Trial Outcomes & Findings for Growth Retardation In Children With Special Pathological Conditions Or Disease (NCT NCT00163215)
NCT ID: NCT00163215
Last Updated: 2012-12-19
Results Overview
Change in annual growth rate (AGR) standard deviation score (SDS) for chronological age (CA) derived by subtracting AGR SDS CA at baseline from each time point (Yx) value. AGR at Yx= (height Yx-height Y\[x-1\])/(\[date of Yx-date of Y{x-1}\]/365.25). AGR as SDS calculated using Sempe reference means and standard deviations (SD) for growth rate. AGR SDS CA (for both baseline and Yx)= (AGR-reference mean for growth rate CA)/reference SD for growth rate CA. CA calculated as integer (Date of height measurement-Date of birth)/365.25\*12. SDS indicates how similar participant was to reference population.
COMPLETED
PHASE3
46 participants
Baseline, Month 36
2012-12-19
Participant Flow
Participant milestones
| Measure |
Genotonorm
Genotonorm (recombinant somatropin) up to maximum of 50 microgram/kilogram/day (mcg/kg/day) subcutaneously as decided by the investigator, divided in 7 daily doses for up to 3 years.
|
|---|---|
|
Overall Study
STARTED
|
46
|
|
Overall Study
COMPLETED
|
31
|
|
Overall Study
NOT COMPLETED
|
15
|
Reasons for withdrawal
| Measure |
Genotonorm
Genotonorm (recombinant somatropin) up to maximum of 50 microgram/kilogram/day (mcg/kg/day) subcutaneously as decided by the investigator, divided in 7 daily doses for up to 3 years.
|
|---|---|
|
Overall Study
Lack of Efficacy
|
3
|
|
Overall Study
Withdrawal by Subject
|
5
|
|
Overall Study
Other
|
7
|
Baseline Characteristics
Growth Retardation In Children With Special Pathological Conditions Or Disease
Baseline characteristics by cohort
| Measure |
Genotonorm
n=46 Participants
Genotonorm (recombinant somatropin) up to maximum of 50 microgram/kilogram/day (mcg/kg/day) subcutaneously as decided by the investigator, divided in 7 daily doses for up to 3 years.
|
|---|---|
|
Age Continuous
|
10.3 years
STANDARD_DEVIATION 3.6 • n=5 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
34 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, Month 36Population: Intent to Treat (ITT) set included all participants who received at least 1 dose of study medication and had at least 1 evaluation of height after start of study medication. Here 'N' (Number of participants analyzed) signifies those participants who were evaluable for this measure and "n" signifies participants evaluated at that time point.
Change in annual growth rate (AGR) standard deviation score (SDS) for chronological age (CA) derived by subtracting AGR SDS CA at baseline from each time point (Yx) value. AGR at Yx= (height Yx-height Y\[x-1\])/(\[date of Yx-date of Y{x-1}\]/365.25). AGR as SDS calculated using Sempe reference means and standard deviations (SD) for growth rate. AGR SDS CA (for both baseline and Yx)= (AGR-reference mean for growth rate CA)/reference SD for growth rate CA. CA calculated as integer (Date of height measurement-Date of birth)/365.25\*12. SDS indicates how similar participant was to reference population.
Outcome measures
| Measure |
Genotonorm
n=42 Participants
Genotonorm (recombinant somatropin) up to maximum of 50 microgram/kilogram/day (mcg/kg/day) subcutaneously as decided by the investigator, divided in 7 daily doses for up to 3 years.
|
|---|---|
|
Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 36 in Intent-to-Treat (ITT) Population
Baseline (n = 42)
|
-1.29 SDS
Standard Deviation 1.79
|
|
Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 36 in Intent-to-Treat (ITT) Population
Change at Month 36 (n = 26)
|
2.36 SDS
Standard Deviation 1.98
|
PRIMARY outcome
Timeframe: Baseline, Month 36Population: Per Protocol (PP) analysis set included all participants in the ITT set without a major protocol violation. Here 'N' (Number of participants analyzed) signifies those participants who were evaluable for this measure and "n" signifies participants evaluated at that time point.
