E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Short Stature children born small for Gestational Age (SGA). |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | HLT |
E.1.2 | Classification code | 10018208 |
E.1.2 | Term | Gestational age and weight conditions |
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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 assess the effect of 24 months of treatment with GH therapy at a dose of 0,035 mg/kg/d on height in short SGA children starting treatment at 24-30 months of age, compared to untreated controls, in randomized patients. |
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E.2.2 | Secondary objectives of the trial |
· To compare overall psychomotor development between the two groups using the Bayley Scale of Infant Development, 2nd edition (BSID-II). . To determine the effect of GH treatment on body weight, body mass index (BMI) head growth and body composition (DXA). · To demonstrate safety of GH treatment in young SGA children. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Caucasian male or female subjects aged between 19-29 months at Screening Visit 1. 2. Signed and dated informed consent. 3. Born SGA (birth length and/or weight <-2 SD for gestational age, using country-specific standards). 4. Height below -2.5 SD at screening (19-29 months of age). 5. At least one measurement of length between 12 and 18 months of age. 6. Normal karyotype in girls to exclude Turners syndrome. 7. Screening laboratory values within normal limits, or abnormalities clinically insignificant in the judgement of the investigator. |
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E.4 | Principal exclusion criteria |
1. Severe Intra-Uterine Growth Retardation (IUGR) (birth length below - 4 SD for gestational age), if associated with dysmorphic features. 2. Severe prematurity (Gestational Age (GA) <32 weeks of gestation). 3. Ongoing catch-up growth (defined as growth velocity SDS at inclusion >0) based on at least 4 months measurement interval). 4. Severe familial short stature defined as: Fathers height below 155 cm or mothers height below 145 cm. 5. Defined neurological defects and/or severe neurodevelopmental delay. 6. Defined syndromes such as fetal alcohol syndrome. 7. Severe perinatal complications like asphyxia, sepsis, Necrotizing Enterocolitis (NEC), respiratory distress syndrome, if associated with long-term sequelae (like short bowel syndrome, Broncho Pulmonary Dysplasia (BPD), cerebral palsy etc). 8. Other specific reason for short stature (eg, osteochondrodysplasia). 9. Any severe, acute or chronic illness (neurological, respiratory, gastrointestinal (eg, Coeliac disease etc) which in the opinion of the investigator may interfere with the interpretation of safety or efficacy evaluations. 10. Will be unlikely to comply with the protocol eg, inability to return for follow-up visits, or is unlikely to complete the study. 11. Is participating in any other studies involving investigational or marketed products. 12. Is currently treated with systemic glucocorticoids or has been treated with glucocorticoids for a period of one week or longer in the past 6 months. Topical or inhaled corticosteroids are permitted. 13. Subjects receiving other hormone treatment are not eligible for this study (except stable thyroid hormone replacement). |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoints will be the change from baseline in height SDS and height velocity SDS, after 24 months of treatment. |
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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 | No |
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 | 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.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
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 | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions 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.8.9.1 | In the Member State concerned years | 4 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 0 |