ClinicalTrials.gov
ClinicalTrials.gov Menu

Neuromuscular Changes In Small For Gestational Age Children During Somatropin Therapy (SGA-POWER)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00625872
Recruitment Status : Terminated (The study terminated on 19-Jan-2011 due to insufficient recruitment of patients. No safety reasons contributed to the termination of the study.)
First Posted : February 28, 2008
Results First Posted : February 29, 2012
Last Update Posted : February 29, 2012
Sponsor:
Information provided by (Responsible Party):
Pfizer

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Growth Hormone Therapy
Infant, Small for Gestational Age
Intervention Drug: Somatropin
Enrollment 23
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description Somatropin 0.035 milligram/kilogram/day (mg/kg/day) was administered subcutaneously (s.c) according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals. No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Period Title: Overall Study
Started 12 11
Treated 12 10
Completed 8 5
Not Completed 4 6
Reason Not Completed
Withdrawal by Subject             1             0
Does not meet inclusion criteria             1             0
Terminated by sponsor             2             5
Randomized but not treated             0             1
Arm/Group Title Somatropin Control Arm Total
Hide Arm/Group Description Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals. No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months. Total of all reporting groups
Overall Number of Baseline Participants 12 11 23
Hide Baseline Analysis Population Description
[Not Specified]
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 12 participants 11 participants 23 participants
6.6  (1.0) 7.6  (1.4) 7.1  (1.3)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants 11 participants 23 participants
Female
3
  25.0%
8
  72.7%
11
  47.8%
Male
9
  75.0%
3
  27.3%
12
  52.2%
1.Primary Outcome
Title Change From Baseline in Peak Jump Power Standard Deviation Score (PJP-SDS; Two-leg-jump) in Full Analysis Set (FAS) Population at Month 6
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
FAS population included participants who received at least 1 dose of study medication and had at least one post baseline efficacy assessment. Number of participants analyzed (N) signifies participants evaluable for the measure.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 4 8
Least Squares Mean (Standard Error)
Unit of Measure: Watt/kilogram (W/kg)
-0.15  (1.01) 0.28  (0.51)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Somatropin, Control Arm
Comments Analysis of covariance (ANCOVA) method was used to calculate p-value with treatment, baseline value, study duration and site as covariates.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.7232
Comments The statistical test was 2-sided and performed at the 5 percent significance level.
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Least squares (LS) mean difference
Estimated Value -0.43
Confidence Interval (2-Sided) 95%
-3.93 to 3.08
Parameter Dispersion
Type: Standard Error of the mean
Value: 1.10
Estimation Comments [Not Specified]
2.Primary Outcome
Title Change From Baseline in Peak Jump Power Standard Deviation Score (PJP-SDS; Two-leg-jump) in Per Protocol (PP) Population at Month 6
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
PP population included participants who received the study medication for at least 22 weeks. Number of participants analyzed (N) signifies participants evaluable for the measure.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 2 6
Least Squares Mean (Standard Error)
Unit of Measure: W/kg
-1.02  (0.43) 0.59  (0.27)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Somatropin, Control Arm
Comments ANCOVA method was used to calculate p-value with treatment, baseline value, study duration and site as covariates.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1941
Comments The statistical test was 2-sided and performed at the 5 percent significance level.
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter LS Mean difference
Estimated Value -1.61
Confidence Interval (2-Sided) 95%
-8.04 to 4.82
Parameter Dispersion
Type: Standard Error of the mean
Value: 0.51
Estimation Comments [Not Specified]
3.Primary Outcome
Title Change From Baseline in Peak Jump Force Standard Deviation Score (PJF-SDS; Two-leg-jump) in Full Analysis Set (FAS) Population at Month 6
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
FAS population included participants who received at least 1 dose of study medication and had at least one post baseline efficacy assessment. Number of participants analyzed (N) signifies participants evaluable for the measure.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 4 8
Least Squares Mean (Standard Error)
Unit of Measure: Newtons
-0.70  (0.62) -0.55  (0.29)
4.Primary Outcome
Title Change From Baseline in Peak Jump Force Standard Deviation Score (PJF-SDS; Two-leg-jump) in Per Protocol (PP) Population at Month 6
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
PP population included participants who received the study medication for at least 22 weeks. Number of participants analyzed (N) signifies participants evaluable for the measure.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 2 6
Least Squares Mean (Standard Error)
Unit of Measure: Newtons
-0.83  (1.27) -0.85  (0.73)
5.Primary Outcome
Title Change From Baseline in Maximum Jump Velocity (Vmax; Two-leg-jump) in Full Analysis Set (FAS) Population at Month 6
Hide Description Vmax was measured by Leonardo Jumping Platform during two-leg jump.