Change in annual growth rate (AGR) standard deviation score (SDS) for chronological age (CA) derived by subtracting AGR SDS CA at baseline from each time point (Yx) value. AGR at Yx= (height Yx-height Y\[x-1\])/(\[date of Yx-date of Y{x-1}\]/365.25). AGR as SDS calculated using Sempe reference means and standard deviations (SD) for growth rate. AGR SDS CA (for both baseline and Yx)= (AGR-reference mean for growth rate CA)/reference SD for growth rate CA. CA calculated as integer (Date of height measurement-Date of birth)/365.25\*12. SDS indicates how similar participant was to reference population.
Outcome measures
| Measure |
Genotonorm
n=14 Participants
Genotonorm (recombinant somatropin) up to maximum of 50 microgram/kilogram/day (mcg/kg/day) subcutaneously as decided by the investigator, divided in 7 daily doses for up to 3 years.
|
|---|---|
|
Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 36 in Per-Protocol (PP) Population
Baseline (n = 14)
|
-1.98 SDS
Standard Deviation 1.06
|
|
Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 36 in Per-Protocol (PP) Population
Change at Month 36 (n = 12)
|
2.70 SDS
Standard Deviation 2.02
|
SECONDARY outcome
Timeframe: Baseline, Month 12, Month 24, Month 36Population: ITT set included all participants who received at least 1 dose of study medication and had at least 1 evaluation of height after start of study medication. Here 'N' (Number of participants analyzed) signifies those participants who were evaluable for this measure and "n" signifies participants evaluated at that time point.
Change in AGR at Yx was derived by subtracting AGR at baseline from Yx value. Annual growth rate was calculated each year and rescaled to 1 year if the interval between Yx and Y\[x-1\] was not 365 days, as long as a participant remained in the study. AGR at Yx was calculated using the previous height measurements (Y\[x-1\]) and height recorded at Yx (AGR Yx = \[height Yx-height Y{x-1}\] / (\[date of Yx - date of Y{x-1}\] /365.25).
Outcome measures
| Measure |
Genotonorm
n=42 Participants
Genotonorm (recombinant somatropin) up to maximum of 50 microgram/kilogram/day (mcg/kg/day) subcutaneously as decided by the investigator, divided in 7 daily doses for up to 3 years.
|
|---|---|
|
Change From Baseline in Annual Growth Rate at Month 12, Month 24 and Month 36 in ITT Population
Baseline (n = 42)
|
4.5 centimeter per year (cm/year)
Standard Deviation 1.8
|
|
Change From Baseline in Annual Growth Rate at Month 12, Month 24 and Month 36 in ITT Population
Change at Month 12 (n = 36)
|
3.3 centimeter per year (cm/year)
Standard Deviation 2.9
|
|
Change From Baseline in Annual Growth Rate at Month 12, Month 24 and Month 36 in ITT Population
Change at Month 24 (n = 35)
|
2.5 centimeter per year (cm/year)
Standard Deviation 2.7
|
|
Change From Baseline in Annual Growth Rate at Month 12, Month 24 and Month 36 in ITT Population
Change at Month 36 (n = 26)
|
2.0 centimeter per year (cm/year)
Standard Deviation 2.0
|
SECONDARY outcome
Timeframe: Baseline, Month 12, Month 24, Month 36Population: PP analysis set included all participants in the ITT set without a major protocol violation. Here 'N' (Number of participants analyzed) signifies those participants who were evaluable for this measure and "n" signifies participants evaluated at that time point.
Change in AGR at Yx was derived by subtracting AGR at baseline from Yx value. Annual growth rate was calculated each year and rescaled to 1 year if the interval between Yx and Y\[x-1\] was not 365 days, as long as a participant remained in the study. AGR at Yx was calculated using the previous height measurements (Y\[x-1\]) and height recorded at Yx (AGR Yx = \[height Yx-height Y{x-1}\] / (\[date of Yx - date of Y{x-1}\] /365.25).
Outcome measures
| Measure |
Genotonorm
n=14 Participants
Genotonorm (recombinant somatropin) up to maximum of 50 microgram/kilogram/day (mcg/kg/day) subcutaneously as decided by the investigator, divided in 7 daily doses for up to 3 years.
|
|---|---|
|
Change From Baseline in Annual Growth Rate at Month 12, Month 24, Month 36 in PP Population
Baseline (n = 14)
|
4.0 cm/year
Standard Deviation 1.0
|
|
Change From Baseline in Annual Growth Rate at Month 12, Month 24, Month 36 in PP Population
Change at Month 12 (n = 14)
|
4.0 cm/year
Standard Deviation 1.0
|
|
Change From Baseline in Annual Growth Rate at Month 12, Month 24, Month 36 in PP Population
Change at Month 24 (n = 14)
|
2.5 cm/year
Standard Deviation 1.3
|
|
Change From Baseline in Annual Growth Rate at Month 12, Month 24, Month 36 in PP Population
Change at Month 36 (n = 12)
|
1.8 cm/year
Standard Deviation 1.1
|
SECONDARY outcome
Timeframe: Baseline, Month 12, Month 24, Month 36Population: ITT set included all participants who received at least 1 dose of study medication and had at least 1 evaluation of height after start of study medication. Here "n" signifies those participants evaluated at that time point.
Height was measured using a wall mounted device (example, Harpenden stadiometer). The standing height of the participant was measured two times and the mean of these measurements was recorded.
Outcome measures
| Measure |
Genotonorm
n=44 Participants
Genotonorm (recombinant somatropin) up to maximum of 50 microgram/kilogram/day (mcg/kg/day) subcutaneously as decided by the investigator, divided in 7 daily doses for up to 3 years.
|
|---|---|
|
Height
Baseline (n = 44)
|
123.9 cm
Standard Deviation 17.3
|
|
Height
Month 24 (n = 39)
|
137.8 cm
Standard Deviation 16.9
|
|
Height
Month 36 (n = 27)
|
143.5 cm
Standard Deviation 15.8
|
|
Height
Month 12 (n = 39)
|
130.3 cm
Standard Deviation 17.5
|
SECONDARY outcome
Timeframe: Baseline, Month 12, Month 24, Month 36Population: ITT set included all participants who received at least 1 dose of study medication and had at least 1 evaluation of height after start of study medication. Here 'N' (Number of participants analyzed) signifies those participants who were evaluable for this measure and "n" signifies participants evaluated at that time point.
Height was measured using a wall mounted device (example, Harpenden stadiometer). The standing height of the participant was measured two times and the mean of these measurements was recorded.
Outcome measures
| Measure |
Genotonorm
n=39 Participants
Genotonorm (recombinant somatropin) up to maximum of 50 microgram/kilogram/day (mcg/kg/day) subcutaneously as decided by the investigator, divided in 7 daily doses for up to 3 years.
|
|---|---|
|
Change From Baseline in Height at Month 12, Month 24 and Month 36
Change at Month 12 (n = 39)
|
7.7 cm
Standard Deviation 2.1
|
|
Change From Baseline in Height at Month 12, Month 24 and Month 36
Change at Month 24 (n = 39)
|
14.9 cm
Standard Deviation 3.4
|
|
Change From Baseline in Height at Month 12, Month 24 and Month 36
Change at Month 36 (n = 27)
|
22.3 cm
Standard Deviation 3.7
|
SECONDARY outcome
Timeframe: Baseline, Month 12, Month 24, Month 36Population: ITT set included all participants who received at least 1 dose of study medication and had at least 1 evaluation of height after start of study medication. Here "n" signifies those participants evaluated at that time point.
Height was measured using a wall mounted device (example, Harpenden stadiometer). The standing height of the participant was measured two times and the mean of these measurements was recorded. Height SDS CA Yx = (height Yx - reference mean for CA Yx) / reference SD for CA Yx. Height in SDS was calculated using Sempe reference means and SD for height. CA calculated as integer (Date of height measurement-Date of birth)/365.25\*12. SDS indicates how similar the participant was to the reference population.
