Infant, Small for Gestational AgePhase 3EGNNCT00627523
Genotropin Treatment In Very Young Children Born Small For Gestational Age
A Phase 3 study testing Genotropin (PN-180,307) Somatropin in people with Infant, Small for Gestational Age, run by Pfizer at 17 sites in 9 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 19 to 29 months
- Conditions the study is for
- Infant, Small for Gestational Age
- What is being tested?
- Genotropin (PN-180,307) Somatropin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 43 participants
- Where
- 17 sitesin 9 countries
- Study dates
- Feb 2008to Dec 2013
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 19 to 29 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
17 sites in 9 countries.
17 sites in 9 countries
Italy4 sites
Centro di Endocrinologia Pediatrica, Dipartimento di Pediatria
Catania, 95125
Policlinico Universitario, Istituto di Clinica Pediatrica
Messina, 98100
Dipartimento Materno Infantile, UO di Pediatria e Neonatologia
Milan, 20132
Dipartimento di Medicina Pediatrica, UO di Endocrinologia e Diabetologia
Roma, 00165
Czechia3 sites
Fakultni nemocnice Olomouc
Olomouc, 775 20
Fakultni nemocnice Ostrava, Detska klinika
Ostrava-Poruba, 708 52
Fakultni nemocnice v Motole
Prague, 150 06
Spain3 sites
Hospital Virgen Del Camino
Pamplona, Navarre, 31008
Consorci Hospitalari Parc Tauli
Sabadell, Barcelona, 08208
Hospital Miguel Servet
Zaragoza, Zaragoza, 50009
Belgium2 sites
Universitair Ziekenhuis Brussel/Pediatrie
Brussels, 1090
Universitair Ziekenhuis Antwerpen / Pediatrie
Edegem, 2650
France1 site
CHU Toulouse, Hôpital des Enfants, Service Endocrinologie
Toulouse, 31059
Germany1 site
Universitaetsklinikum Erlangen, Kinder- und Jugendklinik
Erlangen, 91054
About this study
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)
To demonstrate the effect on height and psychomotor development of Growth Hormone treatment in very young children starting at an age of 2 years.
- Conditions
- Infant, Small for Gestational Age
- Being tested
- Genotropin (PN-180,307) Somatropin, Control-no treatment
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Genotropin (PN-180,307) SomatropinDrug
Injectable Genotropin
Control-no treatmentDrug
Control-no treatment
| Study group | Role | What they receive |
|---|---|---|
| Active | Experimental | The active treatment arm Receives: Drug: Genotropin (PN-180,307) Somatropin |
| Control | Experimental | Control Receives: Drug: Control-no treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline and Month 24
Secondary outcomes (9)
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.
Measured: Baseline and Month 24
Change From Baseline in Height SDS at Month 12.
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.
Measured: Baseline and Month 12
Change From Baseline in Growth Velocity SDS at Month 12.
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Baseline, Months 3, 6, 12, 18, and 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2008 (estimated)
- Study startFeb 2008
- Primary completionDec 2013
- Study completionDec 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Infant, Small for Gestational AgeStudies of the same condition, open and under way.
- More trials run by PfizerThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 43 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00627523
- Sponsor's protocol ID
- A6281287
- Other IDs
- 2007-003949-32, SGA
- Acronym
- EGN
- Status last verified
- Oct 2014
- First submitted
- 22 Feb 2008
- Last updated
- 4 Nov 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
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.
PubMed 26501737
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT00627523
- Acronym / sponsor ID
- EGN / A6281287
- Conditions
- Infant, Small for Gestational Age
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 43 participants (actual)
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- Study start
- Feb 2008
- Primary completion
- Dec 2013 (actual)
- Study completion
- Dec 2013 (actual)
- First posted
- 3 Mar 2008 (estimated)
- Results
- Posted 18 Sep 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Active | Experimental | Drug: Genotropin (PN-180,307) SomatropinThe active treatment arm |
| Control | Experimental | Drug: Control-no treatmentControl |
Who may qualify
- Ages
- 19 to 29 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Genotropin (PN-180,307) SomatropinInjectable Genotropin | Drug | Active |
| Control-no treatmentControl-no treatment | Drug | Control |
Primary outcome
Change From Baseline in Height Standard Deviation Score (SDS) at Month 24.
Time frame: Baseline and 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.
9 secondary outcomes
Change From Baseline in Growth Velocity SDS at Month 24.
Time frame: Baseline and Month 24
Change From Baseline in Height SDS at Month 12.
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.
Time frame: Baseline and Month 12
Change From Baseline in Psychomotor Development Using the Psychomotor Development Index (PDI) of Bayley Scale at Month 12.
Time frame: Baseline and Month 12
Head Circumference SDS at Months 3, 6, 12, 18 and 24.
Time frame: Months 3, 6, 12, 18 and 24
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.
Change From Baseline in Body Weight at Months 3, 6, 12, 18, and 24.
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.
Time frame: Baseline, Months 3, 6, 12, 18, and 24
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Nov 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 26501737De 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.
- To obtain contact information for a study center near you, click here.Link provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Pfizer CT.gov Call Center study director
Pfizer
17 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Genotropin (PN-180,307) Somatropin in people with Infant, Small for Gestational Age, run by Pfizer at 17 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Nov 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in Italy, Czechia, Spain, Belgium, France and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 18 Sep 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in Height Standard Deviation Score (SDS) at Month 24. (time frame: Baseline and Month 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00627523, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Nov 2014.