Time Frame Baseline and Month 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
FAS population included participants who received at least 1 dose of study medication and had at least one post baseline efficacy assessment. Number of participants analyzed (N) signifies participants evaluable for the measure.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 10 11
Least Squares Mean (Standard Error)
Unit of Measure: Meter/second (m/s)
0.01  (0.08) 0.12  (0.09)
6.Primary Outcome
Title Change From Baseline in Maximum Jump Velocity (Vmax; Two-leg-jump) in Per Protocol (PP) Population at Month 6
Hide Description Vmax was measured by Leonardo Jumping Platform during two-leg jump.
Time Frame Baseline and Month 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
PP population included participants who received the study medication for at least 22 weeks. Number of participants analyzed (N) signifies participants evaluable for the measure.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 5 9
Least Squares Mean (Standard Error)
Unit of Measure: m/s
0.01  (0.05) 0.25  (0.06)
7.Secondary Outcome
Title Change From Baseline in Intellectual Performance of Children Using Kaufmann-Assessment Battery for Children (K-ABC) Test Global Scales at Month 6
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
FAS population included participants who received at least 1 dose of study medication and had at least one post baseline efficacy assessment.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 11 11
Mean (Standard Deviation)
Unit of Measure: Units on a scale
Baseline (Sequential Processing) 25.7  (8.35) 28.5  (8.33)
Change at Month 6 (Sequential Processing) 2.6  (5.82) -1.9  (4.15)
Baseline (Simultaneous Processing) 45.7  (7.20) 49.8  (12.74)
Change at Month 6 (Simultaneous Processing) -0.1  (4.53) 1.5  (4.30)
Baseline (Achievement) 300.7  (66.83) 341.8  (76.88)
Change at Month 6 (Achievement) 32.1  (48.79) 16.7  (27.52)
Baseline (Nonverbal) 46.2  (8.65) 49.8  (13.21)
Change at Month 6 (Nonverbal) -1.4  (3.78) 0.0  (5.35)
Baseline (Mental Processing Composite) 70.0  (12.38) 78.1  (19.95)
Change at Month 6 (Mental Processing Composite) 1.4  (6.39) -0.9  (7.28)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Somatropin, Control Arm
Comments For K-ABC Test (Sequential Processing): Kruskal-Wallis Analysis of Variance (ANOVA) model was used to calculate p-value.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0532
Comments The statistical test was 2-sided and performed at the 5 percent significance level.
Method Kruskal-Wallis
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Somatropin, Control Arm
Comments For K-ABC Test (Simultaneous Processing): Kruskal-Wallis ANOVA model was used to calculate p-value.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3383
Comments The statistical test was 2-sided and performed at the 5 percent significance level.
Method Kruskal-Wallis
Comments [Not Specified]
Show Statistical Analysis 3 Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Somatropin, Control Arm
Comments For K-ABC Test (Achievement): Kruskal-Wallis ANOVA model was used to calculate p-value.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6830
Comments The statistical test was 2-sided and performed at the 5 percent significance level.
Method Kruskal-Wallis
Comments [Not Specified]
Show Statistical Analysis 4 Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Somatropin, Control Arm
Comments For K-ABC Test (Non-Verbal): Kruskal-Wallis ANOVA model was used to calculate p-value.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.4935
Comments The statistical test was 2-sided and performed at the 5 percent significance level.
Method Kruskal-Wallis
Comments [Not Specified]
Show Statistical Analysis 5 Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Somatropin, Control Arm
Comments For K-ABC Test (Mental Processing Composite): Kruskal-Wallis ANOVA model was used to calculate p-value.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3458
Comments The statistical test was 2-sided and performed at the 5 percent significance level.
