Treatment With Recombinant Human Growth Hormone (GH) in Children With Short Stature Secondary to a Long Term Corticoid Therapy

• Sponsor: Pfizer (Industry)
• Overall Status: Terminated
• CT.gov ID: NCT00174187
• Collaborator: (none)
• Enrollment: 30
• Locations: 2
• Arm: 1
• Duration (Months): 132
• Patients Per Site: 15
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief Summary

• To assess the effect of a long-term treatment by Genotonorm on linear growth in children with short stature receiving steroid therapy

• To assess the effect of a long term treatment with Genotonorm on bone mineralisation

• To assess the effect of a long term treatment with Genotonorm on body composition

Condition or Disease	Intervention/Treatment	Phase
Endocrine System Diseases	Drug: Somatropin	Phase 3

Detailed Description

This trial terminated on 10-Jun-2011 due to prolonged issues with drug accountability and data collection discrepancies. The decision to terminate was not based on any safety concerns.

Study Design

• Study Type: Interventional
• Actual Enrollment: 30 participants
• Allocation: Non-Randomized
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: 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.
• Study Start Date: Sep 1, 2000
• Actual Primary Completion Date: Sep 1, 2011
• Actual Study Completion Date: Sep 1, 2011

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Somatropin	Drug: Somatropin; liquid, daily, until final height Dosage: 0,46 mg/kg/week . The maximum dose should not exceed 50 µg/Kg/day

Outcome Measures

Primary Outcome Measures

1. Change From Baseline in Height Standard Deviation Score According to Chronological Age (SDS/CA) at Year 3 [Baseline, Year 3]

   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) participant's value was relative to the mean of the reference population.

2. Change From Baseline in Height Standard Deviation Score According to Chronological Age (SDS/CA) at Final Height [Baseline, when final height was reached (assessed up to Year 11)]

   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) participant's value was relative to the mean of the reference population.

3. Change From Baseline in Weight Standard Deviation Score (SDS) at Final Height [Baseline, when final height was reached (assessed up to Year 11)]

   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) participant's value was relative to the mean of the reference population.

4. Puberty Stage at Final Height [When final height was reached (assessed up to Year 11)]

   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.

Secondary Outcome Measures

1. Bone Age [Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11]

   Bone age was determined by the Greulich and Pyle method using left wrist and hand X-ray.

2. Lean Body Mass [Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11]

   Lean body mass, a measurement of body composition, was assessed by Dual Energy X-ray Absorptiometry (DEXA) scan.

3. Annual Percent Change in Lean Body Mass at Year 1, 2 and 3 [Baseline, Year 1, 2, 3]

   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.

4. Percent Change From Baseline in Lean Body Mass at Year 3 [Baseline, Year 3]

   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.

5. Lean Body Mass as Percentage of Total Weight [Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11]

   Lean body mass, a measurement of body composition, was assessed by DEXA scan.

6. Lean Body Mass Standard Deviation Score According to Chronological Age (SDS/CA) [Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11]

   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) participant's value was relative to the mean of the reference population.

7. Fat Mass [Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11]

   Fat mass, a measurement of body composition, was assessed by DEXA scan.

8. Annual Percent Change in Fat Mass at Year 1, 2 and 3 [Baseline, Year 1, 2, 3]

   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.

9. Percent Change From Baseline in Fat Mass at Year 3 [Baseline, Year 3]

   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.

10. Fat Mass as Percentage of Total Weight [Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11]

    Fat mass, a measurement of body composition, was assessed by DEXA scan.

11. Fat Mass Standard Deviation Score According to Chronological Age (SDS/CA) [Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11]

    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) participant's value was relative to the mean of the reference population.

12. Apparent Bone Mineral Density of Lumbar Spine (BMAD [LS]) [Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11]

    BMAD (LS) was assessed by DEXA scan.

13. Apparent Bone Mineral Density Standard Deviation Score of Lumbar Spine According to Chronological Age (BMAD [LS] [SDS/CA]) [Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11]

    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) participant's value was relative to the mean of the reference population.

14. Apparent Bone Mineral Density Standard Deviation Score of Lumber Spine According to Tanner Puberty Stage (BMAD [LS] [SDS/Tanner Puberty Stage]) [Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11]

    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) participant's value was relative to the mean of the reference population.

15. Bone Mineral Density of Total Body (BMD [TB]) [Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11]

    BMD (TB) was assessed by DEXA scan.

16. Bone Mineral Density of Lumbar Spine (BMD [LS]) [Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11]

    BMD (LS) was assessed by DEXA scan.

17. Bone Mineral Content of Total Body (BMC [TB]) [Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11]

    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.

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

    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.

19. Percent Change From Baseline in Bone Mineral Content of Total Body (BMC [TB]) at Year 3 [Baseline, Year 3]

    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.

