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    Clinical Trial Results:
    Treatment With Recombinant Human Growth Hormone Genotonorm (Registered) in Children With Short Stature Secondary to a Long Term Corticoid Therapy. A Study of Efficacy and Safety.

    Summary
    EudraCT number
    		 2014-004104-30
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    23 Sep 2011

    Results information
    Results version number
    		 v2(current)
    This version publication date
    30 Mar 2016
    First version publication date
    09 Jul 2015
    Other versions
    		 v1
    Version creation reason
    • Correction of full data set
    Identified an error in the baseline section.

    Trial information

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    Trial identification
    Sponsor protocol code
    307-MET-9002-0009
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT00174187
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 Alias protocol number: A6281016
    Sponsors
    Sponsor organisation name
    Pfizer Inc.
    Sponsor organisation address
    235 E 42nd Street, New York, United States, NY 10017
    Public contact
    Pfizer ClinicalTrials.gov Call Center, Pfizer Inc., 001 800-718-1021, ClinicalTrials.gov_Inquiries@pfizer.com
    Scientific contact
    Pfizer ClinicalTrials.gov Call Center, Pfizer Inc., 001 800-718-1021, ClinicalTrials.gov_Inquiries@pfizer.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    22 Mar 2012
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    23 Sep 2011
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To assess the effect of a long-term treatment by Genotonorm on linear growth in children with short stature receiving steroid therapy.
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and in compliance with all International Conference on Harmonization (ICH) Good Clinical Practice (GCP) Guidelines. All the local regulatory requirements pertinent to safety of trial subjects were followed.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    21 Sep 2000
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    France: 30
    Worldwide total number of subjects
    30
    EEA total number of subjects
    30
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    17
    Adolescents (12-17 years)
    13
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 This study was conducted in France. The study start date was 21 September 2000 and study end date was 23 September 2011.

    Period 1
    Period 1 title
    Period 1 (up to 3 years)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)
    Arm description
    		 Subjects with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) subcutaneously for up to 3 years. After treatment for 3 years, subjects in this group were assigned to Somatropin- After Year 3 group.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Somatropin
    Investigational medicinal product code
    Other name
    Genotonorm, Genotropin
    Pharmaceutical forms
    Injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Subjects received somatropin 1.4 International Units per kilogram per week (IU/kg/week), equivalent to 0.46 milligram/kg/week (mg/kg/week), divided in 7 daily doses for up to 3 years.

    Arm title
    		 Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Arm description
    		 Subjects with nephrotic syndrome (NeS) received somatropin (Genotropin, Genotonorm) subcutaneously for up to 3 years. After treatment for 3 years, subjects in this group were assigned to Somatropin- After Year 3 group.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Somatropin
    Investigational medicinal product code
    Other name
    Genotonorm, Genotropin
    Pharmaceutical forms
    Injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Subjects received somatropin 1.4 IU/kg/week, equivalent to 0.46 mg/kg/week, divided in 7 daily doses for up to 3 years.

    Number of subjects in period 1
    		Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Started
    15
    15
    Completed
    15
    11
    Not completed
    0
    4
         Consent withdrawn by subject
    -
    1
         Non compliance
    -
    1
         Unspecified
    -
    1
         Serious adverse event
    -
    1
    Period 2
    Period 2 title
    Between Period 1 and Period 2
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Somatropin- After Year 3
    Arm description
    		 Included subjects with JIA or NeS who received somatropin (Genotropin, Genotonorm) subcutaneously for up to 3 years in this study.
    Arm type
    		 No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Number of subjects in period 2
    		Somatropin- After Year 3
    Started
    26
    Consented
    24
    Completed
    21
    Not completed
    5
         Did not consent to continue treatment
    2
         Consented, not assigned to treatment
    3
    Period 3
    Period 3 title
    Period 2 (after 3 years)
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Somatropin- After Year 3
    Arm description
    		 Subjects with JIA/NeS, who consented to receive treatment beyond 3 years, received somatropin (Genotropin, Genotonorm) 1.4 IU/kg/week subcutaneously until the additional study drug dose evaluation visit and thereafter received somatropin (Genotropin, Genotonorm) up to 50 microgram per kilogram per day (mcg/kg/day) subcutaneously until the final height (FH) was reached or up to Year 11. Final height was confirmed to have been achieved if the growth velocity was less than or equal to (<=) 1.5  centimeter (cm) per year during the preceding 12 months and bone age was greater than or equal to (>=) 17 years for boys and 15 years for girls.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Somatropin
    Investigational medicinal product code
    Other name
    Genotonorm, Genotropin
    Pharmaceutical forms
    Injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Subjects received somatropin 1.4 IU/kg/week equivalent to 0.46 mg/kg/week divided in 7 daily doses until the additional study drug dose evaluation visit and thereafter received somatropin up to 50 mcg/kg/day until the FH was reached or up to Year 11.

    Number of subjects in period 3
    		Somatropin- After Year 3
    Started
    21
    Completed
    13
    Not completed
    8
         Consent withdrawn by subject
    1
         Unspecified
    5
         Lost to follow-up
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)
    Reporting group description
    		 Subjects with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) subcutaneously for up to 3 years. After treatment for 3 years, subjects in this group were assigned to Somatropin- After Year 3 group.

    Reporting group title
    Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Reporting group description
    		 Subjects with nephrotic syndrome (NeS) received somatropin (Genotropin, Genotonorm) subcutaneously for up to 3 years. After treatment for 3 years, subjects in this group were assigned to Somatropin- After Year 3 group.

    Reporting group values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome) Total
    Number of subjects
    		 15 15 30
    Age categorical
    Units: Subjects
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 10.6 ( 3.4 ) 11.5 ( 3.8 ) -
    Gender, Male/Female
    Units: participants
        Female
    		 9 2 11
        Male
    		 6 13 19

    End points

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    End points reporting groups
    Reporting group title
    Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)
    Reporting group description
    		 Subjects with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) subcutaneously for up to 3 years. After treatment for 3 years, subjects in this group were assigned to Somatropin- After Year 3 group.

    Reporting group title
    Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Reporting group description
    		 Subjects with nephrotic syndrome (NeS) received somatropin (Genotropin, Genotonorm) subcutaneously for up to 3 years. After treatment for 3 years, subjects in this group were assigned to Somatropin- After Year 3 group.
    Reporting group title
    Somatropin- After Year 3
    Reporting group description
    		 Included subjects with JIA or NeS who received somatropin (Genotropin, Genotonorm) subcutaneously for up to 3 years in this study.
    Reporting group title
    Somatropin- After Year 3
    Reporting group description
    		 Subjects with JIA/NeS, who consented to receive treatment beyond 3 years, received somatropin (Genotropin, Genotonorm) 1.4 IU/kg/week subcutaneously until the additional study drug dose evaluation visit and thereafter received somatropin (Genotropin, Genotonorm) up to 50 microgram per kilogram per day (mcg/kg/day) subcutaneously until the final height (FH) was reached or up to Year 11. Final height was confirmed to have been achieved if the growth velocity was less than or equal to (<=) 1.5  centimeter (cm) per year during the preceding 12 months and bone age was greater than or equal to (>=) 17 years for boys and 15 years for girls.