Method Kruskal-Wallis
Comments [Not Specified]
8.Secondary Outcome
Title Change From Baseline in Intellectual Performance of Children Using Kaufmann-Assessment Battery for Children (K-ABC) Test Global Scales at Months 12 and 18
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
9.Secondary Outcome
Title Change From Baseline in Intellectual Performance of Children Using Kinderversion Der Testbatterie Zur Aufmerksamkeitsprüfung für Kinder (KITAP) Test at Month 6
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
FAS population included participants who received at least 1 dose of study medication and had at least one post baseline efficacy assessment. Number of participants analyzed (N) signifies participants evaluable for the measure and 'n' signifies participants who received the study drug and evaluated at the time point for each group respectively.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 9 11
Mean (Standard Deviation)
Unit of Measure: Seconds
Baseline (Distractibility) 554.3  (201.08) 514.5  (127.41)
Change at 6 Month (Distractibility) (n= 7, 10) 5.6  (230.95) -11.4  (139.52)
Baseline (Alertness) 433.9  (140.74) 384.6  (124.94)
Change at 6 Month (Alertness) (n= 8, 10) -16.1  (80.85) 4.0  (107.42)
Baseline (Flexibility) 1350.0  (470.50) 1313.0  (477.19)
Change at 6 Month (Flexibility) (n= 8, 10) -192.0  (492.73) -211.0  (589.41)
Baseline (Go/No Go) 539.8  (106.73) 514.9  (91.95)
Change at 6 Month (Go/No Go) (n= 8, 9) 35.6  (113.38) -50.2  (69.90)
Baseline (Vigilance) 822.2  (200.65) 693.4  (118.01)
Change at 6 Month (Vigilance) (n= 7, 10) -146.0  (278.60) -31.2  (107.62)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Somatropin, Control Arm
Comments For KITAP Test (Distractibility): Kruskal-Wallis ANOVA model was used to calculate p-value.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6256
Comments The statistical test was 2-sided and performed at the 5 percent significance level.
Method Kruskal-Wallis
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Somatropin, Control Arm
Comments For KITAP Test (Alertness): Kruskal-Wallis ANOVA model was used to calculate p-value.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.8590
Comments The statistical test was 2-sided and performed at the 5 percent significance level.
Method Kruskal-Wallis
Comments [Not Specified]
Show Statistical Analysis 3 Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Somatropin, Control Arm
Comments For KITAP Test (Flexibility): Kruskal-Wallis ANOVA model was used to calculate p-value.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 1.0000
Comments The statistical test was 2-sided and performed at the 5 percent significance level.
Method Kruskal-Wallis
Comments [Not Specified]
Show Statistical Analysis 4 Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Somatropin, Control Arm
Comments For KITAP Test (Go/No Go): Kruskal-Wallis ANOVA model was used to calculate p-value.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1234
Comments The statistical test was 2-sided and performed at the 5 percent significance level.
Method Kruskal-Wallis
Comments [Not Specified]
Show Statistical Analysis 5 Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Somatropin, Control Arm
Comments For KITAP Test (Vigilance): Kruskal-Wallis ANOVA model was used to calculate p-value.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3291
Comments The statistical test was 2-sided and performed at the 5 percent significance level.
Method Kruskal-Wallis
Comments [Not Specified]
10.Secondary Outcome
Title Change From Baseline in Intellectual Performance of Children Using Kinderversion Der Testbatterie Zur Aufmerksamkeitsprüfung für Kinder (KITAP) Test at Months 12 and 18
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
11.Secondary Outcome
Title Change From Baseline in Intellectual Performance of Children Using Non-verbal Learning Test (NVLT) at Month 6
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
FAS population included participants who received at least 1 dose of study medication and had at least one post baseline efficacy assessment. Number of participants analyzed (N) signifies participants evaluable for the measure.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 9 11
Mean (Standard Deviation)
Unit of Measure: Units on a scale
Baseline (Instability Index) 0.3  (0.15) 0.2  (0.21)
Change at Month 6 (Instability Index) -0.1  (0.11) -0.1  (0.20)
Baseline (Working Time) 227.0  (44.27) 218.3  (76.89)
Change at Month 6 (Working Time) 10.6  (92.95) -11.1  (34.86)
Baseline [T-scores (1)] 39.9  (13.60) 43.9  (16.53)
Change at Month 6 [T-scores (1)] 4.4  (11.64) 6.7  (12.12)
Baseline [T-scores (2)] 41.6  (12.97) 46.8  (14.40)
Change at Month 6 [T-scores (2)] 5.0  (10.72) -0.6  (5.50)
Baseline [T-scores (3)] 45.9  (14.89) 48.1  (18.20)
Change at Month 6 [T-scores (3)] 1.5  (5.15) 8.3  (12.24)
Baseline [T-scores (4)] 39.3  (16.97) 46.4  (16.03)
Change at Month 6 [T-scores (4)] 5.6  (15.53) 1.4  (11.75)
Baseline [T-scores (5)] 43.3  (11.12) 43.6  (15.13)
Change at Month 6 [T-scores (5)] 2.6  (9.83) 7.7  (12.59)
Baseline [T-scores (6)] 44.9  (15.83) 52.9  (19.96)
Change at Month 6 [T-scores (6)] 0.9  (16.55) -7.4  (14.88)
Baseline [Age-corrected T-scores (1)] 33.4  (12.18) 38.6  (13.43)
Change at Month 6 [Age-corrected T-scores (1)] 6.0  (10.21) 5.2  (11.26)
Baseline [Age-corrected T-scores (2)] 51.3  (7.55) 54.5  (8.10)
Change at Month 6 [Age-corrected T-scores (2)] 3.1  (5.59) -0.1  (3.38)
Baseline [Age-corrected T-scores (3)] 40.0  (13.86) 41.1  (16.38)
Change at Month 6 [Age-corrected T-scores (3)] 0.6  (4.47) 7.8  (12.13)
Baseline [Age-corrected T-scores (4)] 37.4  (17.01) 45.0  (15.21)
Change at Month 6 [Age-corrected T-scores (4)] 6.3  (14.57) 4.1  (10.63)
Baseline [Age-corrected T-scores (5)] 35.0  (11.88) 37.3  (13.48)
Change at Month 6 [Age-corrected T-scores (5)] 3.3  (10.73) 6.9  (12.57)
Baseline [Age-corrected T-scores (6)] 50.0  (16.49) 55.5  (16.64)
Change at Month 6 [Age-corrected T-scores (6)] 1.5  (19.06) -4.1  (13.77)
12.Secondary Outcome
Title Change From Baseline in Intellectual Performance of Children Using Non-verbal Learning Test (NVLT) at Months 12 and 18
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
13.Secondary Outcome
Title Change From Baseline in Intellectual Performance of Children Using Child Behavior Checklist 4-18 Years (CBCL 4-18) at Months 6, 12 and 18
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
14.Secondary Outcome
Title Change From Baseline in Peak Jump Power Standard Deviation Score (PJP-SDS; One-leg-jump) at Months 6, 12 and 18
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
15.Secondary Outcome
Title Change From Baseline in Peak Jump Force Standard Deviation Score (PJF-SDS; One-leg-jump) at Months 6, 12 and 18
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
16.Secondary Outcome
Title Change From Baseline in Maximum Jump Velocity (Vmax; One-leg-jump) at Months 6, 12 and 18
Hide Description Vmax was measured by Leonardo Jumping Platform during one leg jump.
