Clinical trial NCT00627523: Genotropin Treatment In Very Young Children Born Small For Gestational Age
Official title: A Two Year Multicentre, Randomized Two Arm Study Of Genotropin Treatment In Very Young Children Born Small For Gestational Age: Early Growth And Neurodevelopment(Egn)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Infant, Small for Gestational Age
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2008-03-03
Enrollment: 43
Sex: ALL
Minimum age (years): 19
Maximum age (years): 29
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: To demonstrate the effect on height and psychomotor development of Growth Hormone treatment in very young children starting at an age of 2 years.


Eligibility criteria: Inclusion Criteria:

Caucasian male or female subjects aged between 19-29 months at Screening Visit 1.

Born SGA (birth length and/or weight \<-2 SD for gestational age, using country-specific standards).

Height below -2.5 SD at screening (19-29 months of age). At least one measurement of length between 12 and 18 months of age. Normal karyotype in girls to exclude Turners syndrome.

Exclusion Criteria:

Severe Intra-Uterine Growth Retardation (IUGR) (birth length below - 4 SD for gestational age), if associated with dysmorphic features.

Severe prematurity (Gestational Age (GA) \<32 weeks of gestation). Ongoing catch-up growth (defined as growth velocity SDS at inclusion \>0) based on at least 4 months measurement interval).

Severe familial short stature defined as: Father's height below 155 cm or mother's height below 145 cm.

Defined neurological defects and/or severe neurodevelopmental delay.

Primary outcomes:
measure: Change From Baseline in Height Standard Deviation Score (SDS) at Month 24.
timeFrame: Baseline and Month 24
Secondary outcomes:
measure: Change From Baseline in Growth Velocity SDS at Month 24.
timeFrame: Baseline and Month 24
measure: Change From Baseline in Height SDS at Month 12.
timeFrame: Baseline and Month 12
measure: Change From Baseline in Growth Velocity SDS at Month 12.
timeFrame: Baseline and Month 12
measure: Change From Baseline in Mental Development Using the Mental Development Index (MDI) of Bayley Scale at Month 12.
timeFrame: Baseline and Month 12
measure: Change From Baseline in Psychomotor Development Using the Psychomotor Development Index (PDI) of Bayley Scale at Month 12.
timeFrame: Baseline and Month 12
measure: Head Circumference SDS at Months 3, 6, 12, 18 and 24.
timeFrame: Months 3, 6, 12, 18 and 24
measure: Change From Baseline in Head Circumference SDS at Months 3, 6, 12, 18 and 24.
timeFrame: Baseline, Months 3, 6, 12, 18 and 24.
measure: Change From Baseline in Body Weight at Months 3, 6, 12, 18, and 24.
timeFrame: Baseline, Months 3, 6, 12, 18, and 24
measure: Change From Baseline in Body Mass Index (BMI) at Months 3, 6, 12, 18, and 24.
timeFrame: Baseline, Months 3, 6, 12, 18, and 24
Other outcomes:
Locations:
city: Brussel
country: Belgium
facility: Universitair Ziekenhuis Brussel/Pediatrie
city: Edegem
country: Belgium
facility: Universitair Ziekenhuis Antwerpen / Pediatrie
city: Olomouc
country: Czech Republic
facility: Fakultni nemocnice Olomouc
city: Ostrava-Poruba
country: Czech Republic
facility: Fakultni nemocnice Ostrava, Detska klinika
city: Praha 5
country: Czech Republic
facility: Fakultni nemocnice v Motole
city: Toulouse Cedex 9
country: France
facility: CHU Toulouse, Hôpital des Enfants, Service Endocrinologie
city: Erlangen
country: Germany
facility: Universitaetsklinikum Erlangen, Kinder- und Jugendklinik
city: Catania
country: Italy
facility: Centro di Endocrinologia Pediatrica, Dipartimento di Pediatria
city: Messina
country: Italy
facility: Policlinico Universitario, Istituto di Clinica Pediatrica
city: Milano
country: Italy
facility: Dipartimento Materno Infantile, UO di Pediatria e Neonatologia
city: Roma
country: Italy
facility: Dipartimento di Medicina Pediatrica, UO di Endocrinologia e Diabetologia
city: Rotterdam
country: Netherlands
facility: Sophia Children's Hospital
city: Sabadell
country: Spain
facility: Consorci Hospitalari Parc Tauli
state: Barcelona
city: Pamplona
country: Spain
facility: Hospital Virgen Del Camino
state: Navarra
city: Zaragoza
country: Spain
facility: Hospital Miguel Servet
city: Göteborg
country: Sweden
facility: Drottning Silvias Barn och Ungdomssjukhus
city: CH-3010 Bern
country: Switzerland
facility: Inselspital
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References:
citation: De Schepper J, Vanderfaeillie J, Mullis PE, Rooman R, Robertson A, Dilleen M, Gomez R, Wollmann HA. A 2-year multicentre, open-label, randomized, controlled study of growth hormone (Genotropin(R)) treatment in very young children born small for gestational age: Early Growth and Neurodevelopment (EGN) Study. Clin Endocrinol (Oxf). 2016 Mar;84(3):353-60. doi: 10.1111/cen.12968. Epub 2015 Nov 25.