    Primary: Change from Baseline in Height Standard Deviation Score According to Chronological Age (SDS/CA) at Year 3

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    End point title
    		 Change from Baseline in Height Standard Deviation Score According to Chronological Age (SDS/CA) at Year 3 [1]
    End point description
    Height was measured using a wall mounted device (example, Harpenden stadiometer). Height SDS/CA was obtained by measuring the height, subtracting chronological age- and gender-appropriate mean height and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) subject’s value was relative to the mean of the reference population. Full Analysis Set (FAS) up to Year 3: included all subjects who had at least 1 post-baseline height measurement and were treated with the study drug for at least 1 year.
    End point type
    Primary
    End point timeframe
    Baseline, Year 3
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Only descriptive data was planned to be reported for this endpoint. 
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    15
    15
    Units: Standard Deviation Score (SDS)
    median (inter-quartile range (Q1-Q3))
        Baseline
    -3.6 (-5.1 to -2.3)
    -2.5 (-2.8 to -2.2)
        Change at Year 3
    0.2 (-1.5 to 1.8)
    1.1 (0.8 to 1.5)
    No statistical analyses for this end point

    Primary: Change from Baseline in Height Standard Deviation Score According to Chronological Age (SDS/CA) at Final Height

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    End point title
    		 Change from Baseline in Height Standard Deviation Score According to Chronological Age (SDS/CA) at Final Height [2]
    End point description
    Height was measured using a wall mounted device (example, Harpenden stadiometer). Height SDS/CA was obtained by measuring the height, subtracting chronological age- and gender-appropriate mean height and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) subject’s value was relative to the mean of the reference population. FAS- after Year 3: included all subjects who received at least 1 dose of the study treatment and who had at least 1 post-baseline height measurement. Here, 'n' signifies those subjects who were evaluable for this measure at given time points.
    End point type
    Primary
    End point timeframe
    Baseline, when final height was reached (assessed up to Year 11)
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Only descriptive data was planned to be reported for this endpoint. 
    End point values
    		 Somatropin- After Year 3
    Number of subjects analysed
    21
    Units: SDS
    median (full range (min-max))
        Baseline (n = 21)
    -2.86 (-5.61 to -1)
        Change at Final Height (n = 4)
    0.71 (0.27 to 3.08)
    No statistical analyses for this end point

    Primary: Change from Baseline in Weight Standard Deviation Score (SDS) at Final Height

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    End point title
    		 Change from Baseline in Weight Standard Deviation Score (SDS) at Final Height [3]
    End point description
    Body weight was measured using a balance scale. Weight SDS was obtained by measuring the weight, subtracting age- and gender-appropriate mean weight and dividing the result by standard deviation of that mean (as obtained from age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) subject’s value was relative to the mean of the reference population. FAS- after Year 3: included all subjects who received at least one dose of the study treatment and who had at least one post-baseline height measurement. Here, 'n' signifies those subjects who were evaluable for this measure at given time point.
    End point type
    Primary
    End point timeframe
    Baseline, when final height was reached (assessed up to Year 11)
    Notes
    [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Only descriptive data was planned to be reported for this endpoint. 
    End point values
    		 Somatropin- After Year 3
    Number of subjects analysed
    21
    Units: SDS
    median (full range (min-max))
        Baseline (n = 21)
    -1.53 (-4.77 to 2.64)
        Change at Final Height (n = 4)
    -0.43 (-1.37 to 1.09)
    No statistical analyses for this end point

    Primary: Puberty Stage at Final Height

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    End point title
    		 Puberty Stage at Final Height [4]
    End point description
    Pubertal stage (graded from I to V for breast development and pubic hair development) according to the Tanner's method was collected. A low stage (Stage I) corresponds to a pre-pubertal stage and a high stage (Stage V) to an adult stage. FAS- after Year 3: included all subjects who received at least one dose of study treatment and who had at least one post-baseline height measurement. Here, 'n' signifies subjects who were evaluable for given components of puberty assessment.
    End point type
    Primary
    End point timeframe
    When final height was reached (assessed up to Year 11)
    Notes
    [4] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Only descriptive data was planned to be reported for this endpoint. 
    End point values
    		 Somatropin- After Year 3
    Number of subjects analysed
    4 [5]
    Units: subjects
        Pubic hair: Stage I (n=4)
    0
        Pubic hair: Stage II (n=4)
    0
        Pubic hair: Stage III (n=4)
    0
        Pubic hair: Stage IV (n=4)
    0
        Pubic hair: Stage V (n=4)
    4
        Breast development: Stage I (n=1)
    0
        Breast development: Stage II (n=1)
    0
        Breast development: Stage III (n=1)
    0
        Breast development: Stage IV (n=1)
    0
        Breast development: Stage V (n=1)
    1
    Notes
    [5] - Subjects who were evaluable for this measure.
    No statistical analyses for this end point

    Secondary: Bone Age

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    End point title
    		 Bone Age
    End point description
    Bone age was determined by the Greulich and Pyle method using left wrist and hand X-ray. FAS up to Year 3: included all subjects who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year. Here, 'n' signifies those subjects who were evaluable for this measure at given time points for each group, respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    15
    15
    Units: years
    median (inter-quartile range (Q1-Q3))
        Baseline (n = 14, 15)
    8.9 (7 to 10)
    10.5 (7 to 13)
        Year 1 (n = 14, 15)
    9.5 (7.8 to 11.5)
    11.5 (7 to 14)
        Year 2 (n = 15, 13)
    10 (8 to 13)
    14 (10 to 14.5)
        Year 3 (n = 15, 14)
    11.3 (8.8 to 14.5)
    14 (11.5 to 15)
    No statistical analyses for this end point

    Secondary: Lean Body Mass

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    End point title
    		 Lean Body Mass
    End point description
    Lean body mass, a measurement of body composition, was assessed by Dual Energy X-ray Absorptiometry (DEXA) scan. FAS up to Year 3: included all subjects who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year. Here, 'n' signifies those subjects who were evaluable for this measure at the given time point for each group respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    15
    15
    Units: kilogram (kg)
    median (inter-quartile range (Q1-Q3))
        Baseline (n = 15, 15)
    17.69 (14.96 to 19.96)
    23.56 (16.77 to 32.43)
        Year 1 (n = 15, 13)
    19.77 (16.62 to 24.58)
    31.16 (20.77 to 39.1)
        Year 2 (n = 14, 12)
    21.2 (18.79 to 25.13)
    34.14 (25.3 to 46.3)
        Year 3 (n = 14, 11)
    23.21 (20.88 to 25.55)
    36.38 (26.49 to 42.76)
    No statistical analyses for this end point