Time Frame Baseline, Month 6, Month 12 and Month 18
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
17.Secondary Outcome
Title Change From Baseline in Five-chair Rising Test- Peak Jump Power (PJP) at Month 6
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
FAS population included participants who received at least 1 dose of study medication and had at least one post baseline efficacy assessment. Number of participants analyzed (N) signifies participants evaluable for the measure.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 9 9
Mean (Standard Deviation)
Unit of Measure: kilowatt (kW)
Baseline 0.1  (0.07) 0.1  (0.05)
Change at Month 6 0.0  (0.07) 0.0  (0.05)
18.Secondary Outcome
Title Change From Baseline in Five-chair Rising Test-Peak Jump Power (PJP) at Months 12 and 18
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
19.Secondary Outcome
Title Change From Baseline in Five-chair Rising Test-Peak Jump Force (PJF) at Month 6
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
FAS population included participants who received at least 1 dose of study medication and had at least one post baseline efficacy assessment. Number of participants analyzed (N) signifies participants evaluable for the measure.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 9 9
Mean (Standard Deviation)
Unit of Measure: kilonewton (kN)
Baseline 0.4  (0.11) 0.4  (0.19)
Change at Month 6 -0.0  (0.23) 0.0  (0.20)
20.Secondary Outcome
Title Change From Baseline in Five-chair Rising Test-Peak Jump Force (PJF) at Months 12 and 18
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
21.Secondary Outcome
Title Change From Baseline in Five-chair Rising Test-Maximum Jump Velocity (Vmax) at Month 6
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
FAS population included participants who received at least 1 dose of study medication and had at least one post baseline efficacy assessment. Number of participants analyzed (N) signifies participants evaluable for the measure.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 9 9
Mean (Standard Deviation)
Unit of Measure: m/s
Baseline 0.7  (0.22) 0.6  (0.16)
Change at Month 6 0.2  (0.29) 0.0  (0.18)
22.Secondary Outcome
Title Change From Baseline in Five-chair Rising Test-Maximum Jump Velocity (Vmax) at Months 12 and 18
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
23.Secondary Outcome
Title Change From Baseline in Five-chair Rising Test (Time to Perform the Tasks) at Month 6
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
FAS population included participants who received at least 1 dose of study medication and had at least one post baseline efficacy assessment. Number of participants analyzed (N) signifies participants evaluable for the measure.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 9 9
Mean (Standard Deviation)
Unit of Measure: seconds
Baseline (Average rise time) 1.2  (0.90) 0.7  (0.31)
Change at Month 6 (Average rise time) -0.5  (1.15) 0.1  (0.27)
Baseline (Average time per test) 2.0  (0.70) 2.3  (0.60)
Change at Month 6 (Average time per test) -0.2  (0.54) -0.1  (0.87)
Baseline (Total time to perform 5 tests) 9.6  (3.91) 10.8  (2.24)
Change at Month 6 (Total time to perform 5 tests) 0.1  (3.04) 2.0  (8.48)
24.Secondary Outcome
Title Change From Baseline in Five-chair Rising Test (Time to Perform the Tasks) at Months 12 and 18
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
25.Secondary Outcome
Title Change From Baseline in One-chair Rising Test-Peak Jump Power (PJP) at Months 6, 12 and 18
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
26.Secondary Outcome
Title Change From Baseline in One-chair Rising Test-Peak Jump Force (PJF) at Months 6, 12 and 18
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
27.Secondary Outcome
Title Change From Baseline in One-chair Rising Test (Time to Perform the Tasks) at Months 6, 12 and 18
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
28.Secondary Outcome
Title Change From Baseline in Maximal Isometric Grip Force-Standard Deviation Score (MIGF-SDS) at Month 6
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
FAS population included participants who received at least 1 dose of study medication and had at least one post baseline efficacy assessment. Number of participants analyzed (N) signifies participants evaluable for the measure.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 9 11
Least Squares Mean (Standard Error)
Unit of Measure: kg
1.28  (1.69) 1.13  (1.64)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Somatropin, Control Arm
Comments ANCOVA method was used to calculate p-value with treatment, baseline value, study duration and site as covariates.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.9560
Comments The statistical test was 2-sided and performed at the 5 percent significance level.
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter LS Mean difference
Estimated Value 0.14
Confidence Interval (2-Sided) 95%
-5.71 to 6.00
Parameter Dispersion
Type: Standard Error of the mean
Value: 2.54
Estimation Comments [Not Specified]
29.Secondary Outcome
Title Change From Baseline in Maximal Isometric Grip Force-Standard Deviation Score (MIGF-SDS) at Months 12 and 18
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
30.Secondary Outcome
Title Mean Upper Arm Circumference
Hide Description [Not Specified]
Time Frame Baseline, Month 6, Month 12 and Month 18
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
31.Secondary Outcome
Title Mean Thigh Circumference
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
32.Secondary Outcome
Title Mean Calf Circumference
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
33.Secondary Outcome
Title Mean Height at Month 6
Hide Description Standing height was taken as a mean of 3 consecutive measurements using a wall mounted stadiometer.
Time Frame Month 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
FAS population included participants who received at least 1 dose of study medication and had at least one post baseline efficacy assessment.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 11 11
Mean (Standard Deviation)
Unit of Measure: cm
112.8  (5.13) 115.0  (7.93)
34.Secondary Outcome
Title Mean Height at Months 12 and 18
Hide Description Standing height was taken as a mean of 3 consecutive measurements using a wall mounted stadiometer.
Time Frame Month 12 and Month 18
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
35.Secondary Outcome
Title Mean Growth Velocity at Month 6
Hide Description Growth velocity measures the annual rate of increase in height.
Time Frame Month 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
FAS population included participants who received at least 1 dose of study medication and had at least one post baseline efficacy assessment.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 11 11
Mean (Standard Deviation)
Unit of Measure: cm/year
8.2  (1.93) 4.6  (1.44)
36.Secondary Outcome
Title Mean Growth Velocity at Months 12 and 18
Hide Description Growth velocity measures the annual rate of increase in height.
Time Frame Month 12 and Month 18
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
37.Secondary Outcome
Title Mean Height-Standard Deviation Score (SDS) at Month 6
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
FAS population included participants who received at least 1 dose of study medication and had at least one post baseline efficacy assessment.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 11 11
Mean (Standard Deviation)
Unit of Measure: cm
-3.1  (0.86) -3.7  (0.77)
38.Secondary Outcome
Title Mean Height-Standard Deviation Score (SDS) at Months 12 and 18
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
39.Secondary Outcome
Title Mean Growth Velocity-Standard Deviation Score (SDS) at Month 6
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
FAS population included participants who received at least 1 dose of study medication and had at least one post baseline efficacy assessment.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 11 11
Mean (Standard Deviation)
Unit of Measure: cm/year
3.4  (2.62) -1.1  (1.43)
40.Secondary Outcome
Title Mean Growth Velocity-Standard Deviation Score (SDS) at Months 12 and 18
Hide Description [Not Specified]
Time Frame Month 12 and Month 18
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
41.Secondary Outcome
Title Change From Baseline in Height-Standard Deviation Score (SDS) at Month 6
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
FAS population included participants who received at least 1 dose of study medication and had at least one post baseline efficacy assessment.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 11 11
Least Squares Mean (Standard Error)
Unit of Measure: cm
0.33  (0.21) -0.22  (0.25)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Somatropin, Control Arm
Comments ANCOVA method was used to calculate p-value with treatment, baseline value, study duration and site as covariates.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1107
Comments The statistical test was 2-sided and performed at the 5 percent significance level.
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter LS Mean difference
Estimated Value 0.55
Confidence Interval (2-Sided) 95%
-0.15 to 1.25
Parameter Dispersion
Type: Standard Error of the mean
Value: 0.31
Estimation Comments [Not Specified]
42.Secondary Outcome
Title Change From Baseline in Height-Standard Deviation Score (SDS) at Months 12 and 18
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
43.Secondary Outcome
Title Change From Baseline in Growth Velocity-Standard Deviation Score (SDS) at Month 6
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
FAS population included participants who received at least 1 dose of study medication and had at least one post baseline efficacy assessment. Number of participants analyzed (N) signifies participants evaluable for the measure.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 9 9
Least Squares Mean (Standard Error)
Unit of Measure: cm/year
3.89  (1.02) -0.77  (1.40)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Somatropin, Control Arm
Comments ANCOVA method was used to calculate p-value with treatment, baseline value, study duration and site as covariates.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0161
Comments The statistical test was 2-sided and performed at the 5 percent significance level.
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter LS Mean difference
Estimated Value 4.67
Confidence Interval (2-Sided) 95%
1.13 to 8.21
Parameter Dispersion
Type: Standard Error of the mean
Value: 1.54
Estimation Comments [Not Specified]
44.Secondary Outcome
Title Change From Baseline in Growth Velocity-Standard Deviation Score (SDS) at Months 12 and 18
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
45.Secondary Outcome
Title Sitting Height-Standard Deviation Score (SDS)
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
46.Secondary Outcome
Title Body Mass Index-Standard Deviation Score (BMI-SDS)
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
47.Secondary Outcome
Title Change From Baseline in Head Circumference at Months 6, 12 and 18
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
48.Secondary Outcome
Title Change From Baseline in Head Circumference-Standard Deviation Score (SDS) at Months 6, 12 and 18
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
49.Secondary Outcome
Title Change From Baseline in Skinfold Thickness-Standard Deviation Score (SDS) at Month 6
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
FAS population included participants who received at least 1 dose of study medication and had at least one post baseline efficacy assessment.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 11 11
Least Squares Mean (Standard Error)
Unit of Measure: Millimeter (mm)
Change at Month 6 (Triceps SDS) -0.41  (0.16) -0.06  (0.18)
Change at Month 6 (Subscapular SDS) -0.30  (0.10) -0.16  (0.13)
Change at Month 6 (Suprailiac SDS) -0.60  (0.07) -0.33  (0.12)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Somatropin, Control Arm
Comments Triceps SDS: ANCOVA method was used to calculate p-value with treatment, baseline value, study duration and site as covariates.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1870
Comments The statistical test was 2-sided and performed at the 5 percent significance level.
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter LS Mean difference
Estimated Value -0.35
Confidence Interval (2-Sided) 95%
-0.89 to 0.20
Parameter Dispersion
Type: Standard Error of the mean
Value: 0.25
Estimation Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Somatropin, Control Arm
Comments Subscapular SDS: ANCOVA method was used to calculate p-value with treatment, baseline value, study duration and site as covariates.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3494
Comments The statistical test was 2-sided and performed at the 5 percent significance level.
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter LS Mean difference
Estimated Value -0.15
Confidence Interval (2-Sided) 95%
-0.49 to 0.19
Parameter Dispersion
Type: Standard Error of the mean
Value: 0.15
Estimation Comments [Not Specified]
Show Statistical Analysis 3 Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Somatropin, Control Arm
Comments Suprailiac SDS: ANCOVA method was used to calculate p-value with treatment, baseline value, study duration and site as covariates.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0458
Comments The statistical test was 2-sided and performed at the 5 percent significance level.
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter LS Mean difference
Estimated Value -0.27
Confidence Interval (2-Sided) 95%
-0.54 to -0.01
Parameter Dispersion
Type: Standard Error of the mean
Value: 0.12
Estimation Comments [Not Specified]
50.Secondary Outcome
Title Change From Baseline in Skinfold Thickness-Standard Deviation Score (SDS) at Months 12 and 18
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
51.Other Pre-specified Outcome
Title Change From Baseline in Bone Density Using Peripheral Quantitative Computed Tomography (pqCT) at 6 or 12 or 18 Months
Hide Description Bone Mineral Density (BMD) was measured by pqCT. The Z-score measures the distance of the measured BMD value from the appropriate normal age matched population mean value in units of standard deviation of this population. More negative scores indicate less BMD compared to age matched population and more positive scores indicate higher BMD compared to age matched population.
Time Frame Baseline, Month 6, Month 12 and Month 18
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
52.Other Pre-specified Outcome
Title Change From Baseline in Bone Structure Using Peripheral Quantitative Computed Tomography (pqCT) at 6 or 12 or 18 Months
Hide Description Bone structure was measured by pqCT.Parameters included:total area,cortical area,marrow area,cortical thickness,cortical density of the radius,bone strength,cross-sectional muscle and fat area,total bone density,bone mineral count,trabecular BMD,bone cross-sectional area.Baseline and post-baseline SDS values transformed to age and sex specific z-score([Ln(test result/M)]/S);Ln=natural logarithm;M=age-/height- and sex-specific mean value;S=age-/height- and sex-specific coefficient of variation).Positive values are above the average for participant’s age and sex;negative values are below.
Time Frame Baseline, Month 6, Month 12 and Month 18
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
53.Other Pre-specified Outcome
Title Change From Baseline in Bone Stability Using Peripheral Quantitative Computed Tomography (pqCT) at 6 or 12 or 18 Months
Hide Description Bone stability was measured by pqCT. Baseline and post-baseline SDS values transformed to age and sex specific z-score (Ln(test result/M)]/S); Ln=natural logarithm; M=age- (or height-) and sex-specific mean value; S=age-(or height-) and sex-specific coefficient of variation) then change from baseline is calculated. Positive values are above the average for participant’s age and sex; negative values are below the average.
