD below parental adjusted HSDS) •Age 4-8 years (girls) and 4-9 years (boys) •Pre-pubertal at start of treatment (testis volume < 4ml, breast stage 1) •Height records must be available for 6 months prior to inclusion in the study •Height velocity SDS < 0 during last 6 months (according to country specific references) •No previous growth hormone therapy.
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E.4 | Principal exclusion criteria |
•Known or suspected allergy to growth hormone •Previous participation in growth hormone trial •Severe mental retardation as judged by the investigation •Previous or active malignancy •Benign intracranial hypertension (present or past) •Diabetes mellitus •Growth retardation due to chronic diseases including hypertension, asthma (if on inhaled corticosteroids >800μg/day), syndromes (like fetal alcohol syndrome) and chromosomal anomalies (except Silver Russell syndrome). •Psychological problems likely to lead to significant non-compliance.
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary endpoints: Height gain ΔSDS (3 year) Secondary endpoints: Insulin resistance (IVGTT) IGF-related parameters (IGF-1, IGFBP-3, ALS, free IGF-1) Genetic polymorphisms in the population (IGF-1, VNTR, GH receptor) Changes in body composition and BMD (DEXA) Final height
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | Yes |
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 | Yes |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
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) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
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 | No |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
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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After the last patient assessment 10 years following the last subject completing the trial |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 14 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 14 |