20. Bone Mineral Content Standard Deviation Score of Total Body According to Chronological Age (BMC [TB] [SDS/CA]) [Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11]

    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) participant's value was relative to the mean of the reference population.

21. Bone Mineral Content Standard Deviation Score of Total Body According to Tanner Puberty Stage (BMC [TB] [SDS/Tanner Puberty Stage]) [Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11]

    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) participant's value was relative to the mean of the reference population.

Other Outcome Measures

1. Growth Velocity (GV) [Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11]

   Growth velocity measures the annual rate of increase in height.

2. Growth Velocity Standard Deviation Score According to Chronological Age (GV [SDS/CA]) [Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11]

   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) participant's value was relative to the mean of the reference population.

3. Growth Velocity Standard Deviation Score According to Bone Age (GV [SDS/BA]) [Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11]

   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) participant's value was relative to the mean of the reference population.

4. Insulin-like Growth Factor-1 (IGF-1) Concentration up to Year 3 [Baseline, Year 1, 2, 3]

5. Insulin-like Growth Factor-1 (IGF-1) Concentration After Year 3 [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)]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 11 Years to 17 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Children with juvenile arthritis or nephrotic syndrome

• Before or during puberty

Exclusion Criteria:

• Diabetes Type 1 and 2

• Endocrine disease, except well substituted hypothyroidism

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Pfizer Investigational Site	Paris		France	75019
2	Pfizer Investigational Site	Paris		France	75743

Sponsors and Collaborators

• Pfizer

Investigators

• Study Director: Pfizer CT.gov Call Center, Pfizer

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• To obtain contact information for a study center near you, click here.

Publications

None provided.

• Responsible Party: Pfizer
• ClinicalTrials.gov Identifier: NCT00174187
• Other Study ID Numbers: 307-MET-9002-0009; A6281016
• First Posted: Sep 15, 2005
• Last Update Posted: Dec 4, 2012
• Last Verified: Nov 1, 2012

• Additional relevant MeSH terms: Dwarfism; Endocrine System Diseases

Study Results

Participant Flow

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)	Somatropin- After Year 3
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.	Participants 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 microgram (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.
Period Title: Period 1 (up to 3 Years)
STARTED	15	15	0
COMPLETED	15	11	0
NOT COMPLETED	0	4	0
Period Title: Period 1 (up to 3 Years)
STARTED	0	0	26
Consented	0	0	24
COMPLETED	0	0	21
NOT COMPLETED	0	0	5
Period Title: Period 1 (up to 3 Years)
STARTED	0	0	21
COMPLETED	0	0	13
NOT COMPLETED	0	0	8

Baseline Characteristics

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)	Total
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.	Total of all reporting groups
Overall Participants	15	15	30
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	11.14 (3.32)	11.96 (3.80)	11.55 (3.53)
Sex: Female, Male (Count of Participants)
Female	9 60%	2 13.3%	11 36.7%
Male	6 40%	13 86.7%	19 63.3%

Outcome Measures

1. Primary Outcome

Title	Change From Baseline in Height Standard Deviation Score According to Chronological Age (SDS/CA) at Year 3
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) participant's value was relative to the mean of the reference population.
Time Frame	Baseline, Year 3

Outcome Measure Data

Analysis Population Description

Full Analysis Set (FAS) up to Year 3: included all participants who had at least 1 post-baseline height measurement and were treated with the study drug for at least 1 year.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	15	15
Baseline	-3.6	-2.5
Change at Year 3	0.2	1.1

2. Primary Outcome

Title	Change From Baseline in Height Standard Deviation Score According to Chronological Age (SDS/CA) at Final Height
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) participant's value was relative to the mean of the reference population.
Time Frame	Baseline, when final height was reached (assessed up to Year 11)

Outcome Measure Data

Analysis Population Description

FAS- after Year 3: included all participants who received at least 1 dose of the study treatment and who had at least 1 post-baseline height measurement. Here, 'n' signifies those participants who were evaluable for this measure at given time points.

Arm/Group Title	Somatropin- After Year 3
Arm/Group Description	Participants 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 microgram (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.
Measure Participants	21
Baseline (n = 21)	-2.86
Change at Final Height (n = 4)	0.71

3. Primary Outcome

Title	Change From Baseline in Weight Standard Deviation Score (SDS) at Final Height
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) participant's value was relative to the mean of the reference population.
Time Frame	Baseline, when final height was reached (assessed up to Year 11)

Outcome Measure Data

Analysis Population Description

FAS- after Year 3: included all participants who received at least one dose of the study treatment and who had at least one post-baseline height measurement. Here, 'n' signifies those participants who were evaluable for this measure at given time point.

