The planned study focuses on the effect of a one year Somatropin treatment (0.035 mg/kg/d or 0.067 mg/kg/d) in short children born SGA on neuromuscular function and cognitive performance.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
23
Patients will be randomized at baseline in a 1:1 ratio into treatment group or control group. After six months the control group will undergo GH therapy with a higher dose of 0.067 mg/kg/day, too. All patients are treated with growth hormone for 12 months.
Patients will be randomized at baseline in a 1:1 ratio into treatment group or control group. After six months the control group will undergo GH therapy with a higher dose of 0.067 mg/kg/day, too. All patients are treated with growth hormone for 12 months.
Pfizer Investigational Site
Berlin, Germany
Pfizer Investigational Site
Bonn, Germany
Pfizer Investigational Site
Cologne, Germany
Pfizer Investigational Site
Datteln, Germany
Change From Baseline in Peak Jump Power Standard Deviation Score (PJP-SDS; Two-leg-jump) in Full Analysis Set (FAS) Population at Month 6
Peak jump power (PJP) was defined as the peak of the calculated power (force multiplied by velocity). It was measured by Leonardo Jumping Platform during two-leg jump. The participant performs 3 jumps and the highest peak (PJP) of the 3 recordings was selected for further calculations. The SDS indicates how similar the participant was to the reference population.
Time frame: Baseline and Month 6
Change From Baseline in Peak Jump Power Standard Deviation Score (PJP-SDS; Two-leg-jump) in Per Protocol (PP) Population at Month 6
PJP was defined as the peak of the calculated power (force multiplied by velocity). It was measured by Leonardo Jumping Platform during two-leg jump. The participant performs 3 jumps and the highest peak (PJP) of the 3 recordings was selected for further calculations. The SDS indicates how similar the participant was to the reference population.
Time frame: Baseline and Month 6
Change From Baseline in Peak Jump Force Standard Deviation Score (PJF-SDS; Two-leg-jump) in Full Analysis Set (FAS) Population at Month 6
PJF was defined as the maximum of force of the ascending part of the jump which the participant performed as a counter-movement jump with freely moving arms and as high as possible with the head and chest. It was measured by Leonardo Jumping Platform during two-leg jump. The SDS indicates how similar the participant was to the reference population.
Time frame: Baseline and Month 6
Change From Baseline in Peak Jump Force Standard Deviation Score (PJF-SDS; Two-leg-jump) in Per Protocol (PP) Population at Month 6
PJF was defined as the maximum of force of the ascending part of the jump which the participant performed as a counter-movement jump with freely moving arms and as high as possible with the head and chest. It was measured by Leonardo Jumping Platform during two-leg jump. The SDS indicates how similar the participant was to the reference population.
Time frame: Baseline and Month 6
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Pfizer Investigational Site
Dresden, Germany
Pfizer Investigational Site
Erlangen, Germany
Pfizer Investigational Site
Essen, Germany
Pfizer Investigational Site
Heidelberg, Germany
Pfizer Investigational Site
Kiel, Germany
Pfizer Investigational Site
München, Germany
Change From Baseline in Maximum Jump Velocity (Vmax; Two-leg-jump) in Full Analysis Set (FAS) Population at Month 6
Vmax was measured by Leonardo Jumping Platform during two-leg jump.
Time frame: Baseline and Month 6
Change From Baseline in Maximum Jump Velocity (Vmax; Two-leg-jump) in Per Protocol (PP) Population at Month 6
Vmax was measured by Leonardo Jumping Platform during two-leg jump.
Time frame: Baseline and Month 6
Change From Baseline in Intellectual Performance of Children Using Kaufmann-Assessment Battery for Children (K-ABC) Test Global Scales at Month 6
K-ABC was assessed in children between 2.5-12.5 years. Comprised of 16 subtests; 10 mental processing (intelligence) and 6 achievement subtests. Achievement subtests: expressive vocabulary, faces\&places, arithmetic, riddles, reading/decoding, reading/comprehension. Sixteen subtests were weighted accordingly to form 5 global scales: sequential processing, simultaneous processing, achievement, non-verbal and mental processing composite. Scores were rated as upper extreme \[greater than (\>) 131\], above average (116-130), average (85-115), below average (70-84), lower extreme \[less than (\<) 69\].
