Growth disturbance (current height SDS < -2.5 and parental adjusted height SDS < -1) in short children born small for gestational age (SGA), with a birth weight and/or length below -2 SD, who failed to show catch-up growth (HV SDS < 0 during the last year) by 4 years of age or later. MedDRA version: 9.1 Level: LLT Classification code 10041093 Term: Small for gestational age
Conditions
Sponsors
Pfizer Pharma GmbH
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Pre-pubertal boys between 6 and 10 years of age or girls between 6 and 9 years of age 2. Birth length- and/or birth weight-SDS adjusted to gestational age =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Severe SGA (birth weight or length < -4 SD) and clinically relevant dysmorphic features 2. Severe pre-maturity (GA < 32 weeks of gestation) 3. Severe perinatal complications like asphyxia, sepsis, necrotizing enterocolitis (NEC), respiratory distress syndrome, if associated with long-term sequelae (like short bowel syndrome, bronchopulmonary dysplasia (BPD), cerebral palsy etc) 4. Inability to perform one- or two leg jumps from a standing position 5. Prior GH treatment 6. Other endocrine diseases except for well substituted hypothyroidism (stable replacement therapy for at least 3 months prior to randomization) 7. Any severe acute or chronic diseases (neurological, respiratory, gastrointestinal etc) or medication that might influence linear growth, cognitive performance or insulin sensitivity (e.g. prednisolone for more than 7 days in doses above 10 mg per day or other per oral glucocorticosteroids in equivalent doses are not permitted throughout the trial. Treatment with topical or inhaled steroids is permitted). Individuals should not be under treatment of musculoskeletal diseases. Moreover, the administration of neuropharmacological and psychopharmacological drugs is not allowed (including methylphenidate, atomoxetin) 8. Known diabetes mellitus 9. Active malignancy/tumor or history of malignancy/tumor disease 10. Turner syndrome in girls 11. Other defined chromosomal aberrations or syndromes (e.g. fetal alcohol syndrome, osteochondrodysplasia), except Silver-Russell syndrome 12. Suspected non-compliance or impossibility to follow the treatment schedule, respectively (e.g. social implications) 13. Defined neurological defects, e.g. paresis, severe microcephaly 14. Severe non-corrected impaired vision 15. Ongoing psychotherapy 16. Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject inappropriate for entry into this study 17. Participation in any other study during active treatment phase 18. Any other contraindications and warnings referring to the SGA indication and referenced in the actual local SmPC version of Genotropin®
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Primary objective is to determine whether growth hormone therapy improves motor- performance and coordinative skills in pre-pubertal individuals with SGA as defined by efficiency of muscular function.;Secondary Objective: Secondary objectives are to determine positive effects of growth hormone on cognitive performance (especially attention, alertness and memory), motor performance and coordinative skills as defined by peak jump power, peak jump force and maximal jump velocity, increment in muscle strength (dynamometer), changes in body composition (skinfold thickness measurements), changes in height SDS and growth velocity SDS. Furthermore, it should be demonstrated that growth hormone therapy in SGA children is safe and no new or unexpected side effects compared to the classical indications are probable. ;Primary end point(s): • Changes in efficiency of muscular function (Emf) after six months (two-leg-jump; Leonardo jump plate)(hierarchic testing) | — |
Countries
Germany
Outcome results
None listed