Outcome measures
| Measure |
Genotonorm
n=44 Participants
Genotonorm (recombinant somatropin) up to maximum of 50 microgram/kilogram/day (mcg/kg/day) subcutaneously as decided by the investigator, divided in 7 daily doses for up to 3 years.
|
|---|---|
|
Mean Height Standard Deviation Score (SDS) for Chronological Age (CA)
Baseline (n = 44)
|
-2.84 SDS
Standard Deviation 0.91
|
|
Mean Height Standard Deviation Score (SDS) for Chronological Age (CA)
Month 12 (n = 39)
|
-2.42 SDS
Standard Deviation 0.98
|
|
Mean Height Standard Deviation Score (SDS) for Chronological Age (CA)
Month 24 (n = 39)
|
-2.09 SDS
Standard Deviation 1.08
|
|
Mean Height Standard Deviation Score (SDS) for Chronological Age (CA)
Month 36 (n = 27)
|
-1.72 SDS
Standard Deviation 0.96
|
SECONDARY outcome
Timeframe: Baseline, Month 12, Month 24, Month 36Population: ITT set included all participants who received at least 1 dose of study medication and had at least 1 evaluation of height after start of study medication. Here 'N' (Number of participants analyzed) signifies those participants who were evaluable for this measure and "n" signifies participants evaluated at that time point.
Change in height SDS CA was derived by subtracting height SDS CA at baseline from Yx value. Height SDS CA (for both baseline and Yx) = (height - reference mean for CA)/reference SD for CA. Height in SDS was calculated using Sempe reference means and SD for height. CA calculated as integer (Date of height measurement - Date of birth)/365.25\*12. SDS indicates how similar the participant was to the reference population.
Outcome measures
| Measure |
Genotonorm
n=39 Participants
Genotonorm (recombinant somatropin) up to maximum of 50 microgram/kilogram/day (mcg/kg/day) subcutaneously as decided by the investigator, divided in 7 daily doses for up to 3 years.
|
|---|---|
|
Change From Baseline in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12, Month 24 and Month 36
Change at Month 36 (n = 27)
|
1.03 SDS
Standard Deviation 0.62
|
|
Change From Baseline in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12, Month 24 and Month 36
Change at Month 12 (n = 39)
|
0.41 SDS
Standard Deviation 0.42
|
|
Change From Baseline in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12, Month 24 and Month 36
Change at Month 24 (n = 39)
|
0.81 SDS
Standard Deviation 0.60
|
SECONDARY outcome
Timeframe: Baseline, Month 12, Month 24, Month 36Population: ITT set included all participants who received at least 1 dose of study medication and had at least 1 evaluation of height after start of study medication. Here 'N' (Number of participants analyzed) signifies those participants who were evaluable for this measure and "n" signifies participants evaluated at that time point.
Height SDS BA Yx = (height Yx - reference mean for BA Yx) / reference SD for BA Yx. Height in SDS was calculated using Sempe reference means and SD for height. BA was estimated locally using an X-ray from the left wrist and hand. SDS indicates how similar the participant was to the reference population.
Outcome measures
| Measure |
Genotonorm
n=35 Participants
Genotonorm (recombinant somatropin) up to maximum of 50 microgram/kilogram/day (mcg/kg/day) subcutaneously as decided by the investigator, divided in 7 daily doses for up to 3 years.
|
|---|---|
|
Mean Height Standard Deviation Score (SDS) for Bone Age (BA)
Baseline (n = 35)
|
-0.71 SDS
Standard Deviation 1.52
|
|
Mean Height Standard Deviation Score (SDS) for Bone Age (BA)
Month 12 (n = 32)
|
-0.55 SDS
Standard Deviation 1.68
|
|
Mean Height Standard Deviation Score (SDS) for Bone Age (BA)
Month 24 (n = 32)
|
-0.49 SDS
Standard Deviation 1.77
|
|
Mean Height Standard Deviation Score (SDS) for Bone Age (BA)
Month 36 (n = 20)
|
-0.30 SDS
Standard Deviation 1.46
|
SECONDARY outcome
Timeframe: Baseline, Month 12, Month 24, Month 36Population: ITT set included all participants who received at least 1 dose of study medication and had at least 1 evaluation of height after start of study medication. Here 'N' (Number of participants analyzed) signifies those participants who were evaluable for this measure and "n" signifies participants evaluated at that time point.
