A TWO-YEAR MULTI-CENTRE, RANDOMIZED TWO ARM STUDY OF GENOTROPIN TREATMENT IN VERY YOUNG CHILDREN BORN SMALL FOR GESTATIONAL AGE: EARLY GROWTH AND NEURODEVELOPMENT (EGN) - ND

• Conditions: Short Stature children born small for Gestational Age (SGA).; MedDRA version: 9.1Level: HLTClassification code 10018208Term: Gestational age and weight conditions

• Registration Number: EUCTR2007-003949-32-IT
• Lead Sponsor: PFIZER

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2008-03-17
• Last Update Posted: 2 June 2014

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 88

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.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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: Father?s height below 155 cm or mother?s 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).

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: · 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.;Secondary Objective: · 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.;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.