    Secondary: Annual Percent Change in Lean Body Mass at Year 1, 2 and 3

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    End point title
    		 Annual Percent Change in Lean Body Mass at Year 1, 2 and 3
    End point description
    Lean body mass, a measurement of body composition, was assessed by DEXA scan. Annual percent change: (Lean body mass at current year minus lean body mass at previous year) divided by lean body mass at previous year, multiplied by 100. FAS up to Year 3: included all subjects who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year. Here, 'n' signifies those subjects who were evaluable for this measure at the given time point for each group respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Year 1, 2, 3
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    15
    15
    Units: percent change
    median (inter-quartile range (Q1-Q3))
        Annual Change at Year 1 (n = 15, 13)
    18.95 (10.04 to 21.83)
    22.97 (17.54 to 28.8)
        Annual Change at Year 2 (n = 14, 11)
    7.75 (1.31 to 12.16)
    7.33 (4.76 to 18.91)
        Annual Change at Year 3 (n = 13, 9)
    9.83 (2.67 to 13.33)
    7.9 (1.88 to 15.08)
    No statistical analyses for this end point

    Secondary: Percent Change From Baseline in Lean Body Mass at Year 3

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    End point title
    		 Percent Change From Baseline in Lean Body Mass at Year 3
    End point description
    Lean body mass, a measurement of body composition, was assessed by DEXA scan. Percent change: (Lean body mass at Year 3 minus lean body mass at baseline) divided by lean body mass at baseline, multiplied by 100. FAS up to Year 3: included all subjects who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year.
    End point type
    Secondary
    End point timeframe
    Baseline, Year 3
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    14 [6]
    11 [7]
    Units: percent change
        median (inter-quartile range (Q1-Q3))
    36.82 (22.45 to 52.39)
    54.44 (43.06 to 62.3)
    Notes
    [6] - Subjects who were evaluable for this measure.
    [7] - Subjects who were evaluable for this measure.
    No statistical analyses for this end point

    Secondary: Lean Body Mass as Percentage of Total Weight

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    End point title
    		 Lean Body Mass as Percentage of Total Weight
    End point description
    Lean body mass, a measurement of body composition, was assessed by DEXA scan. FAS up to Year 3: included all subjects who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year. Here, 'n' signifies those subjects who were evaluable for this measure at the given time point for each group respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    15
    15
    Units: percentage of total weight
    median (inter-quartile range (Q1-Q3))
        Baseline (n = 15, 15)
    70.56 (58.25 to 80.1)
    60.69 (55.29 to 69.14)
        Year 1 (n = 15, 13)
    76.57 (64.16 to 83.89)
    65.97 (59.58 to 76.19)
        Year 2 (n = 14, 12)
    73.32 (63.85 to 80.96)
    69.95 (59.55 to 80.07)
        Year 3 (n = 14, 11)
    72.94 (66.79 to 78.21)
    67.24 (59.83 to 74.37)
    No statistical analyses for this end point

    Secondary: Lean Body Mass Standard Deviation Score According to Chronological Age (SDS/CA)

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    End point title
    		 Lean Body Mass Standard Deviation Score According to Chronological Age (SDS/CA)
    End point description
    Lean body mass was assessed by DEXA scan. Lean body mass SDS/CA was obtained by measuring lean body mass, subtracting the chronological age- and gender-appropriate mean lean body mass and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) subject’s value was relative to the mean of the reference population. FAS up to Year 3: included all subjects who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year. Here, 'n' signifies those subjects who were evaluable for this measure at the given time point for each group respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    15
    15
    Units: SDS
    median (inter-quartile range (Q1-Q3))
        Baseline (n = 15, 15)
    -1.5 (-1.81 to -0.94)
    -0.9 (-1.4 to -0.54)
        Year 1 (n = 15, 13)
    -1.13 (-1.64 to -0.67)
    -0.52 (-0.97 to 0.2)
        Year 2 (n = 14, 12)
    -1.26 (-1.94 to -0.78)
    -0.87 (-1.4 to -0.32)
        Year 3 (n = 13, 11)
    -1.75 (-1.96 to -0.94)
    -1.29 (-2.36 to -0.14)
    No statistical analyses for this end point

    Secondary: Fat Mass

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    End point title
    		 Fat Mass
    End point description
    Fat mass, a measurement of body composition, was assessed by DEXA scan. FAS up to Year 3: included all subjects who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year. Here, 'n' signifies those subjects who were evaluable for this measure at the given time point for each group respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    15
    15
    Units: kg
    median (inter-quartile range (Q1-Q3))
        Baseline (n = 15, 15)
    5.47 (4.04 to 11.34)
    13.62 (9.51 to 18.05)
        Year 1 (n = 15, 13)
    4.81 (2.64 to 10.21)
    10.99 (7.22 to 18.97)
        Year 2 (n = 14, 12)
    6.97 (3.12 to 11.48)
    16.7 (5.99 to 20.59)
        Year 3 (n = 14, 11)
    6.9 (4.94 to 11.33)
    15.19 (10.28 to 22.87)
    No statistical analyses for this end point

    Secondary: Annual Percent Change in Fat Mass at Year 1, 2 and 3

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    End point title
    		 Annual Percent Change in Fat Mass at Year 1, 2 and 3
    End point description
    Fat mass, a measurement of body composition, was assessed by DEXA scan. Annual percent change: (Fat mass at current year minus fat mass at previous year) divided by fat mass at previous year, multiplied by 100. FAS up to Year 3: included all subjects who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year. Here, 'n' signifies those subjects who were evaluable for this measure at the given time point for each group respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Year 1, 2, 3
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    15
    15
    Units: percent change
    median (inter-quartile range (Q1-Q3))
        Annual Change at Year 1 (n = 15, 13)
    -11.21 (-24.18 to 27.46)
    -3.71 (-19.44 to 15.73)
        Annual Change at Year 2 (n = 14, 11)
    29.52 (4.97 to 34.56)
    -1.05 (-20.82 to 79.15)
        Annual Change at Year 3 (n = 13, 9)
    26.61 (-4.54 to 52.9)
    19.37 (10.6 to 40.12)
    No statistical analyses for this end point