Arm/Group Title	Somatropin- After Year 3
Arm/Group Description	Participants 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 microgram (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.
Measure Participants	21
Baseline (n = 21)	-1.53
Change at Final Height (n = 4)	-0.43

4. Primary Outcome

Title	Puberty Stage at Final Height
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.
Time Frame	When final height was reached (assessed up to Year 11)

Outcome Measure Data

Analysis Population Description

FAS- after Year 3: included all participants who received at least one dose of study treatment and who had at least one post-baseline height measurement. 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. 'n'=participants who were evaluable for given components of puberty assessment.

Arm/Group Title	Somatropin- After Year 3
Arm/Group Description	Participants 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 microgram (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.
Measure Participants	4
Pubic hair: Stage I (n=4)	0 0%
Pubic hair: Stage II (n=4)	0 0%
Pubic hair: Stage III (n=4)	0 0%
Pubic hair: Stage IV (n=4)	0 0%
Pubic hair: Stage V (n=4)	4 26.7%
Breast development: Stage I (n=1)	0 0%
Breast development: Stage II (n=1)	0 0%
Breast development: Stage III (n=1)	0 0%
Breast development: Stage IV (n=1)	0 0%
Breast development: Stage V (n=1)	1 6.7%

5. Secondary Outcome

Title	Bone Age
Description	Bone age was determined by the Greulich and Pyle method using left wrist and hand X-ray.
Time Frame	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11

Outcome Measure Data

Analysis Population Description

FAS up to Year 3: included all participants 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 participants who were evaluable for this measure at given time points for each group, respectively.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	15	15
Baseline (n = 14, 15)	8.9	10.5
Year 1 (n = 14, 15)	9.5	11.5
Year 2 (n = 15, 13)	10.0	14.0
Year 3 (n = 15, 14)	11.3	14.0

6. Secondary Outcome

Title	Lean Body Mass
Description	Lean body mass, a measurement of body composition, was assessed by Dual Energy X-ray Absorptiometry (DEXA) scan.
Time Frame	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11

Outcome Measure Data

Analysis Population Description

FAS up to Year 3: included all participants 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 participants who were evaluable for this measure at the given time point for each group respectively.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	15	15
Baseline (n = 15, 15)	17.69	23.56
Year 1 (n = 15, 13)	19.77	31.16
Year 2 (n = 14, 12)	21.20	34.14
Year 3 (n = 14, 11)	23.21	36.38

7. Secondary Outcome

Title	Annual Percent Change in Lean Body Mass at Year 1, 2 and 3
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.
Time Frame	Baseline, Year 1, 2, 3

Outcome Measure Data

Analysis Population Description

FAS up to Year 3: included all participants 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 participants who were evaluable for this measure at the given time point for each group respectively.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	15	15
Annual Change at Year 1 (n = 15, 13)	18.95	22.97
Annual Change at Year 2 (n = 14, 11)	7.75	7.33
Annual Change at Year 3 (n = 13, 9)	9.83	7.90

8. Secondary Outcome

Title	Percent Change From Baseline in Lean Body Mass at Year 3
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.
Time Frame	Baseline, Year 3

Outcome Measure Data

Analysis Population Description

FAS up to Year 3: included all participants who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year. Here, 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	14	11
Median (Inter-Quartile Range) [percent change]	36.82	54.44

9. Secondary Outcome

Title	Lean Body Mass as Percentage of Total Weight
Description	Lean body mass, a measurement of body composition, was assessed by DEXA scan.
Time Frame	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11

Outcome Measure Data

Analysis Population Description

FAS up to Year 3: included all participants 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 participants who were evaluable for this measure at the given time point for each group respectively.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	15	15
Baseline (n = 15, 15)	70.56	60.69
Year 1 (n = 15, 13)	76.57	65.97
Year 2 (n = 14, 12)	73.32	69.95
Year 3 (n = 14, 11)	72.94	67.24

10. Secondary Outcome

Title	Lean Body Mass Standard Deviation Score According to Chronological Age (SDS/CA)
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) participant's value was relative to the mean of the reference population.
Time Frame	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11

Outcome Measure Data

Analysis Population Description

FAS up to Year 3: included all participants 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 participants who were evaluable for this measure at the given time point for each group respectively.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	15	15
Baseline (n = 15, 15)	-1.50	-0.90
Year 1 (n = 15, 13)	-1.13	-0.52
Year 2 (n = 14, 12)	-1.26	-0.87
Year 3 (n = 13, 11)	-1.75	-1.29

11. Secondary Outcome

Title	Fat Mass
Description	Fat mass, a measurement of body composition, was assessed by DEXA scan.
Time Frame	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11

Outcome Measure Data

Analysis Population Description

FAS up to Year 3: included all participants 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 participants who were evaluable for this measure at the given time point for each group respectively.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	15	15
Baseline (n = 15, 15)	5.47	13.62
Year 1 (n = 15, 13)	4.81	10.99
Year 2 (n = 14, 12)	6.97	16.70
Year 3 (n = 14, 11)	6.90	15.19

