Now Available for Public Comment: Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

Genotropin Treatment In Very Young Children Born Small For Gestational Age (EGN)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT00627523
First received: February 22, 2008
Last updated: October 27, 2014
Last verified: October 2014
  Purpose

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


Condition Intervention Phase
Infant, Small for Gestational Age
Drug: Genotropin (PN-180,307) Somatropin
Drug: Control-no treatment
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
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)

Resource links provided by NLM:


Further study details as provided by Pfizer:

Primary Outcome Measures:
  • Change From Baseline in Height Standard Deviation Score (SDS) at Month 24. [ Time Frame: Baseline and Month 24 ] [ Designated as safety issue: No ]
    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.


Secondary Outcome Measures:
  • Change From Baseline in Growth Velocity SDS at Month 24. [ Time Frame: Baseline and Month 24 ] [ Designated as safety issue: No ]
    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.

  • Change From Baseline in Height SDS at Month 12. [ Time Frame: Baseline and Month 12 ] [ Designated as safety issue: No ]
    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.

  • Change From Baseline in Growth Velocity SDS at Month 12. [ Time Frame: Baseline and Month 12 ] [ Designated as safety issue: No ]
    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.

  • Change From Baseline in Mental Development Using the Mental Development Index (MDI) of Bayley Scale at Month 12. [ Time Frame: Baseline and Month 12 ] [ Designated as safety issue: No ]
    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.

  • Change From Baseline in Psychomotor Development Using the Psychomotor Development Index (PDI) of Bayley Scale at Month 12. [ Time Frame: Baseline and Month 12 ] [ Designated as safety issue: No ]
    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.

  • Head Circumference SDS at Months 3, 6, 12, 18 and 24. [ Time Frame: Months 3, 6, 12, 18 and 24 ] [ Designated as safety issue: No ]
    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.

  • Change From Baseline in Head Circumference SDS at Months 3, 6, 12, 18 and 24. [ Time Frame: Baseline, Months 3, 6, 12, 18 and 24. ] [ Designated as safety issue: No ]
    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.

  • Change From Baseline in Body Weight at Months 3, 6, 12, 18, and 24. [ Time Frame: Baseline, Months 3, 6, 12, 18, and 24 ] [ Designated as safety issue: No ]
    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.

  • Change From Baseline in Body Mass Index (BMI) at Months 3, 6, 12, 18, and 24. [ Time Frame: Baseline, Months 3, 6, 12, 18, and 24 ] [ Designated as safety issue: No ]
    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.


Enrollment: 43
Study Start Date: February 2008
Study Completion Date: December 2013
Primary Completion Date: December 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Active
The active treatment arm
Drug: Genotropin (PN-180,307) Somatropin
Injectable Genotropin
Other Name: Genotropin (PN-180,307) Somatropin
Experimental: Control
Control
Drug: Control-no treatment
Control-no treatment

  Eligibility

Ages Eligible for Study:   19 Months to 29 Months
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00627523

Locations
Belgium
Universitair Ziekenhuis Brussel/Pediatrie
Brussel, Belgium, 1090
Universitair Ziekenhuis Antwerpen / Pediatrie
Edegem, Belgium, 2650
Czech Republic
Fakultni nemocnice Olomouc
Olomouc, Czech Republic, 775 20
Fakultni nemocnice Ostrava, Detska klinika
Ostrava-Poruba, Czech Republic, 708 52
Fakultni nemocnice v Motole
Praha 5, Czech Republic, 150 06
France
CHU Toulouse, Hôpital des Enfants, Service Endocrinologie
Toulouse Cedex 9, France, 31059
Germany
Universitaetsklinikum Erlangen, Kinder- und Jugendklinik
Erlangen, Germany, 91054
Italy
Centro di Endocrinologia Pediatrica, Dipartimento di Pediatria
Catania, Italy, 95125
Policlinico Universitario, Istituto di Clinica Pediatrica
Messina, Italy, 98100
Dipartimento Materno Infantile, UO di Pediatria e Neonatologia
Milano, Italy, 20132
Dipartimento di Medicina Pediatrica, UO di Endocrinologia e Diabetologia
Roma, Italy, 00165
Netherlands
Sophia Children's Hospital
Rotterdam, Netherlands, 3015 GJ
Spain
Consorci Hospitalari Parc Tauli
Sabadell, Barcelona, Spain, 08208
Hospital Virgen Del Camino
Pamplona, Navarra, Spain, 31008
Hospital Miguel Servet
Zaragoza, Spain, 50009
Sweden
Drottning Silvias Barn och Ungdomssjukhus
Göteborg, Sweden
Switzerland
Inselspital
CH-3010 Bern, Switzerland
Sponsors and Collaborators
Pfizer
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer
  More Information

Additional Information:
No publications provided

Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT00627523     History of Changes
Other Study ID Numbers: A6281287, 2007-003949-32, SGA
Study First Received: February 22, 2008
Results First Received: September 10, 2014
Last Updated: October 27, 2014
Health Authority: United States: Food and Drug Administration

ClinicalTrials.gov processed this record on November 20, 2014