Short Stature Born Small for Gestational Age (SGA)
Conditions
Keywords
Genotropin(somatropin), SGA, Short Stature, Long-term, Safety
Brief summary
To assess the long-term safety of Genotropin(somatropin) on Small for Gestational Age (SGA) without epiphyseal closing.
Interventions
DRUGGenotropin (somatropin)
Genotropin(somatoropin) 12 mg, Genotropin(somatropin) will be given at a dose of 0.067 mg/kg/day or 0.033 mg/kg/day to all participating patients
Sponsors
Pfizer
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
4 Years to 8 Years
Healthy volunteers
No
Inclusion criteria
Children with short stature due to SGA who received treatment in the study GENASG-0021-002.
Exclusion criteria
* Children who have any chronic disease requiring treatment with steroid hormone that may affect growth promotion including estrogen, androgen, anabolic hormone, and corticosteroids (except those for external use), and have received the treatment. * Children who have received radiotherapy or chemotherapy. * Children who have serious cardiac disease, renal disease, or hepatic disease. * Children who have diabetes mellitus with a manifestation of abnormal glucose metabolism. * Children who have serious chronic disease. * Children who have malignant tumor. * Children who are allergic to m-cresol.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | Month 12 (at the end of previous study) to 156 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Height Velocity Standard Deviation Score (SDS) for Chronological Age | Month 12 (at the end of previous study) to 156 | Height velocity is the yearly height gain. Height velocity SDS is calculated as following formula; Height velocity SDS = (height velocity - mean) / standard deviation, where mean and standard deviation were based on standard Japanese values of the participants age and gender. The scores were centred around zero. Negative score indicated a participant was smaller for their age/gender. |
| Height Velocity | Month 12 (at the end of previous study) to 156 | Height velocity is the yearly height gain |
| Height SDS for Chronological Age | Month 12 (at the end of previous study) to 156 | Height SDS is calculated as following formula; Height SDS = (height - mean) / standard deviation, where mean and standard deviation were based on standard Japanese values on the participant age and gender. The scores were centred around zero. Negative score indicated a participant was smaller for their age/gender. |
| Height Velocity SDS for Bone Age | Month 12 (at the end of previous study) to 156 | To measure bone age, X-ray images of the left hand were centrally assessed by an independent specialist using the Tanner-Whitehouse 2 (RUS) method standardized for Japanese children. Height velocity is the yearly height gain. Height velocity SDS for bone age is calculated as following formula; Height velocity SDS = (height velocity - mean) / standard deviation, where mean and standard deviation were based on standard Japanese values corresponding to bone age and gender. The scores were centred around zero. Negative score indicated a participant was smaller for their age/gender. |
| Height SDS for Bone Age | Month 12 (at the end of previous study) to 156 | To measure bone age, X-ray images of the left hand were centrally assessed by an independent specialist using the Tanner-Whitehouse 2 (RUS) method standardized for Japanese children. Height SDS for bone age is calculated as following formula; Height SDS = (height - mean) / standard deviation, where mean and standard deviation were based on standard Japanese values corresponding to bone age and gender. The scores were centred around zero. Negative score indicated a participant was smaller for their age/gender. |
Countries
Japan
Participant flow
Pre-assignment details
Before enrolled this study, participants with short stature due to SGA had completed the 1-year(12-month) treatment in previous study. Out of 62 participants who were enrolled this study, 61 participants were treated.
Participants by arm
| Arm | Count |
|---|---|
| Dose-Increasing Group Participants who were treated with somatropin 0.033 mg/kg/day in previous study for 12 months received a dose of 0.067 mg/kg/day | 29 |
| Dose-Remaining Group Participants who were treated with somatropin 0.067 mg/kg/day in previous study for 12 months were maintained on the same dose | 32 |
| Total | 61 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse Event | 0 | 1 |
| Overall Study | Family Matters | 0 | 1 |
| Overall Study | Physician Decision | 2 | 4 |
| Overall Study | Protocol Violation | 1 | 1 |
| Overall Study | Withdrawal by Subject | 11 | 10 |
Baseline characteristics
| Characteristic | Dose-Increasing Group | Dose-Remaining Group | Total |
|---|---|---|---|
| Age, Continuous | 5.20 years STANDARD_DEVIATION 1.64 | 5.40 years STANDARD_DEVIATION 1.27 | 5.31 years STANDARD_DEVIATION 1.45 |
| Sex: Female, Male Female | 14 Participants | 14 Participants | 28 Participants |
| Sex: Female, Male Male | 15 Participants | 18 Participants | 33 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 27 / 29 | 31 / 32 |
| serious Total, serious adverse events | 10 / 29 | 5 / 32 |
Outcome results
Primary
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Month 12 (at the end of previous study) to 156
Population: Full Analysis Set (FAS) included participants who received at least 1 dose of study medication and had at least one observation after enrollment of this study.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Dose-Increasing Group | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | AE | 27 participant |
| Dose-Increasing Group | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | SAE | 10 participant |
| Dose-Remaining Group | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | AE | 31 participant |
| Dose-Remaining Group | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | SAE | 5 participant |
Secondary
Height SDS for Bone Age
To measure bone age, X-ray images of the left hand were centrally assessed by an independent specialist using the Tanner-Whitehouse 2 (RUS) method standardized for Japanese children. Height SDS for bone age is calculated as following formula; Height SDS = (height - mean) / standard deviation, where mean and standard deviation were based on standard Japanese values corresponding to bone age and gender. The scores were centred around zero. Negative score indicated a participant was smaller for their age/gender.