    Secondary: Percent Change from baseline in Fat Mass at Year 3

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    End point title
    		 Percent Change from baseline in Fat Mass at Year 3
    End point description
    Fat mass, a measurement of body composition, was assessed by DEXA scan. Percent change: (Fat mass at Year 3 minus fat mass at baseline) divided by fat mass at baseline, multiplied by 100. FAS up to Year 3: included all subjects who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year.
    End point type
    Secondary
    End point timeframe
    Baseline, Year 3
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    14 [8]
    11 [9]
    Units: percent change
        median (inter-quartile range (Q1-Q3))
    19.46 (3.83 to 73.79)
    37.56 (8.18 to 44.15)
    Notes
    [8] - Subjects who were evaluable for this measure.
    [9] - Subjects who were evaluable for this measure.
    No statistical analyses for this end point

    Secondary: Fat Mass as Percentage of Total Weight

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    End point title
    		 Fat Mass as Percentage of Total Weight
    End point description
    Fat mass, a measurement of body composition, was assessed by DEXA scan. FAS up to Year 3: included all subjects who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year. Here, 'n' signifies those subjects who were evaluable for this measure at the given time point for each group respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    15
    15
    Units: percentage of total weight
    median (inter-quartile range (Q1-Q3))
        Baseline (n = 15, 15)
    25 (13.7 to 35.8)
    33.8 (29.5 to 37.2)
        Year 1 (n = 15, 13)
    17.4 (12.3 to 31.4)
    30 (21.9 to 34.7)
        Year 2 (n = 14, 12)
    21.1 (14.2 to 32.4)
    27.1 (15 to 36.2)
        Year 3 (n = 14, 11)
    21.3 (17.1 to 28.6)
    26.4 (19.1 to 37.6)
    No statistical analyses for this end point

    Secondary: Fat Mass Standard Deviation Score According to Chronological Age (SDS/CA)

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    End point title
    		 Fat Mass Standard Deviation Score According to Chronological Age (SDS/CA)
    End point description
    Fat mass was assessed by DEXA scan. Fat mass SDS/CA was obtained by measuring fat mass, subtracting chronological age- and gender-appropriate mean fat mass and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) subject’s value was relative to the mean of the reference population. FAS up to Year 3: included all subjects who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year. Here, 'n' signifies those subjects who were evaluable for this measure at the given time point for each group respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    15
    15
    Units: SDS
    median (inter-quartile range (Q1-Q3))
        Baseline (n = 15, 15)
    1.51 (0.5 to 2.22)
    2.2 (1.95 to 2.39)
        Year 1 (n = 15, 13)
    0.8 (-0.33 to 1.64)
    1.98 (0.88 to 2.23)
        Year 2 (n = 14, 12)
    0.86 (-0.15 to 1.66)
    1.79 (0.55 to 2.33)
        Year 3 (n = 13, 11)
    1.27 (0.25 to 1.55)
    1.74 (0.9 to 2.41)
    No statistical analyses for this end point

    Secondary: Apparent Bone Mineral Density of Lumbar Spine (BMAD [LS])

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    End point title
    		 Apparent Bone Mineral Density of Lumbar Spine (BMAD [LS])
    End point description
    BMAD (LS) was assessed by DEXA scan. FAS up to Year 3: included all subjects who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year. Here, 'n' signifies those subjects who were evaluable for this measure at the given time point for each group respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    15
    15
    Units: gram per cubic centimeter (g/cm^3)
    median (inter-quartile range (Q1-Q3))
        Baseline (n = 15, 14)
    0.184 (0.168 to 0.242)
    0.223 (0.211 to 0.241)
        Year 1 (n = 14, 13)
    0.209 (0.163 to 0.243)
    0.237 (0.201 to 0.244)
        Year 2 (n = 15, 14)
    0.215 (0.181 to 0.248)
    0.249 (0.217 to 0.266)
        Year 3 (n = 13, 12)
    0.239 (0.205 to 0.254)
    0.242 (0.223 to 0.266)
    No statistical analyses for this end point

    Secondary: Apparent Bone Mineral Density Standard Deviation Score of Lumbar Spine According to Chronological Age (BMAD [LS] [SDS/CA])

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    End point title
    		 Apparent Bone Mineral Density Standard Deviation Score of Lumbar Spine According to Chronological Age (BMAD [LS] [SDS/CA])
    End point description
    BMAD (LS) was assessed by DEXA scan. BMAD (LS) (SDS/CA) was obtained by measuring the BMAD (LS), subtracting chronological age- and gender-appropriate mean BMAD (LS) and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) subject’s value was relative to the mean of the reference population. FAS up to Year 3: included all subjects who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year. Here, 'n' signifies those subjects who were evaluable for this measure at the given time point for each group respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    15
    15
    Units: SDS
    median (inter-quartile range (Q1-Q3))
        Baseline (n = 15, 14)
    -3.21 (-4.02 to -1.26)
    -1.53 (-1.94 to -1.35)
        Year 1 (n = 14, 13)
    -2.66 (-3.7 to -1.07)
    -1.51 (-2.32 to -0.95)
        Year 2 (n = 15, 14)
    -2.55 (-3.74 to -0.95)
    -1.42 (-1.86 to -0.72)
        Year 3 (n 12, 12)
    -1.77 (-2.43 to -1.21)
    -1.63 (-2.43 to -0.74)
    No statistical analyses for this end point

    Secondary: Apparent Bone Mineral Density Standard Deviation Score of Lumber Spine According to Tanner Puberty Stage (BMAD [LS] [SDS/Tanner Puberty Stage])

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    End point title
    		 Apparent Bone Mineral Density Standard Deviation Score of Lumber Spine According to Tanner Puberty Stage (BMAD [LS] [SDS/Tanner Puberty Stage])
    End point description
    BMAD (LS) was assessed by DEXA scan. BMAD (LS) (SDS/Tanner Puberty Stage) was obtained by measuring BMAD (LS), subtracting Tanner puberty stage- and gender-appropriate mean BMAD (LS) and dividing the result by standard deviation of that mean (as obtained from Tanner puberty stage- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) subject’s value was relative to the mean of the reference population. FAS up to Year 3: included all subjects who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year. Here, 'n' signifies those subjects who were evaluable for this measure at the given time point for each group respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    15
    15
    Units: SDS
    median (inter-quartile range (Q1-Q3))
        Baseline (n = 8, 8)
    -4.27 (-5.47 to -2.38)
    -3.09 (-3.36 to -2.31)
        Year 1 (n = 10, 7)
    -3.7 (-4.8 to -2.48)
    -3.23 (-4.64 to -1.74)
        Year 2 (n = 10, 9)
    -4.11 (-4.36 to -1.72)
    -2.6 (-3.04 to -2.17)
        Year 3 (n = 10, 8)
    -3.04 (-4.06 to -2.27)
    -2.96 (-3.37 to -2.76)
    No statistical analyses for this end point

    Secondary: Bone Mineral Density of Total Body (BMD [TB])

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    End point title
    		 Bone Mineral Density of Total Body (BMD [TB])
    End point description
    BMD (TB) was assessed by DEXA scan. FAS up to Year 3: included all subjects who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year. Here, 'n' signifies those subjects who were evaluable for this measure at the given time point for each group respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    15
    15
    Units: gram per square centimeter (g/cm^2)
    median (inter-quartile range (Q1-Q3))
        Baseline (n = 15, 15)
    0.78 (0.76 to 0.86)
    0.9 (0.81 to 1)
        Year 1 (n = 15, 14)
    0.8 (0.76 to 0.87)
    0.92 (0.85 to 1.02)
        Year 2 (n = 14, 11)
    0.81 (0.77 to 0.89)
    0.96 (0.86 to 1.09)
        Year 3 (n = 13, 11)
    0.84 (0.8 to 0.91)
    0.99 (0.89 to 1.08)
    No statistical analyses for this end point

    Secondary: Bone Mineral Density of Lumbar Spine (BMD [LS])

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    End point title
    		 Bone Mineral Density of Lumbar Spine (BMD [LS])
    End point description
    BMD (LS) was assessed by DEXA scan. FAS up to Year 3: included all subjects who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year. Here, 'n' signifies those subjects who were evaluable for this measure at the given time point for each group respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    15
    15
    Units: g/cm^2
    median (inter-quartile range (Q1-Q3))
        Baseline (n = 15, 15)
    0.55 (0.47 to 0.64)
    0.72 (0.67 to 0.85)
        Year 1 (n = 14, 14)
    0.61 (0.48 to 0.74)
    0.76 (0.66 to 0.91)
        Year 2 (n = 15, 14)
    0.64 (0.56 to 0.84)
    0.85 (0.77 to 1.04)
        Year 3 (n = 13, 12)
    0.74 (0.6 to 0.85)
    0.93 (0.79 to 1.07)
    No statistical analyses for this end point

    Secondary: Bone Mineral Content of Total Body (BMC [TB])

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    End point title
    		 Bone Mineral Content of Total Body (BMC [TB])
    End point description
    DEXA scan of BMC was used to evaluate potential bone effects of treatment. BMC is an estimate of the amount of mineral (such as calcium) in the bone. FAS up to Year 3: included all subjects who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year. Here, 'n' signifies those subjects who were evaluable for this measure at the given time point for each group respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    15
    15
    Units: gram
    median (inter-quartile range (Q1-Q3))
        Baseline (n = 15, 15)
    636.36 (498.47 to 939.71)
    1233.58 (772.41 to 1650.08)
        Year 1 (n = 15, 14)
    775.46 (578.97 to 1119.6)
    1576.15 (939.71 to 1739.38)
        Year 2 (n = 14, 11)
    855.7 (690.07 to 1059.67)
    2003.49 (1070.42 to 2243.61)
        Year 3 (n = 13, 11)
    1112.78 (807.3 to 1176.7)
    1997.74 (1296 to 2195.17)
    No statistical analyses for this end point

    Secondary: Annual Percent Change in Bone Mineral Content of Total Body (BMC [TB]) at Year 1, 2 and 3

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    End point title
    		 Annual Percent Change in Bone Mineral Content of Total Body (BMC [TB]) at Year 1, 2 and 3
    End point description
    BMC is an estimate of the amount of mineral (such as calcium) in the bone. Annual percent change: (BMC [TB] at current year minus BMC [TB] at previous year) divided by BMC [TB] at previous year, multiplied by 100. FAS up to Year 3: included all subjects who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year. Here, 'n' signifies those subjects who were evaluable for this measure at the given time point for each group respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Year 1, 2, 3
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    15
    15
    Units: percent change
    median (inter-quartile range (Q1-Q3))
        Year 1 (n = 15, 14)
    12.51 (7.66 to 21.86)
    20.16 (11.74 to 23.05)
        Year 2 (n = 14, 11)
    17.47 (12.2 to 19.93)
    13.91 (10.63 to 18.93)
        Year 3 (n = 12, 9)
    12.03 (4.5 to 14.54)
    7.39 (7.29 to 13.3)
    No statistical analyses for this end point

    Secondary: Percent Change from Baseline in Bone Mineral Content of Total Body (BMC [TB]) at Year 3

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    End point title
    		 Percent Change from Baseline in Bone Mineral Content of Total Body (BMC [TB]) at Year 3
    End point description
    BMC is an estimate of the amount of mineral (such as calcium) in the bone. Percent change: (BMC [TB] at Year 3 minus BMC [TB] at baseline) divided by BMC [TB] at baseline, multiplied by 100. FAS up to Year 3: included all subjects who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year.
    End point type
    Secondary
    End point timeframe
    Baseline, Year 3
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    13 [10]
    11 [11]
    Units: percent change
        median (inter-quartile range (Q1-Q3))
    46.63 (24.94 to 73.91)
    60.56 (36.49 to 67.79)
    Notes
    [10] - Subjects who were evaluable for this measure.
    [11] - Subjects who were evaluable for this measure.
    No statistical analyses for this end point

    Secondary: Bone Mineral Content Standard Deviation Score of Total Body According to Chronological Age (BMC [TB] [SDS/CA])

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    End point title
    		 Bone Mineral Content Standard Deviation Score of Total Body According to Chronological Age (BMC [TB] [SDS/CA])
    End point description
    BMC (TB) was measured by DEXA scan. BMC (TB) (SDS/CA) was obtained by measuring BMC (TB), subtracting the chronological age- and gender-appropriate mean BMC (TB) and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) subject’s value was relative to the mean of the reference population. FAS up to Year 3: included all subjects who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year. Here, 'n' signifies those subjects who were evaluable for this measure at the given time point for each group respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    15
    15
    Units: SDS
    median (inter-quartile range (Q1-Q3))
        Baseline (n = 15, 15)
    -1.95 (-2.6 to -1.03)
    -0.42 (-1.23 to 0.19)
        Year 1 (n = 15, 14)
    -1.85 (-2.43 to -0.73)
    -0.19 (-1.13 to 0.37)
        Year 2 (n = 14, 11)
    -1.66 (-2.27 to -1.14)
    -0.37 (-1.24 to 0.43)
        Year 3 (n = 12, 11)
    -1.65 (-2.22 to -0.35)
    -0.09 (-0.72 to 0.6)
    No statistical analyses for this end point

    Secondary: Bone Mineral Content Standard Deviation Score of Total Body According to Tanner Puberty Stage (BMC [TB] [SDS/Tanner Puberty Stage])