12. Secondary Outcome

Title	Annual Percent Change in Fat Mass at Year 1, 2 and 3
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.
Time Frame	Baseline, Year 1, 2, 3

Outcome Measure Data

Analysis Population Description

FAS up to Year 3: included all participants 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 participants who were evaluable for this measure at the given time point for each group respectively.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	15	15
Annual Change at Year 1 (n = 15, 13)	-11.21	-3.71
Annual Change at Year 2 (n = 14, 11)	29.52	-1.05
Annual Change at Year 3 (n = 13, 9)	26.61	19.37

13. Secondary Outcome

Title	Percent Change From Baseline in Fat Mass at Year 3
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.
Time Frame	Baseline, Year 3

Outcome Measure Data

Analysis Population Description

FAS up to Year 3: included all participants who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year. Here, 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	14	11
Median (Inter-Quartile Range) [percent change]	19.46	37.56

14. Secondary Outcome

Title	Fat Mass as Percentage of Total Weight
Description	Fat mass, a measurement of body composition, was assessed by DEXA scan.
Time Frame	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11

Outcome Measure Data

Analysis Population Description

FAS up to Year 3: included all participants 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 participants who were evaluable for this measure at the given time point for each group respectively.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	15	15
Baseline (n = 15, 15)	25.0	33.8
Year 1 (n = 15, 13)	17.4	30.0
Year 2 (n = 14, 12)	21.1	27.1
Year 3 (n = 14, 11)	21.3	26.4

15. Secondary Outcome

Title	Fat Mass Standard Deviation Score According to Chronological Age (SDS/CA)
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) participant's value was relative to the mean of the reference population.
Time Frame	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11

Outcome Measure Data

Analysis Population Description

FAS up to Year 3: included all participants 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 participants who were evaluable for this measure at the given time point for each group respectively.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	15	15
Baseline (n = 15, 15)	1.51	2.20
Year 1 (n = 15, 13)	0.80	1.98
Year 2 (n = 14, 12)	0.86	1.79
Year 3 (n = 13, 11)	1.27	1.74

16. Secondary Outcome

Title	Apparent Bone Mineral Density of Lumbar Spine (BMAD [LS])
Description	BMAD (LS) was assessed by DEXA scan.
Time Frame	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11

Outcome Measure Data

Analysis Population Description

FAS up to Year 3: included all participants 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 participants who were evaluable for this measure at the given time point for each group respectively.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	15	15
Baseline (n = 15, 14)	0.184	0.223
Year 1 (n = 14, 13)	0.209	0.237
Year 2 (n = 15, 14)	0.215	0.249
Year 3 (n = 13, 12)	0.239	0.242

17. Secondary Outcome

Title	Apparent Bone Mineral Density Standard Deviation Score of Lumbar Spine According to Chronological Age (BMAD [LS] [SDS/CA])
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) participant's value was relative to the mean of the reference population.
Time Frame	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11

Outcome Measure Data

Analysis Population Description

FAS up to Year 3: included all participants 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 participants who were evaluable for this measure at the given time point for each group respectively.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	15	15
Baseline (n = 15, 14)	-3.21	-1.53
Year 1 (n = 14, 13)	-2.66	-1.51
Year 2 (n = 15, 14)	-2.55	-1.42
Year 3 (n 12, 12)	-1.77	-1.63

18. Secondary Outcome

Title	Apparent Bone Mineral Density Standard Deviation Score of Lumber Spine According to Tanner Puberty Stage (BMAD [LS] [SDS/Tanner Puberty Stage])
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) participant's value was relative to the mean of the reference population.
Time Frame	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11

Outcome Measure Data

Analysis Population Description

FAS up to Year 3: included all participants 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 participants who were evaluable for this measure at the given time point for each group respectively.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	15	15
Baseline (n = 8, 8)	-4.27	-3.09
Year 1 (n = 10, 7)	-3.70	-3.23
Year 2 (n = 10, 9)	-4.11	-2.60
Year 3 (n = 10, 8)	-3.04	-2.96

19. Secondary Outcome

Title	Bone Mineral Density of Total Body (BMD [TB])
Description	BMD (TB) was assessed by DEXA scan.
Time Frame	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11

Outcome Measure Data

Analysis Population Description

FAS up to Year 3: included all participants 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 participants who were evaluable for this measure at the given time point for each group respectively.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	15	15
Baseline (n = 15, 15)	0.78	0.90
Year 1 (n = 15, 14)	0.80	0.92
Year 2 (n = 14, 11)	0.81	0.96
Year 3 (n = 13, 11)	0.84	0.99

20. Secondary Outcome

Title	Bone Mineral Density of Lumbar Spine (BMD [LS])
Description	BMD (LS) was assessed by DEXA scan.
Time Frame	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11

Outcome Measure Data

Analysis Population Description

FAS up to Year 3: included all participants 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 participants who were evaluable for this measure at the given time point for each group respectively.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	15	15
Baseline (n = 15, 15)	0.55	0.72
Year 1 (n = 14, 14)	0.61	0.76
Year 2 (n = 15, 14)	0.64	0.85
Year 3 (n = 13, 12)	0.74	0.93

21. Secondary Outcome

Title	Bone Mineral Content of Total Body (BMC [TB])
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.
Time Frame	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11

Outcome Measure Data

Analysis Population Description

FAS up to Year 3: included all participants 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 participants who were evaluable for this measure at the given time point for each group respectively.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	15	15
Baseline (n = 15, 15)	636.36	1233.58
Year 1 (n = 15, 14)	775.46	1576.15
Year 2 (n = 14, 11)	855.70	2003.49
Year 3 (n = 13, 11)	1112.78	1997.74

22. Secondary Outcome

Title	Annual Percent Change in Bone Mineral Content of Total Body (BMC [TB]) at Year 1, 2 and 3
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.
Time Frame	Baseline, Year 1, 2, 3

Outcome Measure Data

Analysis Population Description

FAS up to Year 3: included all participants 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 participants who were evaluable for this measure at the given time point for each group respectively.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	15	15
Year 1 (n = 15, 14)	12.51	20.16
Year 2 (n = 14, 11)	17.47	13.91
Year 3 (n = 12, 9)	12.03	7.39

23. Secondary Outcome

Title	Percent Change From Baseline in Bone Mineral Content of Total Body (BMC [TB]) at Year 3
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.
Time Frame	Baseline, Year 3

Outcome Measure Data

Analysis Population Description

FAS up to Year 3: included all participants who had at least one post-baseline height measurement and were treated with the study drug for at least 1 year. Here, 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	13	11
Median (Inter-Quartile Range) [percent change]	46.63	60.56

24. Secondary Outcome

Title	Bone Mineral Content Standard Deviation Score of Total Body According to Chronological Age (BMC [TB] [SDS/CA])
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) participant's value was relative to the mean of the reference population.
Time Frame	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11

Outcome Measure Data

Analysis Population Description

FAS up to Year 3: included all participants 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 participants who were evaluable for this measure at the given time point for each group respectively.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	15	15
Baseline (n = 15, 15)	-1.95	-0.42
Year 1 (n = 15, 14)	-1.85	-0.19
Year 2 (n = 14, 11)	-1.66	-0.37
Year 3 (n = 12, 11)	-1.65	-0.09

25. Secondary Outcome

Title	Bone Mineral Content Standard Deviation Score of Total Body According to Tanner Puberty Stage (BMC [TB] [SDS/Tanner Puberty Stage])
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) participant's value was relative to the mean of the reference population.
Time Frame	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11

Outcome Measure Data

Analysis Population Description

FAS up to Year 3: included all participants 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 participants who were evaluable for this measure at the given time point for each group respectively.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	15	15
Baseline (n = 8, 8)	-2.19	1.41
Year 1 (n = 10, 8)	-1.56	-0.52
Year 2 (n = 9, 7)	-2.38	-0.47
Year 3 (n = 9, 7)	-2.38	-0.17

26. Other Pre-specified Outcome

Title	Growth Velocity (GV)
Description	Growth velocity measures the annual rate of increase in height.
Time Frame	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11

Outcome Measure Data

Analysis Population Description

FAS up to Year 3: included all participants 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 participants who were evaluable for this measure at the given time point for each group respectively.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	15	15
Baseline (n = 11, 14)	2.9	3.8
Year 1 (n = 14, 15)	6.5	8.3
Year 2 (n = 14, 15)	5.3	7.0
Year 3 (n = 14, 14)	4.5	5.9

27. Other Pre-specified Outcome

Title	Growth Velocity Standard Deviation Score According to Chronological Age (GV [SDS/CA])
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) participant's value was relative to the mean of the reference population.
Time Frame	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11

Outcome Measure Data

Analysis Population Description

FAS up to Year 3: included all participants 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 participants who were evaluable for this measure at the given time point for each group respectively.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	15	15
Baseline (n = 10, 14)	-1.7	-2.2
Year 1 (n = 14, 15)	1.0	1.3
Year 2 (n = 14, 15)	0.1	2.9
Year 3 (n = 14, 14)	0.4	2.9

28. Other Pre-specified Outcome

Title	Growth Velocity Standard Deviation Score According to Bone Age (GV [SDS/BA])
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) participant's value was relative to the mean of the reference population.
Time Frame	Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11

Outcome Measure Data

Analysis Population Description

FAS up to Year 3: included all participants 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 participants who were evaluable for this measure at the given time point for each group respectively.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	15	15
Baseline (n = 9, 14)	-2.8	-2.1
Year 1 (n = 13, 15)	0.8	0.6
Year 2 (n = 14, 13)	-0.3	1.4
Year 3 (n = 14, 14)	-0.9	0.1

29. Other Pre-specified Outcome

Title	Insulin-like Growth Factor-1 (IGF-1) Concentration up to Year 3
Time Frame	Baseline, Year 1, 2, 3

Outcome Measure Data

Analysis Population Description

FAS up to Year 3: included all participants 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 participants who were evaluable for this measure at the given time point for each group respectively.

Arm/Group Title	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)	Somatropin- Up To Year 3 (Nephrotic Syndrome)
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.	Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.
Measure Participants	15	15
Baseline (n = 15, 12)	157.0	344.5
Year 1 (n = 15, 15)	400.0	952.0
Year 2 (n = 15, 14)	388.0	880.0
Year 3 (n = 15, 14)	405.0	657.0

30. Other Pre-specified Outcome

Title	Insulin-like Growth Factor-1 (IGF-1) Concentration After Year 3
Time Frame	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)

Outcome Measure Data

Analysis Population Description

FAS After year 3: included all participants who received at least 1 dose of the study treatment and who had at least 1 post-baseline height measurement. Here, 'n' signifies those participants who were evaluable for this measure at given time point.

Arm/Group Title	Somatropin- After Year 3
Arm/Group Description	Participants 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 microgram (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.
Measure Participants	21
Year 3.5 (n = 8)	0.05
Year 4 (n = 19)	0.06
Year 4.5 (n = 16)	0.06
Year 5 (n = 17)	0.05
Year 5.5 (n = 11)	0.06
Year 6 (n = 14)	0.05
Year 6.5 (n = 8)	0.07
Year 7 (n = 8)	0.06
Year 7.5 (n = 4)	0.05
Year 8 (n = 6)	0.06
Year 8.5 (n = 2)	0.08
Year 9 (n = 2)	0.06
Year 9.5 (n = 1)	0.07
Year 10 (n = 1)	0.05
0.5 years after somatropin discontinuation (n = 2)	0.03
1 year after somatropin discontinuation (n = 2)	0.03
Final height (n = 3)	0.06

Adverse Events

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- Up To Year 3 (Juvenile Idiopathic Arthritis)		Somatropin- Up To Year 3 (Nephrotic Syndrome)		Somatropin- After Year 3
Arm/Group Description	Participants with juvenile idiopathic arthritis (JIA) received somatropin (Genotropin, Genotonorm) 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 subcutaneously for up to 3 years. After treatment for 3 years, participants in this group were assigned to Somatropin- After Year 3 group.		Participants with nephrotic syndrome (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, participants in this group were assigned to Somatropin- After Year 3 group.		Participants 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 microgram (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.
All Cause Mortality
	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)		Somatropin- Up To Year 3 (Nephrotic Syndrome)		Somatropin- After Year 3
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	/ (NaN)		/ (NaN)		/ (NaN)
Serious Adverse Events
	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)		Somatropin- Up To Year 3 (Nephrotic Syndrome)		Somatropin- After Year 3
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	13/15 (86.7%)		14/15 (93.3%)		19/21 (90.5%)
Ear and labyrinth disorders
Vertigo	0/15 (0%)		1/15 (6.7%)		0/21 (0%)
Eye disorders
Visual acuity reduced	1/15 (6.7%)		0/15 (0%)		0/21 (0%)
Glaucoma	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Keratopathy	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Gastrointestinal disorders
Abdominal pain	2/15 (13.3%)		1/15 (6.7%)		4/21 (19%)
Dyspepsia	0/15 (0%)		1/15 (6.7%)		0/21 (0%)
Gastric perforation	1/15 (6.7%)		0/15 (0%)		1/21 (4.8%)
Inguinal hernia	1/15 (6.7%)		0/15 (0%)		1/21 (4.8%)
Intussusception	0/15 (0%)		1/15 (6.7%)		0/21 (0%)
General disorders
Condition aggravated	3/15 (20%)		2/15 (13.3%)		0/21 (0%)
Disease recurrence	1/15 (6.7%)		1/15 (6.7%)		0/21 (0%)
Inflammation	1/15 (6.7%)		0/15 (0%)		0/21 (0%)
Oedema	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Pyrexia	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Headache and abdominal pain	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Infections and infestations
Cellulitis	0/15 (0%)		1/15 (6.7%)		0/21 (0%)
Gastroenteritis	2/15 (13.3%)		1/15 (6.7%)		2/21 (9.5%)
Herpes zoster	0/15 (0%)		1/15 (6.7%)		1/21 (4.8%)
Herpes zoster ophthalmic	1/15 (6.7%)		0/15 (0%)		1/21 (4.8%)
Pneumonia	0/15 (0%)		1/15 (6.7%)		0/21 (0%)
Appendicitis	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Tooth infection	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Viral infection	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Injury, poisoning and procedural complications
Wrist fracture	0/15 (0%)		1/15 (6.7%)		1/21 (4.8%)
Head injury	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Toxicity to various agents	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Investigations
Biopsy kidney	0/15 (0%)		1/15 (6.7%)		2/21 (9.5%)
Metabolism and nutrition disorders
Dehydration	0/15 (0%)		1/15 (6.7%)		0/21 (0%)
Diabetes mellitus	1/15 (6.7%)		2/15 (13.3%)		2/21 (9.5%)
Hypovolaemia	0/15 (0%)		1/15 (6.7%)		0/21 (0%)
Insulin resistant diabetes	0/15 (0%)		1/15 (6.7%)		0/21 (0%)
Musculoskeletal and connective tissue disorders
Arthritis	1/15 (6.7%)		0/15 (0%)		0/21 (0%)
Juvenile arthritis	2/15 (13.3%)		0/15 (0%)		6/21 (28.6%)
Osteonecrosis	1/15 (6.7%)		0/15 (0%)		2/21 (9.5%)
Rheumatoid arthritis	3/15 (20%)		0/15 (0%)		1/21 (4.8%)
Arthralgia	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Thyroid neoplasm	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Nervous system disorders
Headache	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Psychiatric disorders
Depression	1/15 (6.7%)		0/15 (0%)		1/21 (4.8%)
Renal and urinary disorders
Nephrotic syndrome	0/15 (0%)		9/15 (60%)		4/21 (19%)
Renal colic	1/15 (6.7%)		0/15 (0%)		2/21 (9.5%)
Renal failure acute	0/15 (0%)		1/15 (6.7%)		0/21 (0%)
Reproductive system and breast disorders
Ovarian cyst	0/15 (0%)		1/15 (6.7%)		2/21 (9.5%)
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism	0/15 (0%)		2/15 (13.3%)		0/21 (0%)
Surgical and medical procedures
Hip arthroplasty	0/15 (0%)		0/15 (0%)		3/21 (14.3%)
Vascular disorders
Hypertension	0/15 (0%)		1/15 (6.7%)		1/21 (4.8%)
Other (Not Including Serious) Adverse Events
	Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis)		Somatropin- Up To Year 3 (Nephrotic Syndrome)		Somatropin- After Year 3
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	13/15 (86.7%)		14/15 (93.3%)		21/21 (100%)
Blood and lymphatic system disorders
Hypercoagulation	0/15 (0%)		1/15 (6.7%)		0/21 (0%)
Cardiac disorders
Angina pectoris	1/15 (6.7%)		0/15 (0%)		2/21 (9.5%)
Left ventricular hypertrophy	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Tachycardia	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Ear and labyrinth disorders
Auricular perichondritis	1/15 (6.7%)		0/15 (0%)		1/21 (4.8%)
Deafness	0/15 (0%)		1/15 (6.7%)		0/21 (0%)
Eye disorders
Uveitis	1/15 (6.7%)		0/15 (0%)		3/21 (14.3%)
Gastrointestinal disorders
Abdominal pain	2/15 (13.3%)		1/15 (6.7%)		3/21 (14.3%)
Constipation	1/15 (6.7%)		0/15 (0%)		1/21 (4.8%)
Nausea	2/15 (13.3%)		1/15 (6.7%)		2/21 (9.5%)
Vomiting	1/15 (6.7%)		2/15 (13.3%)		2/21 (9.5%)
Cheilitis	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Diarrhoea	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
General disorders
Face oedema	1/15 (6.7%)		0/15 (0%)		1/21 (4.8%)
Hyperthermia	1/15 (6.7%)		0/15 (0%)		1/21 (4.8%)
Oedema	0/15 (0%)		1/15 (6.7%)		1/21 (4.8%)
Pyrexia	0/15 (0%)		2/15 (13.3%)		2/21 (9.5%)
Injection site haemorrhage	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Bronchopneumopathy due to mycoplasm's infection	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Fever, rhynopharyngal infection, macular exenthema and Asthenia	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Ocular hypertony	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Uremic rectal bleeding (pratouayia)	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Immune system disorders
Hypersensitivity	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Infections and infestations
Bronchitis	1/15 (6.7%)		1/15 (6.7%)		1/21 (4.8%)
Diarrhoea infectious	1/15 (6.7%)		0/15 (0%)		1/21 (4.8%)
Ear infection	0/15 (0%)		3/15 (20%)		2/21 (9.5%)
Gastroenteritis	0/15 (0%)		2/15 (13.3%)		3/21 (14.3%)
Influenza	1/15 (6.7%)		1/15 (6.7%)		2/21 (9.5%)
Nasopharyngitis	0/15 (0%)		3/15 (20%)		2/21 (9.5%)
Pharyngitis	0/15 (0%)		1/15 (6.7%)		1/21 (4.8%)
Rhinitis	0/15 (0%)		3/15 (20%)		2/21 (9.5%)
Tracheobronchitis	0/15 (0%)		1/15 (6.7%)		1/21 (4.8%)
Viral infection	1/15 (6.7%)		0/15 (0%)		2/21 (9.5%)
Viral tracheitis	1/15 (6.7%)		0/15 (0%)		1/21 (4.8%)
Urinary tract infection	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Injury, poisoning and procedural complications
Joint sprain	2/15 (13.3%)		0/15 (0%)		0/21 (0%)
Radius fracture	0/15 (0%)		1/15 (6.7%)		1/21 (4.8%)
Hand fracture	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Ligament sprain	0/15 (0%)		0/15 (0%)		2/21 (9.5%)
Wound	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Investigations
Insulin-like growth factor increased	0/15 (0%)		3/15 (20%)		3/21 (14.3%)
Red blood cell sedimentation rate increased	5/15 (33.3%)		0/15 (0%)		4/21 (19%)
Blood human immunodeficiency virus (HIV) ribo-nucleic acid (RNA) increased	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Blood follicle stimulating hormone increased	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Weight increased	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Metabolism and nutrition disorders
Anorexia	0/15 (0%)		1/15 (6.7%)		0/21 (0%)
Fluid retention	0/15 (0%)		1/15 (6.7%)		0/21 (0%)
Hypercholesterolaemia	0/15 (0%)		2/15 (13.3%)		1/21 (4.8%)
Hyperinsulinaemia	1/15 (6.7%)		0/15 (0%)		1/21 (4.8%)
Hypertriglyceridaemia	0/15 (0%)		3/15 (20%)		1/21 (4.8%)
Malnutrition	1/15 (6.7%)		0/15 (0%)		0/21 (0%)
Sodium retention	0/15 (0%)		1/15 (6.7%)		0/21 (0%)
Decreased appetite	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Diabetes mellitus	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Glucose tolerance impaired	0/15 (0%)		0/15 (0%)		3/21 (14.3%)
Insulin resistance	0/15 (0%)		0/15 (0%)		4/21 (19%)
Mineral deficiency	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Vitamin D deficiency	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Musculoskeletal and connective tissue disorders
Arthralgia	1/15 (6.7%)		1/15 (6.7%)		4/21 (19%)
Joint effusion	4/15 (26.7%)		0/15 (0%)		4/21 (19%)
Juvenile arthritis	3/15 (20%)		0/15 (0%)		5/21 (23.8%)
Osteopenia	1/15 (6.7%)		0/15 (0%)		0/21 (0%)
Synovitis	1/15 (6.7%)		0/15 (0%)		1/21 (4.8%)
Amyotrophy	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Knee deformity	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Limb asymmetry	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Muscle atrophy	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Tendonitis	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Joint destruction	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Nervous system disorders
Headache	2/15 (13.3%)		3/15 (20%)		3/21 (14.3%)
Hypotonia	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Presyncope	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Psychiatric disorders
Depression	1/15 (6.7%)		0/15 (0%)		1/21 (4.8%)
Renal and urinary disorders
Dysuria	1/15 (6.7%)		0/15 (0%)		1/21 (4.8%)
Hypercalciuria	0/15 (0%)		1/15 (6.7%)		0/21 (0%)
Nephrotic syndrome	0/15 (0%)		11/15 (73.3%)		7/21 (33.3%)
Proteinuria	0/15 (0%)		6/15 (40%)		3/21 (14.3%)
Reproductive system and breast disorders
Ovarian cyst	0/15 (0%)		1/15 (6.7%)		1/21 (4.8%)
Respiratory, thoracic and mediastinal disorders
Cough	1/15 (6.7%)		0/15 (0%)		1/21 (4.8%)
Oropharyngeal pain	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Skin and subcutaneous tissue disorders
Acanthosis nigricans	0/15 (0%)		2/15 (13.3%)		1/21 (4.8%)
Acne	0/15 (0%)		0/15 (0%)		3/21 (14.3%)
Eczema	0/15 (0%)		0/15 (0%)		2/21 (9.5%)
Nail dystrophy	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Rash	0/15 (0%)		0/15 (0%)		1/21 (4.8%)
Vascular disorders
Haematoma	0/15 (0%)		1/15 (6.7%)		0/21 (0%)
Hypertension	0/15 (0%)		0/15 (0%)		2/21 (9.5%)

Limitations/Caveats

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.

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Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

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.