Time frame: Month 12 (at the end of previous study) to 156
Population: Full Analysis Set (FAS) included participants who received at least 1 dose of study medication and had at least one observation after enrollment of this study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Dose-Increasing Group | Height SDS for Bone Age | Month 36 (Increasing: n=24, Remaining: n=27) | -1.20 SDS | Standard Deviation 1.35 |
| Dose-Increasing Group | Height SDS for Bone Age | Month 96 (Increasing: n=10, Remaining: n=13) | -1.77 SDS | Standard Deviation 1.12 |
| Dose-Increasing Group | Height SDS for Bone Age | Month 60 (Increasing: n=20, Remaining: n=19) | -1.16 SDS | Standard Deviation 1.21 |
| Dose-Increasing Group | Height SDS for Bone Age | Month 108 (Increasing: n=8, Remaining: n=7) | -1.41 SDS | Standard Deviation 0.97 |
| Dose-Increasing Group | Height SDS for Bone Age | Month 24 (Increasing: n=26, Remaining: n=31) | -1.15 SDS | Standard Deviation 1.15 |
| Dose-Increasing Group | Height SDS for Bone Age | Month 120 (Increasing: n=5, Remaining: n=5) | -1.58 SDS | Standard Deviation 1.5 |
| Dose-Increasing Group | Height SDS for Bone Age | Month 72 (Increasing: n=16, Remaining: n=14) | -1.78 SDS | Standard Deviation 1.02 |
| Dose-Increasing Group | Height SDS for Bone Age | Month 132 (Increasing: n=4, Remaining: n=4) | -2.10 SDS | Standard Deviation 1.21 |
| Dose-Increasing Group | Height SDS for Bone Age | Month 48 (Increasing: n=22, Remaining: n=22) | -0.74 SDS | Standard Deviation 1.32 |
| Dose-Increasing Group | Height SDS for Bone Age | Month 144 (Increasing: n=3, Remaining: n=2) | -1.40 SDS | Standard Deviation 0.17 |
| Dose-Increasing Group | Height SDS for Bone Age | Month 84 (Increasing: n=13, Remaining: n=13) | -1.85 SDS | Standard Deviation 1.17 |
| Dose-Increasing Group | Height SDS for Bone Age | Month 156 (Increasing: n=2, Remaining: n=0) | -1.55 SDS | Standard Deviation 0.92 |
| Dose-Increasing Group | Height SDS for Bone Age | Month 12 (Increasing: n=27, Remaining: n=31) | -1.19 SDS | Standard Deviation 1.2 |
| Dose-Remaining Group | Height SDS for Bone Age | Month 156 (Increasing: n=2, Remaining: n=0) | NA SDS | — |
| Dose-Remaining Group | Height SDS for Bone Age | Month 12 (Increasing: n=27, Remaining: n=31) | -0.68 SDS | Standard Deviation 1.54 |
| Dose-Remaining Group | Height SDS for Bone Age | Month 24 (Increasing: n=26, Remaining: n=31) | -0.88 SDS | Standard Deviation 1.79 |
| Dose-Remaining Group | Height SDS for Bone Age | Month 36 (Increasing: n=24, Remaining: n=27) | -1.17 SDS | Standard Deviation 1.62 |
| Dose-Remaining Group | Height SDS for Bone Age | Month 48 (Increasing: n=22, Remaining: n=22) | -1.46 SDS | Standard Deviation 1.01 |
| Dose-Remaining Group | Height SDS for Bone Age | Month 60 (Increasing: n=20, Remaining: n=19) | -1.80 SDS | Standard Deviation 0.97 |
| Dose-Remaining Group | Height SDS for Bone Age | Month 72 (Increasing: n=16, Remaining: n=14) | -1.70 SDS | Standard Deviation 0.77 |
| Dose-Remaining Group | Height SDS for Bone Age | Month 84 (Increasing: n=13, Remaining: n=13) | -2.15 SDS | Standard Deviation 0.92 |
| Dose-Remaining Group | Height SDS for Bone Age | Month 96 (Increasing: n=10, Remaining: n=13) | -2.27 SDS | Standard Deviation 1.06 |
| Dose-Remaining Group | Height SDS for Bone Age | Month 108 (Increasing: n=8, Remaining: n=7) | -1.94 SDS | Standard Deviation 1.37 |