Change in height SDS BA was derived by subtracting height SDS BA at baseline from Yx value. Height SDS BA (for both baseline and Yx) = (height-reference mean for BA)/reference SD for BA. Height in SDS was calculated using Sempe reference means and SD for height. BA was estimated locally using an X-ray from the left wrist and hand. SDS indicates how similar the participant was to the reference population.
Outcome measures
| Measure |
Genotonorm
n=28 Participants
Genotonorm (recombinant somatropin) up to maximum of 50 microgram/kilogram/day (mcg/kg/day) subcutaneously as decided by the investigator, divided in 7 daily doses for up to 3 years.
|
|---|---|
|
Change From Baseline in Height Standard Deviation Score (SDS) for Bone Age (BA) at Month 12, Month 24 and Month 36
Change at Month 12 (n = 28)
|
-0.05 SDS
Standard Deviation 1.04
|
|
Change From Baseline in Height Standard Deviation Score (SDS) for Bone Age (BA) at Month 12, Month 24 and Month 36
Change at Month 24 (n = 27)
|
0.06 SDS
Standard Deviation 1.36
|
|
Change From Baseline in Height Standard Deviation Score (SDS) for Bone Age (BA) at Month 12, Month 24 and Month 36
Change at Month 36 (n = 17)
|
0.05 SDS
Standard Deviation 1.32
|
SECONDARY outcome
Timeframe: Baseline, Month 12, Month 24Population: ITT set included all participants who received at least 1 dose of study medication and had at least 1 evaluation of height after start of study medication. Here 'N' (Number of participants analyzed) signifies those participants who were evaluable for this measure.
Change in AGR SDS for CA derived by subtracting AGR SDS CA at baseline from Yx value. AGR at Yx= (height Yx-height Y\[x-1\])/(\[date of Yx-date of Y{x-1}\]/365.25). AGR as SDS calculated using Sempe reference means and standard deviations (SD) for growth rate. AGR SDS CA (for both baseline and Yx)= (AGR-reference mean for growth rate CA)/reference SD for growth rate CA. CA calculated as integer (Date of height measurement-Date of birth)/365.25\*12. SDS indicates how similar participant was to reference population.
Outcome measures
| Measure |
Genotonorm
n=36 Participants
Genotonorm (recombinant somatropin) up to maximum of 50 microgram/kilogram/day (mcg/kg/day) subcutaneously as decided by the investigator, divided in 7 daily doses for up to 3 years.
|
|---|---|
|
Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12 and Month 24 in ITT Population
Change at Month 12 (n = 36)
|
3.23 SDS
Standard Deviation 2.66
|
|
Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12 and Month 24 in ITT Population
Change at Month 24 (n = 35)
|
2.62 SDS
Standard Deviation 2.05
|
SECONDARY outcome
Timeframe: Baseline, Month 12, Month 24Population: PP analysis set included all participants in the ITT set without a major protocol violation. Here 'N' (Number of participants analyzed) signifies those participants who were evaluable for this measure.
Change in annual growth rate (AGR) standard deviation score (SDS) for chronological age (CA) derived by subtracting AGR SDS CA at baseline from each time point (Yx) value. AGR at Yx= (height Yx-height Y\[x-1\])/(\[date of Yx-date of Y{x-1}\]/365.25). AGR as SDS calculated using Sempe reference means and standard deviations (SD) for growth rate. AGR SDS CA (for both baseline and Yx)= (AGR-reference mean for growth rate CA)/reference SD for growth rate CA. CA calculated as integer (Date of height measurement-Date of birth)/365.25\*12. SDS indicates how similar participant was to reference population.