Time frame: Baseline and Month 6
Change From Baseline in Intellectual Performance of Children Using Kaufmann-Assessment Battery for Children (K-ABC) Test Global Scales at Months 12 and 18
K-ABC was assessed in children between 2.5-12.5 years. Comprised of 16 subtests; 10 mental processing (intelligence) and 6 achievement subtests. Achievement subtests: expressive vocabulary, faces\&places, arithmetic, riddles, reading/decoding, reading/comprehension. Sixteen subtests were weighted accordingly to form 5 global scales: sequential processing, simultaneous processing, achievement, non-verbal and mental processing composite. Scores were rated as upper extreme \[greater than (\>) 131\], above average (116-130), average (85-115), below average (70-84), lower extreme \[less than (\<) 69\].
Time frame: Baseline, Month 12 and Month 18
Change From Baseline in Intellectual Performance of Children Using Kinderversion Der Testbatterie Zur Aufmerksamkeitsprüfung für Kinder (KITAP) Test at Month 6
The KITAP is a computer aided standardized neuro-cognitive development test which allows examination of a wide range of attention and executive functions such as shift of attention (Distractibility); simple reaction time (Alertness); "Sustained Attention", change of reaction (Flexibility); "Divided Attention", controlled reaction disposition (Go/No go) and "Vigilance". It has been designed appropriately for children between the age of 6 to 10 years to allow optimal motivation during testing and to increase validity of results.
Time frame: Baseline and Month 6
Change From Baseline in Intellectual Performance of Children Using Kinderversion Der Testbatterie Zur Aufmerksamkeitsprüfung für Kinder (KITAP) Test at Months 12 and 18
The KITAP is a computer aided standardized neuro-cognitive development test which allows examination of a wide range of attention and executive functions such as shift of attention (Distractibility); simple reaction time (Alertness); "Sustained Attention", change of reaction (Flexibility); "Divided Attention", controlled reaction disposition (Go/No go) and "Vigilance". It has been designed appropriately for children between the age of 6 to 10 years to allow optimal motivation during testing and to increase validity of results.
Time frame: Baseline, Month 12 and Month 18
Change From Baseline in Intellectual Performance of Children Using Non-verbal Learning Test (NVLT) at Month 6
NVLT was assessed for visual memorization that was difficult to verbalize. Test recorded instability index, T-scores\[sum of differences of correct {C} - incorrect {IC} "Yes" answers(1);sum of C "Yes" answers(2);sum of IC "Yes" answers(3);sum of differences of C-IC "Yes" answers with high associative items{ 87%-95%}(4);sum of differences of C-IC "Yes" answers with low associative items{ 54%-64%}(5); difference between difference values for high and low associative items(6)\].Scores were rated as below average(\<40), average(40-60), above average(\>60) and working time ranging between 9-12 minutes.
Time frame: Baseline and Month 6
Change From Baseline in Intellectual Performance of Children Using Non-verbal Learning Test (NVLT) at Months 12 and 18
NVLT was assessed for visual memorization that was difficult to verbalize. Test recorded instability index, T-scores\[sum of differences of correct {C} - incorrect {IC} "Yes" answers(1);sum of C "Yes" answers(2);sum of IC "Yes" answers(3);sum of differences of C-IC "Yes" answers with high associative items{ 87%-95%}(4);sum of differences of C-IC "Yes" answers with low associative items{ 54%-64%}(5); difference between difference values for high and low associative items(6)\].Scores were rated as below average(\<40), average(40-60), above average(\>60) and working time ranging between 9-12 minutes.
Time frame: Baseline, Month 12 and Month 18
Change From Baseline in Intellectual Performance of Children Using Child Behavior Checklist 4-18 Years (CBCL 4-18) at Months 6, 12 and 18
CBCL was standardized for children ages 4 to 18 years and measured child internalizing and externalizing behaviors and total problems. The 4-18 years' checklist contains 140 questions and responses were recorded on a Likert scale: 0 = Not True, 1 = Somewhat or Sometimes True, 2 = Very True or Often True. The range of possible values was 0-280 (0=good to 280=worst).
Time frame: Baseline, Month 6, Month 12 and Month 18
Change From Baseline in Peak Jump Power Standard Deviation Score (PJP-SDS; One-leg-jump) at Months 6, 12 and 18
PJP was defined as the peak of the calculated power (force multiplied by velocity). It was measured by Leonardo Jumping Platform during one leg jump. The participant performs 3 jumps and the highest peak (PJP) of the 3 recordings was selected for further calculations. The SDS indicates how similar the participant was to the reference population.
Time frame: Baseline, Month 6 , Month 12 and Month 18
Change From Baseline in Peak Jump Force Standard Deviation Score (PJF-SDS; One-leg-jump) at Months 6, 12 and 18
PJF was defined as the maximum of force of the ascending part of the jump which the participant performed as a counter-movement jump with freely moving arms and as high as possible with the head and chest. It was measured by Leonardo Jumping Platform during one leg jump. The SDS indicates how similar the participant was to the reference population.
Time frame: Baseline, Month 6, Month 12 and Month 18
Change From Baseline in Maximum Jump Velocity (Vmax; One-leg-jump) at Months 6, 12 and 18
Vmax was measured by Leonardo Jumping Platform during one leg jump.
Time frame: Baseline, Month 6, Month 12 and Month 18
Change From Baseline in Five-chair Rising Test- Peak Jump Power (PJP) at Month 6
The Chair rising test is a performance test (total power output) to measure neuromuscular function of complex movement in standing up. Test allows diagnostics of movement deficits using Leonardo jump plate. Five stand up test: five repetitions of rising from a chair on jump plate as quickly as possible with arms crossed over the chest (time to perform the tasks, maximal PJP, maximal velocity and maximal PJF). PJP is defined as the peak of the calculated power (force multiplied by velocity).
Time frame: Baseline and Month 6
Change From Baseline in Five-chair Rising Test-Peak Jump Power (PJP) at Months 12 and 18
The Chair rising test is a performance test (total power output) to measure neuromuscular function of complex movement in standing up. Test allows diagnostics of movement deficits using Leonardo jump plate. Five stand up test: five repetitions of rising from a chair on jump plate as quickly as possible with arms crossed over the chest (time to perform the tasks, maximal PJP, maximal velocity and maximal PJF). PJP is defined as the peak of the calculated power (force multiplied by velocity).
Time frame: Baseline, Month 12 and Month 18
Change From Baseline in Five-chair Rising Test-Peak Jump Force (PJF) at Month 6
The Chair rising test is a performance test (total power output) to measure neuromuscular function of complex movement in standing up. Test allows diagnostics of movement deficits using Leonardo jump plate. Five stand up test: five repetitions of rising from a chair on jump plate as quickly as possible with arms crossed over the chest (time to perform the tasks, maximal PJP, maximal velocity and maximal PJF). PJF is the maximum force of the ascending part of the jump which the participant performed as a counter-movement jump with freely moving arms as high as possible with the head and chest.
Time frame: Baseline and Month 6
Change From Baseline in Five-chair Rising Test-Peak Jump Force (PJF) at Months 12 and 18
The Chair rising test is a performance test (total power output) to measure neuromuscular function of complex movement in standing up. Test allows diagnostics of movement deficits using Leonardo jump plate. Five stand up test: five repetitions of rising from a chair on jump plate as quickly as possible with arms crossed over the chest (time to perform the tasks, maximal PJP, maximal velocity and maximal PJF). PJF is the maximum force of the ascending part of the jump which the participant performed as a counter-movement jump with freely moving arms as high as possible with the head and chest.
Time frame: Baseline, Month 12 and Month 18
Change From Baseline in Five-chair Rising Test-Maximum Jump Velocity (Vmax) at Month 6
The Chair rising test is a performance test (total power output) to measure neuromuscular function of complex movement in standing up. Test allows diagnostics of movement deficits using Leonardo jump plate. Five stand up test: five repetitions of rising from a chair on jump plate as quickly as possible with arms crossed over the chest (time to perform the tasks, maximal PJP, maximal velocity and maximal PJF). Vmax is defined as the maximum jump velocity.
Time frame: Baseline and Month 6
Change From Baseline in Five-chair Rising Test-Maximum Jump Velocity (Vmax) at Months 12 and 18
The Chair rising test is a performance test (total power output) to measure neuromuscular function of complex movement in standing up. Test allows diagnostics of movement deficits using Leonardo jump plate. Five stand up test: five repetitions of rising from a chair on jump plate as quickly as possible with arms crossed over the chest (time to perform the tasks, maximal PJP, maximal velocity and maximal PJF). Vmax is defined as the maximum jump velocity.
Time frame: Baseline, Month 12 and Month 18
Change From Baseline in Five-chair Rising Test (Time to Perform the Tasks) at Month 6
Chair rising test is performance test (total power output) to measure neuromuscular function of complex movement in standing up. Test allows diagnostics of movement deficits using Leonardo jump plate. Five stand up test: 5 repetitions of rising from a chair on jump plate as quickly as possible with arms crossed over chest (time to perform tasks, maximal PJP, maximal velocity and maximal PJF). Time to perform task includes: Average (avg) rise time which is avg time to perform 1 rise, avg time per test is the avg time to perform 1 test (rise and sitting down) and total time to perform 5 tests.
Time frame: Baseline and Month 6
Change From Baseline in Five-chair Rising Test (Time to Perform the Tasks) at Months 12 and 18
Chair rising test is performance test (total power output) to measure neuromuscular function of complex movement in standing up. Test allows diagnostics of movement deficits using Leonardo jump plate. Five stand up test: 5 repetitions of rising from a chair on jump plate as quickly as possible with arms crossed over chest (time to perform tasks, maximal PJP, maximal velocity and maximal PJF). Time to perform task includes: Average (avg) rise time which is avg time to perform 1 rise, avg time per test is the avg time to perform 1 test (rise and sitting down) and total time to perform 5 tests.
Time frame: Baseline, Month 12 and Month 18
Change From Baseline in One-chair Rising Test-Peak Jump Power (PJP) at Months 6, 12 and 18
The Chair rising test is a performance test (total power output) to measure neuromuscular function of complex movement to stand up. Test allows diagnostics of movement deficits using Leonardo jump plate. One stand up test: rising from a chair on the jump plate as quickly as possible with arms crossed over the chest (analysis of time, PJP, PJF and time of fastest rising). PJP is defined as the peak of the calculated power (force multiplied by velocity).
Time frame: Baseline, Month 6 , Month 12 and Month 18
Change From Baseline in One-chair Rising Test-Peak Jump Force (PJF) at Months 6, 12 and 18
The Chair rising test is a performance test (total power output) to measure neuromuscular function of complex movement to stand up. Test allows diagnostics of movement deficits using Leonardo jump plate. One stand up test: rising from a chair on the jump plate as quickly as possible with arms crossed over the chest (analysis of time, PJP, PJF and time of fastest rising). PJF is the maximum force of the ascending part of the jump which the participant performed as a counter-movement jump with freely moving arms as high as possible with the head and chest.
Time frame: Baseline, Month 6 , Month 12 and Month 18
Change From Baseline in One-chair Rising Test (Time to Perform the Tasks) at Months 6, 12 and 18
The Chair rising test is a performance test (total power output) to measure neuromuscular function of complex movement to stand up. Test allows diagnostics of movement deficits using Leonardo jump plate. One stand up test: rising from a chair on the jump plate as quickly as possible with arms crossed over the chest (analysis of time, PJP, PJF and time of fastest rising).
Time frame: Baseline, Month 6 , Month 12 and Month 18
Change From Baseline in Maximal Isometric Grip Force-Standard Deviation Score (MIGF-SDS) at Month 6
MIGF was assessed using standard adjustable Jamar dynamometer. MIGF (in Newtons) was calculated by multiplying the dynamometer reading (in kilograms) by a factor of 9.81. The SDS indicates how similar the participant was to the reference population.
Time frame: Baseline and Month 6
Change From Baseline in Maximal Isometric Grip Force-Standard Deviation Score (MIGF-SDS) at Months 12 and 18
MIGF was assessed using standard adjustable Jamar dynamometer. MIGF (in Newtons) was calculated by multiplying the dynamometer reading (in kilograms) by a factor of 9.81. The SDS indicates how similar the participant was to the reference population.
Time frame: Baseline, Month 12 and Month 18
Mean Upper Arm Circumference
Time frame: Baseline, Month 6, Month 12 and Month 18
Mean Thigh Circumference
Thigh measurements were taken as a mean of 3 consecutive measurements at upper thigh about an inch down from the crotch line.
Time frame: Baseline, Month 6, Month 12 and Month 18
Mean Calf Circumference
Calf measurements were taken as a mean of 3 consecutive measurements at largest part of calf muscle, usually about 4 inches down from below the knee.
Time frame: Baseline, Month 6, Month 12 and Month 18
Mean Height at Month 6
Standing height was taken as a mean of 3 consecutive measurements using a wall mounted stadiometer.
Time frame: Month 6
Mean Height at Months 12 and 18
Standing height was taken as a mean of 3 consecutive measurements using a wall mounted stadiometer.
Time frame: Month 12 and Month 18
Mean Growth Velocity at Month 6
Growth velocity measures the annual rate of increase in height.