Time Frame Baseline, Month 6, Month 12 and Month 18
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not analyzed as study was prematurely terminated due to insufficient number of participants.
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description:
Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals.
No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame [Not Specified]
Adverse Event Reporting Description The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
 
Arm/Group Title Somatropin Control Arm
Hide Arm/Group Description Somatropin 0.035 mg/kg/day was administered s.c according to exact body weight specific calculation for 12 months. Dose adjustments were made at 6 month intervals. No treatment for initial 6 months in control group, after 6 months, somatropin 0.067 mg/kg/day administered s.c. according to exact body weight specific calculation for 12 months.
All-Cause Mortality
Somatropin Control Arm
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Somatropin Control Arm
Affected / at Risk (%) Affected / at Risk (%)
Total   0/12 (0.00%)   0/11 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Somatropin Control Arm
Affected / at Risk (%) Affected / at Risk (%)
Total   10/12 (83.33%)   7/11 (63.64%) 
Congenital, familial and genetic disorders     
Cryptorchism * 1  1/12 (8.33%)  0/11 (0.00%) 
Endocrine disorders     
Hypothyroidism * 1  1/12 (8.33%)  0/11 (0.00%) 
Eye disorders     
Conjunctivitis * 1  0/12 (0.00%)  1/11 (9.09%) 
Eyelid oedema * 1  1/12 (8.33%)  0/11 (0.00%) 
Gastrointestinal disorders     
Abdominal pain * 1  1/12 (8.33%)  0/11 (0.00%) 
Constipation * 1  0/12 (0.00%)  1/11 (9.09%) 
Diarrhoea * 1  1/12 (8.33%)  0/11 (0.00%) 
Infections and infestations     
Acute tonsillitis * 1  0/12 (0.00%)  1/11 (9.09%) 
Bronchitis * 1  0/12 (0.00%)  1/11 (9.09%) 
Gastroenteritis * 1  0/12 (0.00%)  1/11 (9.09%) 
Herpes zoster * 1  1/12 (8.33%)  0/11 (0.00%) 
Lice infestation * 1  1/12 (8.33%)  0/11 (0.00%) 
Otitis media * 1  1/12 (8.33%)  1/11 (9.09%) 
Respiratory tract infection * 1  2/12 (16.67%)  0/11 (0.00%) 
Rhinitis * 1  0/12 (0.00%)  1/11 (9.09%) 
Scarlet fever * 1  1/12 (8.33%)  0/11 (0.00%) 
Tonsillitis * 1  0/12 (0.00%)  1/11 (9.09%) 
Tooth infection * 1  1/12 (8.33%)  0/11 (0.00%) 
Upper respiratory tract infection * 1  1/12 (8.33%)  1/11 (9.09%) 
Urinary tract infection * 1  0/12 (0.00%)  1/11 (9.09%) 
Injury, poisoning and procedural complications     
Concussion * 1  0/12 (0.00%)  1/11 (9.09%) 
Musculoskeletal and connective tissue disorders     
Arthralgia * 1  0/12 (0.00%)  1/11 (9.09%) 
Lordosis * 1  1/12 (8.33%)  0/11 (0.00%) 
Pain in extremity * 1  1/12 (8.33%)  0/11 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Lipoma * 1  0/12 (0.00%)  1/11 (9.09%) 
Nervous system disorders     
Headache * 1  2/12 (16.67%)  2/11 (18.18%) 
Renal and urinary disorders     
Enuresis * 1  1/12 (8.33%)  0/11 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Cough * 1  1/12 (8.33%)  1/11 (9.09%) 
Skin and subcutaneous tissue disorders     
Eczema * 1  0/12 (0.00%)  1/11 (9.09%) 
Rash * 1  1/12 (8.33%)  0/11 (0.00%) 
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA v14.0
The study was prematurely discontinued, therefore not all data was powered.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
Results Point of Contact
Name/Title: Pfizer ClinicalTrials.gov Call Center
Organization: Pfizer, Inc.
Phone: 1-800-718-1021
Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT00625872     History of Changes
Other Study ID Numbers: A6281283
First Submitted: February 19, 2008
First Posted: February 28, 2008
Results First Submitted: January 27, 2012
Results First Posted: February 29, 2012
Last Update Posted: February 29, 2012