Outcome measures
| Measure |
Genotonorm
n=14 Participants
Genotonorm (recombinant somatropin) up to maximum of 50 microgram/kilogram/day (mcg/kg/day) subcutaneously as decided by the investigator, divided in 7 daily doses for up to 3 years.
|
|---|---|
|
Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12 and Month 24 in PP Population
Change at Month 12 (n = 14)
|
4.40 SDS
Standard Deviation 2.12
|
|
Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12 and Month 24 in PP Population
Change at Month 24 (n = 14)
|
2.97 SDS
Standard Deviation 1.68
|
SECONDARY outcome
Timeframe: Month 12, Month 24, Month 36Population: ITT set included all participants who received at least 1 dose of study medication and had at least 1 evaluation of height after start of study medication. Here 'N' (Number of participants analyzed) signifies those participants who were evaluable for this measure and "n" signifies participants evaluated at that time point.
AGR at Yx was derived by subtracting AGR at baseline from Yx value. AGR was calculated each year and rescaled to 1 year if the interval between Yx and Y\[x-1\] was not 365 days, as long as a participant remained in the study. AGR at Yx = \[height Yx-height Y{x-1}\] / (\[date of Yx - date of Y{x-1}\] /365.25). GR in SDS was calculated using Sempe reference means and SD for growth. BA was estimated locally using an X-ray from the left wrist and hand. SDS indicates how similar the participant was to the reference population.
Outcome measures
| Measure |
Genotonorm
n=28 Participants
Genotonorm (recombinant somatropin) up to maximum of 50 microgram/kilogram/day (mcg/kg/day) subcutaneously as decided by the investigator, divided in 7 daily doses for up to 3 years.
|
|---|---|
|
Mean Growth Rate Standard Deviation Score (SDS) for Bone Age (BA)
Month 12 (n = 28)
|
1.06 SDS
Standard Deviation 1.57
|
|
Mean Growth Rate Standard Deviation Score (SDS) for Bone Age (BA)
Month 24 (n = 25)
|
0.46 SDS
Standard Deviation 1.15
|
|
Mean Growth Rate Standard Deviation Score (SDS) for Bone Age (BA)
Month 36 (n = 19)
|
-0.01 SDS
Standard Deviation 1.19
|
SECONDARY outcome
Timeframe: Baseline, Month 12, Month 24, Month 36Population: ITT set included all participants who received at least 1 dose of study medication and had at least 1 evaluation of height after start of study medication. Here "n" signifies those participants evaluated at that time point.
BMI was used to measure body fat based on height and weight. It was calculated as body weight (kilogram) divided by the height (meter) squared.
Outcome measures
| Measure |
Genotonorm
n=44 Participants
Genotonorm (recombinant somatropin) up to maximum of 50 microgram/kilogram/day (mcg/kg/day) subcutaneously as decided by the investigator, divided in 7 daily doses for up to 3 years.
|
|---|---|
|
Body Mass Index (BMI)
Baseline (n = 44)
|
16.57 kilogram per square meter (kg/m^2)
Standard Deviation 2.37
|
|
Body Mass Index (BMI)
Month 12 (n = 39)
|
16.96 kilogram per square meter (kg/m^2)
Standard Deviation 2.64
|
|
Body Mass Index (BMI)
Month 24 (n = 39)
|
17.59 kilogram per square meter (kg/m^2)
Standard Deviation 2.69
|
|
Body Mass Index (BMI)
Month 36 (n = 27)
|
17.67 kilogram per square meter (kg/m^2)
Standard Deviation 2.08
|
SECONDARY outcome
Timeframe: Baseline, Month 12, Month 24, Month 36Population: ITT set included all participants who received at least 1 dose of study medication and had at least 1 evaluation of height after start of study medication. Here 'N' (Number of participants analyzed) signifies those participants who were evaluable for this measure and "n" signifies participants evaluated at that time point.