| Dose-Remaining Group | Height SDS for Bone Age | Month 120 (Increasing: n=5, Remaining: n=5) | -1.38 SDS | Standard Deviation 0.98 |
| Dose-Remaining Group | Height SDS for Bone Age | Month 132 (Increasing: n=4, Remaining: n=4) | -0.98 SDS | Standard Deviation 0.74 |
| Dose-Remaining Group | Height SDS for Bone Age | Month 144 (Increasing: n=3, Remaining: n=2) | -0.65 SDS | Standard Deviation 0.64 |
Secondary
Height SDS for Chronological Age
Height SDS is calculated as following formula; Height SDS = (height - mean) / standard deviation, where mean and standard deviation were based on standard Japanese values on the participant age and gender. The scores were centred around zero. Negative score indicated a participant was smaller for their age/gender.
Time frame: Month 12 (at the end of previous study) to 156
Population: Full Analysis Set (FAS) included participants who received at least 1 dose of study medication and had at least one observation after enrollment of this study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Dose-Increasing Group | Height SDS for Chronological Age | Month 156 (Increasing: n=3, Remaining: n=0) | -1.77 SDS | Standard Deviation 0.76 |
| Dose-Increasing Group | Height SDS for Chronological Age | Month 12 (Increasing: n=29, Remaining: n=32) | -2.53 SDS | Standard Deviation 0.92 |
| Dose-Increasing Group | Height SDS for Chronological Age | Month 24 (Increasing: n=28, Remaining: n=32) | -2.02 SDS | Standard Deviation 0.97 |
| Dose-Increasing Group | Height SDS for Chronological Age | Month 36 (Increasing: n=26, Remaining: n=28) | -1.80 SDS | Standard Deviation 0.99 |
| Dose-Increasing Group | Height SDS for Chronological Age | Month 48 (Increasing: n=24, Remaining: n=23) | -1.48 SDS | Standard Deviation 1.05 |
| Dose-Increasing Group | Height SDS for Chronological Age | Month 60 (Increasing: n=21, Remaining: n=20) | -1.53 SDS | Standard Deviation 1.06 |
| Dose-Increasing Group | Height SDS for Chronological Age | Month 72 (Increasing: n=20, Remaining: n=16) | -1.56 SDS | Standard Deviation 1.11 |
| Dose-Increasing Group | Height SDS for Chronological Age | Month 84 (Increasing: n=15, Remaining: n=16) | -1.73 SDS | Standard Deviation 1.13 |
| Dose-Increasing Group | Height SDS for Chronological Age | Month 96 (Increasing: n=11, Remaining: n=14) | -1.52 SDS | Standard Deviation 0.89 |
| Dose-Increasing Group | Height SDS for Chronological Age | Month 108 (Increasing: n=9, Remaining: n=8) | -1.52 SDS | Standard Deviation 1.01 |
| Dose-Increasing Group | Height SDS for Chronological Age | Month 120 (Increasing: n=6, Remaining: n=6) | -1.52 SDS | Standard Deviation 1.2 |
| Dose-Increasing Group | Height SDS for Chronological Age | Month 132 (Increasing: n=5, Remaining: n=4) | -1.96 SDS | Standard Deviation 1.11 |
| Dose-Increasing Group | Height SDS for Chronological Age | Month 144 (Increasing: n=4, Remaining: n=2) | -1.73 SDS | Standard Deviation 0.87 |
| Dose-Remaining Group | Height SDS for Chronological Age | Month 120 (Increasing: n=6, Remaining: n=6) | -1.25 SDS | Standard Deviation 0.59 |
| Dose-Remaining Group | Height SDS for Chronological Age | Month 84 (Increasing: n=15, Remaining: n=16) | -1.58 SDS | Standard Deviation 1.17 |
| Dose-Remaining Group | Height SDS for Chronological Age | Month 12 (Increasing: n=29, Remaining: n=32) | -2.17 SDS | Standard Deviation 0.96 |
| Dose-Remaining Group | Height SDS for Chronological Age | