Genotropin Treatment In Very Young Children Born Small For Gestational Age

Pfizer43 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Infant, Small for Gestational Age

Interventions

Genotropin (PN-180,307) SomatropinDRUG

Injectable Genotropin

Control-no treatmentDRUG

Control-no treatment

Eligibility

Sex: ALLMin age: 19 MonthsMax age: 29 Months

Medical Language ↔ Plain English

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.

Locations (17)

Universitair Ziekenhuis Brussel/Pediatrie

Brussels, Belgium

Universitair Ziekenhuis Antwerpen / Pediatrie

Edegem, Belgium

Outcomes

Primary Outcomes

Change From Baseline in Height Standard Deviation Score (SDS) at Month 24.

Height SDS was calculated at the relevant visit by means of the following formula: Height SDS = (participant height) - (normal height)/normal height standard deviation. Where participant height refers to the participant's height at the relevant visit, and normal height and the normal height standard deviation equals the population mean and standard deviation values for participants of a similar age and gender. The change from Baseline value for height SDS was calculated as the difference between the parameter values at a specific visit, and the Baseline parameter values. The scores were centred around zero. Negative score indicated a participant was smaller for their age/gender.

Time frame: Baseline and Month 24

Secondary Outcomes

Change From Baseline in Growth Velocity SDS at Month 24.

The growth velocity SDS was calculated at the relevant visit by means of the following formula: Growth velocity SDS = (participant growth velocity) - (normal growth velocity)/normal growth velocity standard deviation. Where, participant growth velocity refers to the participant's growth velocity at the relevant visit, and normal growth velocity and the normal growth velocity standard deviation equals the population mean and standard deviation values for participants of a similar age and gender. The change from Baseline value for growth velocity SDS was calculated as the difference between the parameter values at a specific visit, and the Baseline parameter values. A negative score indicated that a participant had slower growth for their age/gender.

Time frame: Baseline and Month 24

Change From Baseline in Height SDS at Month 12.

Data from ClinicalTrials.gov

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Fakultni nemocnice Olomouc

Olomouc, Czechia

Fakultni nemocnice Ostrava, Detska klinika

Ostrava-Poruba, Czechia

Fakultni nemocnice v Motole

Prague, Czechia

CHU Toulouse, Hôpital des Enfants, Service Endocrinologie

Toulouse, France

Universitaetsklinikum Erlangen, Kinder- und Jugendklinik

Erlangen, Germany

Centro di Endocrinologia Pediatrica, Dipartimento di Pediatria

Catania, Italy

Policlinico Universitario, Istituto di Clinica Pediatrica

Messina, Italy

Dipartimento Materno Infantile, UO di Pediatria e Neonatologia

Milan, Italy

...and 7 more locations

Time frame: Baseline and Month 12

Change From Baseline in Growth Velocity SDS at Month 12.

Time frame: Baseline and Month 12

Change From Baseline in Mental Development Using the Mental Development Index (MDI) of Bayley Scale at Month 12.

The Bayley Scale of Infant Development (BSID-II) measured the mental and psychomotor development and test behavior of participants from 1 to 42 months of age. The scale was used to describe the current developmental functioning of infants and to assist in diagnosis and treatment planning for infants with developmental delays or disabilities. The BSID-II provided the mental raw score which was used to calculate the MDI score. Possible MDI scores ranged from 50-150. A score of 69 and below indicates significantly delayed performance, 70 to 84 indicates mildly delayed performance, 85 to 114 indicates normal limits and 115 and above indicates accelerated performance.

Time frame: Baseline and Month 12

Change From Baseline in Psychomotor Development Using the Psychomotor Development Index (PDI) of Bayley Scale at Month 12.

BSID-II measured the mental and psychomotor development and test behavior of participants from 1 to 42 months of age. The scale was used to describe the current developmental functioning of infants and to assist in diagnosis and treatment planning for infants with developmental delays or disabilities. The BSID-II provided the psychomotor raw score which was used to calculate the PDI score. Possible PDI scores ranged from 50-150. A score of 69 and below indicates significantly delayed performance, 70 to 84 indicates mildly delayed performance, 85 to 114 indicates normal limits and 115 and above indicates accelerated performance.

Time frame: Baseline and Month 12

Head Circumference SDS at Months 3, 6, 12, 18 and 24.

Head circumference SDS was calculated by means of the following formula = (Participant head circumference)-(Normal head circumference)/ Normal head circumference standard deviation. Where participant head circumference refers to the participant's head circumference at the relevant visit, and normal head circumference and the normal head circumference standard deviation equals the population mean and standard deviation values for participants of a similar age and gender. A negative score indicated a participant had a smaller head circumference for their age/gender.

Time frame: Months 3, 6, 12, 18 and 24

Change From Baseline in Head Circumference SDS at Months 3, 6, 12, 18 and 24.

Head circumference SDS was calculated by means of the following formula = (Participant head circumference)-(Normal head circumference)/Normal head circumference standard deviation. Where participant head circumference refers to the participant's head circumference at the relevant visit, and normal head circumference and the normal head circumference standard deviation equals the population mean and standard deviation values for participants of a similar age and gender. A negative score indicated a participant had a smaller head circumference for their age/gender.

Time frame: Baseline, Months 3, 6, 12, 18 and 24.

Change From Baseline in Body Weight at Months 3, 6, 12, 18, and 24.

Body weight was measured at all the relevant visits. The change from Baseline in body weight was calculated as the difference between the parameter values at each visit, and the Baseline parameter values.

Time frame: Baseline, Months 3, 6, 12, 18, and 24

Change From Baseline in Body Mass Index (BMI) at Months 3, 6, 12, 18, and 24.

Body mass index was calculated for all visits by means of the following formula: BMI (kg/m2) = Weight (kg)/(Height\[m\])2. The change from Baseline BMI was calculated as the difference between the parameter values at each visit, and the Baseline parameter values.

Time frame: Baseline, Months 3, 6, 12, 18, and 24