Time frame: Month 6
Mean Growth Velocity at Months 12 and 18
Growth velocity measures the annual rate of increase in height.
Time frame: Month 12 and Month 18
Mean Height-Standard Deviation Score (SDS) at Month 6
Standing height was taken as a mean of 3 consecutive measurements using a wall mounted stadiometer. The SDS indicates how similar the participant was to the reference population.
Time frame: Month 6
Mean Height-Standard Deviation Score (SDS) at Months 12 and 18
Standing height was taken as a mean of 3 consecutive measurements using a wall mounted stadiometer. The SDS indicates how similar the participant was to the reference population.
Time frame: Month 12 and Month 18
Mean Growth Velocity-Standard Deviation Score (SDS) at Month 6
Growth velocity measures the annual rate of increase in height. The SDS indicates how similar the participant is to the reference population.
Time frame: Month 6
Mean Growth Velocity-Standard Deviation Score (SDS) at Months 12 and 18
Time frame: Month 12 and Month 18
Change From Baseline in Height-Standard Deviation Score (SDS) at Month 6
Standing height was taken as a mean of 3 consecutive measurements using a wall mounted stadiometer. The SDS indicates how similar the participant was to the reference population.
Time frame: Baseline and Month 6
Change From Baseline in Height-Standard Deviation Score (SDS) at Months 12 and 18
Standing height was taken as a mean of 3 consecutive measurements using a wall mounted stadiometer. The SDS indicates how similar the participant was to the reference population.
Time frame: Baseline, Month 12 and Month 18
Change From Baseline in Growth Velocity-Standard Deviation Score (SDS) at Month 6
Growth velocity measures the annual rate of increase in height. The SDS indicates how similar the participant is to the reference population.
Time frame: Baseline and Month 6
Change From Baseline in Growth Velocity-Standard Deviation Score (SDS) at Months 12 and 18
Growth velocity measures the annual rate of increase in height. The SDS indicates how similar the participant is to the reference population.
Time frame: Baseline, Month 12 and Month 18
Sitting Height-Standard Deviation Score (SDS)
Sitting height was measured using a stadiometer with a specialized chair. The SDS indicates how similar the participant was to the reference population.
Time frame: Baseline, Month 6, Month 12 and Month 18
Body Mass Index-Standard Deviation Score (BMI-SDS)
The BMI was used to measure body fat based on height and weight. It was calculated by body weight (kg) divided by the height (m) squared. The SDS indicates how similar the participant was to the reference population.
Time frame: Baseline, Month 6, Month 12 and Month 18
Change From Baseline in Head Circumference at Months 6, 12 and 18
The maximum head circumference (usually horizontal just above the eyebrow ridges), was measured from just above the glabella area to the area near the top of the occipital bone (opisthocranion).
Time frame: Baseline, Month 6, Month 12 and Month 18
Change From Baseline in Head Circumference-Standard Deviation Score (SDS) at Months 6, 12 and 18
The maximum head circumference (usually horizontal just above the eyebrow ridges), was measured from just above the glabella area to the area near the top of the occipital bone (opisthocranion). The SDS indicates how similar the participant was to the reference population.
Time frame: Baseline, Month 6, Month 12 and Month 18
Change From Baseline in Skinfold Thickness-Standard Deviation Score (SDS) at Month 6
Triceps, supra-iliac and subscapular skinfolds were measured on the right side of the body to the nearest 0.1 mm with a Holtain skinfold caliper. The measurement was performed at the left side of the participant. Triceps skinfold thickness was measured halfway down the left upper arm, while the arm was hanging relaxed at the participant's side. Suprascapular skinfold was measured laterally just below the angle of the left scapula. Suprailiac skinfold was measured just above the iliac crest in the middle-axillary line. SDS indicates how similar the participant was to the reference population.
Time frame: Baseline and Month 6
Change From Baseline in Skinfold Thickness-Standard Deviation Score (SDS) at Months 12 and 18
Triceps, supra-iliac and subscapular skinfolds were measured on the right side of the body to the nearest 0.1 mm with a Holtain skinfold caliper. The measurement was performed at the left side of the participant. Triceps skinfold thickness was measured halfway down the left upper arm, while the arm was hanging relaxed at the participant's side. Suprascapular skinfold was measured laterally just below the angle of the left scapula. Suprailiac skinfold was measured just above the iliac crest in the middle-axillary line. SDS indicates how similar the participant was to the reference population.
Time frame: Baseline, Month 12 and Month 18