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Trial · Interventional

CompletedPhase 3Results postedRegistry data

Genotropin Treatment In Very Young Children Born Small For Gestational Age

Short name EGN

NCTNCT00627523 (opens ClinicalTrials.gov in a new tab)Other idA6281287Other id2007-003949-32Other idSGA
Open at ClinicalTrials.govAsk about this trial

Overview

Status
Completed
Phase
Phase 3
Enrollment
43 participants (actual)
Sponsor
Pfizer · Industry
Design
Randomized · Open label (no masking)
Countries
9 countries

Belgium, Czechia, France, Germany, Italy, Netherlands, Spain, Sweden, Switzerland

Start
Feb 2008
Primary completion
Dec 2013
Completion
Dec 2013
First posted
3 Mar 2008 (estimated)
Last update posted
4 Nov 2014 (estimated)
Conditions
Infant, Small for Gestational Age

Timeline

1 trial · 1 completed · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
2008200920102011201220132014
  1. Ph 3

    EGN

    NCT00627523

    Feb 2008 to Dec 2013

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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

Interventions

  • Genotropin (PN-180,307) SomatropinDrug
  • Control-no treatmentDrug

Arms2

  • Experimental

    Active

    The active treatment arm

  • Experimental

    Control

    Control

Primary outcomes1

Outcomes the sponsor listed as primary.

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

    Time frame · Baseline and Month 24

    Registry description

    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 outcomes(9)

  1. Change From Baseline in Growth Velocity SDS at Month 24.

    Time frame · Baseline and Month 24

    Registry description

    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.

  2. Change From Baseline in Height SDS at Month 12.

    Time frame · Baseline and Month 12

    Registry description

    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.

  3. Change From Baseline in Growth Velocity SDS at Month 12.

    Time frame · Baseline and Month 12

    Registry description

    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.

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

    Time frame · Baseline and Month 12

    Registry description

    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.

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

    Time frame · Baseline and Month 12

    Registry description

    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.

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

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

    Registry description

    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.

  7. 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.

    Registry description

    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.

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

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

    Registry description

    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.

  9. 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

    Registry description

    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.

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeLeast squares mean· Baseline and Month 24

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

GroupValue (SDS)Spread
Genotropin®1.630.13
Control0.430.13
Secondary and other outcome results(7)
Secondary outcomeLeast squares mean· Baseline and Month 24

Change From Baseline in Growth Velocity SDS at Month 24.

GroupValue (SDS)Spread
Genotropin®0.740.57
Control-0.030.57
Secondary outcomeLeast squares mean· Baseline and Month 12

Change From Baseline in Height SDS at Month 12.

GroupValue (SDS)Spread
Genotropin®1.030.12
Control0.140.12
Secondary outcomeLeast squares mean· Baseline and Month 12

Change From Baseline in Growth Velocity SDS at Month 12.

GroupValue (SDS)Spread
Genotropin®1.650.56
Control-1.590.56
Secondary outcomeLeast squares mean· Baseline and Month 12

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

GroupValue (Units on a scale)Spread
Genotropin®10.975.34
Control8.554.74
Secondary outcomeLeast squares mean· Baseline and Month 12

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

GroupValue (Units on a scale)Spread
Genotropin®4.043.04
Control8.552.84
Secondary outcomeMean· Months 3, 6, 12, 18 and 24

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

GroupValue (SDS)Spread
Genotropin®-0.931.217
Control-1.371.122
Secondary outcomeMean· Baseline, Months 3, 6, 12, 18 and 24.

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

GroupValue (SDS)Spread
Genotropin®0.270.977
Control0.360.693

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
Genotropin®Participants received Genotropin® at a dose of 0.035 mg/kg/d for 24 months. The dose was calculated based on the actual body weight, and the closest dosing step of the 5 mg pen used. The starting dose for the first 2 weeks was 1/3 of the calculated dose. After 2 weeks the dose was increased to 2/3 of the calculated dose. After 4 weeks the daily dose was the dose calculated on body weight at randomization.–6 / 2121 / 21
ControlThis group was the untreated control group and was not administered placebo.–2 / 2218 / 22

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Other registry references

References listed at the registry that are not in PepBank. They link to PubMed.

  • DerivedDe 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. PMID 26501737

Eligibility

Who could join, as posted.

Sex
All
Age
from 19 Months up to 29 Months
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
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.

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