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    End point title
    		 Bone Mineral Content Standard Deviation Score of Total Body According to Tanner Puberty Stage (BMC [TB] [SDS/Tanner Puberty Stage])
    End point description
    BMC (TB) was measured by DEXA scan. BMC (TB) (SDS/Tanner Puberty Stage) was obtained by measuring BMC (TB), subtracting the Tanner puberty stage- and gender-appropriate mean BMC (TB) and dividing the result by standard deviation of that mean (as obtained from Tanner puberty stage- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) subject’s value was relative to the mean of the reference population. FAS up to Year 3: included all subjects who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year. Here, 'n' signifies those subjects who were evaluable for this measure at the given time point for each group respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    15
    15
    Units: SDS
    median (inter-quartile range (Q1-Q3))
        Baseline (n = 8, 8)
    -2.19 (-2.47 to -0.96)
    1.41 (-0.39 to 2.46)
        Year 1 (n = 10, 8)
    -1.56 (-2.24 to -0.92)
    -0.52 (-1.53 to 1.65)
        Year 2 (n = 9, 7)
    -2.38 (-2.72 to -1.14)
    -0.47 (-1.53 to 0.37)
        Year 3 (n = 9, 7)
    -2.38 (-2.56 to -1.28)
    -0.17 (-1.22 to 0.83)
    No statistical analyses for this end point

    Other pre-specified: Growth Velocity (GV)

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    End point title
    		 Growth Velocity (GV)
    End point description
    Growth velocity measures the annual rate of increase in height. FAS up to Year 3: included all subjects who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year. Here, 'n' signifies those subjects who were evaluable for this measure at the given time point for each group respectively.
    End point type
    Other pre-specified
    End point timeframe
    Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    15
    15
    Units: cm/year
    median (inter-quartile range (Q1-Q3))
        Baseline (n = 11, 14)
    2.9 (1.1 to 4.8)
    3.8 (2.7 to 4.4)
        Year 1 (n = 14, 15)
    6.5 (3.5 to 8.6)
    8.3 (5.4 to 8.7)
        Year 2 (n = 14, 15)
    5.3 (1.8 to 6.9)
    7 (4.1 to 9.2)
        Year 3 (n = 14, 14)
    4.5 (1.9 to 6.8)
    5.9 (2.3 to 7.2)
    No statistical analyses for this end point

    Other pre-specified: Growth Velocity Standard Deviation Score According to Chronological Age (GV [SDS/CA])

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    End point title
    		 Growth Velocity Standard Deviation Score According to Chronological Age (GV [SDS/CA])
    End point description
    GV measures the annual rate of increase in height. GV (SDS/CA) was obtained by measuring GV, subtracting the chronological age- and gender-appropriate mean GV and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) subject’s value was relative to the mean of the reference population. FAS up to Year 3: included all subjects who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year. Here, 'n' signifies those subjects who were evaluable for this measure at the given time point for each group respectively.
    End point type
    Other pre-specified
    End point timeframe
    Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    15
    15
    Units: SDS
    median (inter-quartile range (Q1-Q3))
        Baseline (n = 10, 14)
    -1.7 (-2.8 to -1.3)
    -2.2 (-2.9 to -1.3)
        Year 1 (n = 14, 15)
    1 (-1.5 to 3.9)
    1.3 (-0.7 to 1.9)
        Year 2 (n = 14, 15)
    0.1 (-1.9 to 3.7)
    2.9 (1.4 to 4.2)
        Year 3 (n = 14, 14)
    0.4 (-0.5 to 2.1)
    2.9 (-0.1 to 5.7)
    No statistical analyses for this end point

    Other pre-specified: Growth Velocity Standard Deviation Score According to Bone Age (GV [SDS/BA])

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    End point title
    		 Growth Velocity Standard Deviation Score According to Bone Age (GV [SDS/BA])
    End point description
    GV measures the annual rate of increase in height. GV (SDS/BA) was obtained by measuring GV, subtracting the bone age- and gender-appropriate mean GV and dividing the result by standard deviation of that mean (as obtained from bone age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) subject’s value was relative to the mean of the reference population. FAS up to Year 3: included all subjects who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year. Here, 'n' signifies those subjects who were evaluable for this measure at the given time point for each group respectively.
    End point type
    Other pre-specified
    End point timeframe
    Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    15
    15
    Units: SDS
    median (inter-quartile range (Q1-Q3))
        Baseline (n = 9, 14)
    -2.8 (-3.9 to -1.7)
    -2.1 (-2.4 to -1.1)
        Year 1 (n = 13, 15)
    0.8 (-0.9 to 2.9)
    0.6 (-0.6 to 2.4)
        Year 2 (n = 14, 13)
    -0.3 (-3.9 to 1.1)
    1.4 (-0.6 to 2)
        Year 3 (n = 14, 14)
    -0.9 (-2.2 to 0.9)
    0.1 (-1.7 to 3.4)
    No statistical analyses for this end point

    Other pre-specified: Insulin-like Growth Factor-1 (IGF-1) Concentration up to Year 3

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    End point title
    		 Insulin-like Growth Factor-1 (IGF-1) Concentration up to Year 3
    End point description
    FAS up to Year 3: included all subjects who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year. Here, 'n' signifies those subjects who were evaluable for this measure at the given time point for each group respectively.
    End point type
    Other pre-specified
    End point timeframe
    Baseline, Year 1, 2, 3
    End point values
    		 Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Number of subjects analysed
    15
    15
    Units: nanogram per milliliter (ng/mL)
    median (full range (min-max))
        Baseline (n = 15, 12)
    157 (61 to 345)
    344.5 (191 to 719)
        Year 1 (n = 15, 15)
    400 (154 to 1370)
    952 (424 to 1658)
        Year 2 (n = 15, 14)
    388 (188 to 1338)
    880 (311 to 1718)
        Year 3 (n = 15, 14)
    405 (239 to 1160)
    657 (323 to 1298)
    No statistical analyses for this end point

    Other pre-specified: Insulin-like Growth Factor-1 (IGF-1) Concentration After Year 3

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    End point title
    		 Insulin-like Growth Factor-1 (IGF-1) Concentration After Year 3
    End point description
    FAS after year 3: included all subjects who received at least 1 dose of the study treatment and who had at least 1 post-baseline height measurement. Here, 'n' signifies those subjects who were evaluable for this measure at given time point.
    End point type
    Other pre-specified
    End point timeframe
    Year 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5, 9, 9.5, 10; 0.5 and 1 year after somatropin discontinuation, Final Height (assessed up to Year 11)
    End point values
    		 Somatropin- After Year 3
    Number of subjects analysed
    21
    Units: milligram per deciliter (mg/dL)
    median (full range (min-max))
        Year 3.5 (n = 8)
    0.05 (0.04 to 0.09)
        Year 4 (n = 19)
    0.06 (0.03 to 0.1)
        Year 4.5 (n = 16)
    0.06 (0.02 to 0.14)
        Year 5 (n = 17)
    0.05 (0.02 to 0.09)
        Year 5.5 (n = 11)
    0.06 (0.02 to 0.09)
        Year 6 (n = 14)
    0.05 (0.02 to 0.08)
        Year 6.5 (n = 8)
    0.07 (0.02 to 0.09)
        Year 7 (n = 8)
    0.06 (0.02 to 0.07)
        Year 7.5 (n = 4)
    0.05 (0.03 to 0.07)
        Year 8 (n = 6)
    0.06 (0.03 to 0.07)
        Year 8.5 (n = 2)
    0.08 (0.07 to 0.08)
        Year 9 (n = 2)
    0.06 (0.06 to 0.07)
        Year 9.5 (n = 1)
    0.07 (0.07 to 0.07)
        Year 10 (n = 1)
    0.05 (0.05 to 0.05)
        0.5 years after somatropin discontinuation (n = 2)
    0.03 (0.03 to 0.04)
        1 year after somatropin discontinuation (n = 2)
    0.03 (0.02 to 0.05)
        Final height (n = 3)
    0.06 (0.05 to 0.07)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Up to 7 days after last dose of study drug
    Adverse event reporting additional 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.
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    17.1
    Reporting groups
    Reporting group title
    Somatropin- After Year 3
    Reporting group description
    		 Subjects with JIA/NeS, who consented to receive treatment beyond 3 years, received somatropin (Genotropin, Genotonorm) 1.4 IU/kg/week, equivalent to 0.46 mg/kg/week divided in 7 daily doses subcutaneously until the additional study drug dose evaluation visit and thereafter received somatropin (Genotropin, Genotonorm) up to 50 mcg/kg/day subcutaneously until the FH was reached or up to Year 11. Final height was confirmed to have been achieved if the growth velocity was <=1.5 cm per year during the preceding 12 months and bone age was >=17 years for boys and 15 years for girls.

    Reporting group title
    Somatropin- Up To Year 3 (Nephrotic Syndrome)
    Reporting group description
    		 Subjects with NeS received somatropin (Genotropin, Genotonorm) 1.4 IU/kg/week, equivalent to 0.46 mg/kg/week, divided in 7 daily doses subcutaneously for up to 3 years. After treatment for 3 years, subjects in this group were assigned to Somatropin- After Year 3 group.

    Reporting group title
    Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)
    Reporting group description
    		 Subjects with JIA received somatropin (Genotropin, Genotonorm) 1.4 IU/kg/week, equivalent to 0.46 mg/kg/week, divided in 7 daily doses subcutaneously for up to 3 years. After treatment for 3 years, subjects in this group were assigned to Somatropin- After Year 3 group.

    Serious adverse events
    		 Somatropin- After Year 3 Somatropin- Up To Year 3 (Nephrotic Syndrome) Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    19 / 21 (90.48%)
    10 / 15 (66.67%)
    10 / 15 (66.67%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Thyroid neoplasm
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Vascular disorders
    Hypertension
         subjects affected / exposed
    1 / 21 (4.76%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Surgical and medical procedures
    Hip arthroplasty
         subjects affected / exposed
    3 / 21 (14.29%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 5
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Oedema
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pyrexia
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Reproductive system and breast disorders
    Ovarian cyst
         subjects affected / exposed
    2 / 21 (9.52%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pulmonary embolism
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Psychiatric disorders
    Depression
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Investigations
    Biopsy kidney
         subjects affected / exposed
    2 / 21 (9.52%)
    3 / 15 (20.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 3
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Wrist fracture
         subjects affected / exposed
    1 / 21 (4.76%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Head injury
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Toxicity to various agents
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Ear and labyrinth disorders
    Vertigo
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Eye disorders
    Glaucoma
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Keratopathy
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    4 / 21 (19.05%)
    1 / 15 (6.67%)
    2 / 15 (13.33%)
         occurrences causally related to treatment / all
    0 / 5
    0 / 1
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Functional gastrointestinal disorder
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Inguinal hernia
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Intussusception
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastric perforation
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Abdominal pain upper
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Renal and urinary disorders
    Glycosuria
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nephrotic syndrome
         subjects affected / exposed
    4 / 21 (19.05%)
    6 / 15 (40.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 10
    0 / 8
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Renal colic
         subjects affected / exposed
    2 / 21 (9.52%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Renal failure
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Arthritis
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Juvenile idiopathic arthritis
         subjects affected / exposed
    5 / 21 (23.81%)
    0 / 15 (0.00%)
    3 / 15 (20.00%)
         occurrences causally related to treatment / all
    0 / 9
    0 / 0
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Musculoskeletal disorder
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Osteonecrosis
         subjects affected / exposed
    2 / 21 (9.52%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Arthralgia
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Cellulitis
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastroenteritis
         subjects affected / exposed
    2 / 21 (9.52%)
    1 / 15 (6.67%)
    2 / 15 (13.33%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 1
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Herpes zoster
         subjects affected / exposed
    1 / 21 (4.76%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Ophthalmic herpes zoster
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pneumonia mycoplasmal
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Appendicitis
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Tooth infection
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Viral infection
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Diabetes mellitus
         subjects affected / exposed
    2 / 21 (9.52%)
    2 / 15 (13.33%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    2 / 2
    2 / 3
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Hypovolaemia
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Somatropin- After Year 3 Somatropin- Up To Year 3 (Nephrotic Syndrome) Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    21 / 21 (100.00%)
    14 / 15 (93.33%)
    13 / 15 (86.67%)
    Vascular disorders
    Haematoma
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    Hypertension
         subjects affected / exposed
    3 / 21 (14.29%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    3
    0
    0
    General disorders and administration site conditions
    Face oedema
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    Hyperthermia
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    Pyrexia
         subjects affected / exposed
    3 / 21 (14.29%)
    2 / 15 (13.33%)
    0 / 15 (0.00%)
         occurrences all number
    3
    2
    0
    Oedema
         subjects affected / exposed
    1 / 21 (4.76%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    1
    1
    0
    Asthenia
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Injection site haemorrhage
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Reproductive system and breast disorders
    Ovarian cyst
         subjects affected / exposed
    1 / 21 (4.76%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    2
    2
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    Oropharyngeal pain
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    2
    0
    0
    Psychiatric disorders
    Depression
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    2
    0
    1
    Investigations
    Blood cholesterol
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    0
    2
    0
    Blood triglycerides
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    0
    2
    0
    Insulin-like growth factor increased
         subjects affected / exposed
    3 / 21 (14.29%)
    3 / 15 (20.00%)
    0 / 15 (0.00%)
         occurrences all number
    3
    3
    0
    Red blood cell sedimentation rate increased
         subjects affected / exposed
    4 / 21 (19.05%)
    0 / 15 (0.00%)
    5 / 15 (33.33%)
         occurrences all number
    7
    0
    10
    Blood follicle stimulating hormone increased
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Blood HIV RNA increased
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Injury, poisoning and procedural complications
    Ligament sprain
         subjects affected / exposed
    2 / 21 (9.52%)
    0 / 15 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    2
    0
    2
    Radius fracture
         subjects affected / exposed
    1 / 21 (4.76%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    1
    1
    0
    Hand fracture
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Wound
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Cardiac disorders
    Angina pectoris
         subjects affected / exposed
    2 / 21 (9.52%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    2
    0
    1
    Left ventricular hypertrophy
         subjects affected / exposed
    2 / 21 (9.52%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    2
    0
    0
    Tachycardia
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    3 / 21 (14.29%)
    3 / 15 (20.00%)
    2 / 15 (13.33%)
         occurrences all number
    4
    4
    3
    Hypotonia
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Presyncope
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Blood and lymphatic system disorders
    Hypercoagulation
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    Anaemia
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Ear and labyrinth disorders
    Deafness
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    Eye disorders
    Uveitis
         subjects affected / exposed
    3 / 21 (14.29%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    5
    0
    1
    Ocular hypertension
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    4 / 21 (19.05%)
    1 / 15 (6.67%)
    2 / 15 (13.33%)
         occurrences all number
    5
    2
    2
    Constipation
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    Nausea
         subjects affected / exposed
    2 / 21 (9.52%)
    1 / 15 (6.67%)
    2 / 15 (13.33%)
         occurrences all number
    2
    1
    2
    Vomiting
         subjects affected / exposed
    2 / 21 (9.52%)
    2 / 15 (13.33%)
    1 / 15 (6.67%)
         occurrences all number
    2
    2
    1
    Cheilitis
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Diarrhoea
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Rectal haemorrhage
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Skin and subcutaneous tissue disorders
    Acanthosis nigricans
         subjects affected / exposed
    1 / 21 (4.76%)
    2 / 15 (13.33%)
    0 / 15 (0.00%)
         occurrences all number
    1
    2
    0
    Acne
         subjects affected / exposed
    3 / 21 (14.29%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    3
    0
    0
    Eczema
         subjects affected / exposed
    2 / 21 (9.52%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    2
    0
    0
    Rash
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Nail dystrophy
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Rash macular
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Renal and urinary disorders
    Dysuria
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    Nephrotic syndrome
         subjects affected / exposed
    7 / 21 (33.33%)
    11 / 15 (73.33%)
    0 / 15 (0.00%)
         occurrences all number
    32
    35
    0
    Hypercalciuria
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    Proteinuria
         subjects affected / exposed
    3 / 21 (14.29%)
    6 / 15 (40.00%)
    0 / 15 (0.00%)
         occurrences all number
    6
    11
    0
    Renal failure
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    4 / 21 (19.05%)
    1 / 15 (6.67%)
    1 / 15 (6.67%)
         occurrences all number
    6
    1
    3
    Joint effusion
         subjects affected / exposed
    4 / 21 (19.05%)
    0 / 15 (0.00%)
    4 / 15 (26.67%)
         occurrences all number
    5
    0
    5
    Osteopenia
         subjects affected / exposed
    0 / 21 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Juvenile idiopathic arthritis
         subjects affected / exposed
    6 / 21 (28.57%)
    0 / 15 (0.00%)
    3 / 15 (20.00%)
         occurrences all number
    9
    0
    4
    Synovitis
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    Amyotrophy
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Knee deformity
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Muscle atrophy
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Limb asymmetry
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    1 / 21 (4.76%)
    1 / 15 (6.67%)
    1 / 15 (6.67%)
         occurrences all number
    1
    1
    1
    Ear infection
         subjects affected / exposed
    2 / 21 (9.52%)
    3 / 15 (20.00%)
    0 / 15 (0.00%)
         occurrences all number
    3
    4
    0
    Influenza
         subjects affected / exposed
    2 / 21 (9.52%)
    1 / 15 (6.67%)
    1 / 15 (6.67%)
         occurrences all number
    2
    1
    1
    Gastroenteritis
         subjects affected / exposed
    4 / 21 (19.05%)
    2 / 15 (13.33%)
    1 / 15 (6.67%)
         occurrences all number
    4
    2
    1
    Nasopharyngitis
         subjects affected / exposed
    3 / 21 (14.29%)
    3 / 15 (20.00%)
    0 / 15 (0.00%)
         occurrences all number
    5
    4
    0
    Pharyngitis
         subjects affected / exposed
    1 / 21 (4.76%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    1
    1
    0
    Perichondritis
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    Rhinitis
         subjects affected / exposed
    2 / 21 (9.52%)
    3 / 15 (20.00%)
    0 / 15 (0.00%)
         occurrences all number
    2
    3
    0
    Tracheobronchitis
         subjects affected / exposed
    1 / 21 (4.76%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    1
    1
    0
    Viral infection
         subjects affected / exposed
    2 / 21 (9.52%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    2
    0
    1
    Viral tracheitis
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    Pneumonia mycoplasmal
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Urinary tract infection
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    5
    0
    0
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    1 / 21 (4.76%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    1
    1
    0
    Fluid retention
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    Hypercholesterolaemia
         subjects affected / exposed
    1 / 21 (4.76%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    1
    1
    0
    Hyperinsulinism
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    Malnutrition
         subjects affected / exposed
    0 / 21 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    Hypertriglyceridaemia
         subjects affected / exposed
    1 / 21 (4.76%)
    2 / 15 (13.33%)
    0 / 15 (0.00%)
         occurrences all number
    1
    2
    0
    Sodium retention
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    Diabetes mellitus
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Glucose tolerance impaired
         subjects affected / exposed
    3 / 21 (14.29%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    3
    0
    0
    Insulin resistance
         subjects affected / exposed
    4 / 21 (19.05%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    4
    0
    0
    Mineral deficiency
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Vitamin D deficiency
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    Tendonitis
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    06 Aug 2001
    The study duration was increased from 48 months to 69 months.
    20 Oct 2003
    For the children who did not wish to continue the treatment beyond 36 months, it was proposed that they had to undergo an evaluation of body composition by a DEXA scan and glucose tolerance by an oral glucose tolerance test, 1 year after the discontinuation of treatment.
    29 Nov 2007
    Treatment with somatropin was adapted according to IGF-1 level. If the level of IGF-1 was greater than (>)+2 standard deviation (SD) for the chronological age and sex, the dose of the growth hormone was to be decreased by 20 percent (%).

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    Results for secondary and other pre-specified endpoints (except IGF-1) are reported for only up to 3 years because data beyond Year 3 was not summarized as the study was terminated due to Good Clinical Practice (GCP) non-compliance issues.
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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