BMI was used to measure body fat based on height and weight. It was calculated as body weight (kilogram) divided by the height (meter) squared.
Outcome measures
| Measure |
Genotonorm
n=39 Participants
Genotonorm (recombinant somatropin) up to maximum of 50 microgram/kilogram/day (mcg/kg/day) subcutaneously as decided by the investigator, divided in 7 daily doses for up to 3 years.
|
|---|---|
|
Change From Baseline in Body Mass Index (BMI) at Month 12, Month 24 and Month 36
Change at Month 36 (n = 27)
|
1.89 kg/m^2
Standard Deviation 1.61
|
|
Change From Baseline in Body Mass Index (BMI) at Month 12, Month 24 and Month 36
Change at Month 12 (n = 39)
|
0.52 kg/m^2
Standard Deviation 1.01
|
|
Change From Baseline in Body Mass Index (BMI) at Month 12, Month 24 and Month 36
Change at Month 24 (n = 39)
|
1.30 kg/m^2
Standard Deviation 1.35
|
SECONDARY outcome
Timeframe: Baseline, Month 12, Month 24, Month 36Population: ITT set included all participants who received at least 1 dose of study medication and had at least 1 evaluation of height after start of study medication. Here 'N' (Number of participants analyzed) signifies those participants who were evaluable for this measure and "n" signifies participants evaluated at that time point.
BA was estimated locally using an X-ray from the left wrist and hand.
Outcome measures
| Measure |
Genotonorm
n=40 Participants
Genotonorm (recombinant somatropin) up to maximum of 50 microgram/kilogram/day (mcg/kg/day) subcutaneously as decided by the investigator, divided in 7 daily doses for up to 3 years.
|
|---|---|
|
Change From Baseline in Bone Age (BA) at Month 12, Month 24 and Month 36
Baseline (n = 40)
|
8.70 years
Standard Deviation 3.28
|
|
Change From Baseline in Bone Age (BA) at Month 12, Month 24 and Month 36
Change at Month 12 (n = 28)
|
1.69 years
Standard Deviation 0.90
|
|
Change From Baseline in Bone Age (BA) at Month 12, Month 24 and Month 36
Change at Month 24 (n = 28)
|
2.60 years
Standard Deviation 0.97
|
|
Change From Baseline in Bone Age (BA) at Month 12, Month 24 and Month 36
Change at Month 36 (n = 21)
|
4.04 years
Standard Deviation 1.09
|
SECONDARY outcome
Timeframe: Baseline, Month 12, Month 24, Month 36Population: ITT set included all participants who received at least 1 dose of study medication and had at least 1 evaluation of height after start of study medication. Here 'N' (Number of participants analyzed) signifies those participants who were evaluable for this measure and "n" signifies those participants evaluated at that time point.
BA was estimated locally using an X-ray from the left wrist and hand. CA at the date of corresponding X-ray (Date of X-ray - Date of birth)/365.25. Ratio of BA/CA at each annual study visit was calculated.
Outcome measures
| Measure |
Genotonorm
n=40 Participants
Genotonorm (recombinant somatropin) up to maximum of 50 microgram/kilogram/day (mcg/kg/day) subcutaneously as decided by the investigator, divided in 7 daily doses for up to 3 years.
|
|---|---|
|
Ratio of Bone Age (BA) to Chronological Age (CA)
Month 12 (n = 30)
|
0.83 ratio
Standard Deviation 0.15
|
|
Ratio of Bone Age (BA) to Chronological Age (CA)
Baseline (n = 40)
|
0.79 ratio
Standard Deviation 0.15
|
|
Ratio of Bone Age (BA) to Chronological Age (CA)
Month 24 (n = 32)
|
0.85 ratio
Standard Deviation 0.12
|
|
Ratio of Bone Age (BA) to Chronological Age (CA)
Month 36 (n = 23)
|
0.89 ratio
Standard Deviation 0.14
|
Adverse Events
Genotonorm
Serious adverse events
| Measure |
Genotonorm
n=46 participants at risk
Genotonorm (recombinant somatropin) up to maximum of 50 microgram/kilogram/day (mcg/kg/day) subcutaneously as decided by the investigator, divided in 7 daily doses for up to 3 years.
|
|---|---|
|
Congenital, familial and genetic disorders
Cryptorchism
|
2.2%
1/46
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.
|
|
Infections and infestations
Laryngitis
|
2.2%
1/46
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.
|
|
Infections and infestations
Staphylococcal infection
|
2.2%
1/46
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.
|
|
Infections and infestations
Staphylococcal sepsis
|
2.2%
1/46
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.
|
|
Infections and infestations
Viral infection
|
2.2%
1/46
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.
|
|
Injury, poisoning and procedural complications
Head injury
|
2.2%
1/46
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.
|
|
Investigations
Weight decreased
|
2.2%
1/46
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.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Histiocytosis haematophagic
|
2.2%
1/46
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.
|
Other adverse events
| Measure |
Genotonorm
n=46 participants at risk
Genotonorm (recombinant somatropin) up to maximum of 50 microgram/kilogram/day (mcg/kg/day) subcutaneously as decided by the investigator, divided in 7 daily doses for up to 3 years.
|
|---|---|
|
Gastrointestinal disorders
Diarrhoea
|
4.3%
2/46
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.
|
|
Infections and infestations
Bronchopulmonary aspergillosis allergic
|
2.2%
1/46
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.
|
|
Infections and infestations
Influenza
|
2.2%
1/46
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.
|
|
Infections and infestations
Laryngitis
|
2.2%
1/46
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.
|
|
Infections and infestations
Nasopharyngitis
|
2.2%
1/46
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.
|
|
Infections and infestations
Oral herpes
|
4.3%
2/46
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.
|
|
Infections and infestations
Tracheitis
|
2.2%
1/46
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.
|
|
Injury, poisoning and procedural complications
Joint injury
|
2.2%
1/46
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.
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
2.2%
1/46
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.
|
|
Investigations
Ammonia increased
|
2.2%
1/46
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.
|
|
Metabolism and nutrition disorders
Glucose tolerance impaired
|
2.2%
1/46
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.
|
|
Metabolism and nutrition disorders
Insulin resistance
|
2.2%
1/46
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.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
2.2%
1/46
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.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
2.2%
1/46
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.
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
2.2%
1/46
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.
|
|
Musculoskeletal and connective tissue disorders
Knee deformity
|
2.2%
1/46
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.
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
2.2%
1/46
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.
|
|
Musculoskeletal and connective tissue disorders
Rickets
|
2.2%
1/46
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.
|
|
Musculoskeletal and connective tissue disorders
Scoliosis
|
2.2%
1/46
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.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Melanocytic naevus
|
2.2%
1/46
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.
|
|
Nervous system disorders
Headache
|
10.9%
5/46
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.
|
|
Nervous system disorders
Loss of consciousness
|
2.2%
1/46
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.
|
|
Psychiatric disorders
Abnormal behaviour
|
2.2%
1/46
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.
|
|
Psychiatric disorders
Encopresis
|
2.2%
1/46
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.
|
|
Renal and urinary disorders
Enuresis
|
2.2%
1/46
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.
|
|
Renal and urinary disorders
Renal failure
|
2.2%
1/46
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.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
2.2%
1/46
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.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
2.2%
1/46
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.
|
|
Respiratory, thoracic and mediastinal disorders
Sleep apnoea syndrome
|
2.2%
1/46
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.
|
|
Skin and subcutaneous tissue disorders
Nail disorder
|
2.2%
1/46
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.
|
|
Skin and subcutaneous tissue disorders
Seborrhoeic dermatitis
|
2.2%
1/46
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.
|
|
Skin and subcutaneous tissue disorders
Skin irritation
|
2.2%
1/46
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.
|
|
Surgical and medical procedures
Ear tube insertion
|
2.2%
1/46
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.
|
|
Vascular disorders
Haematoma
|
2.2%
1/46
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.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER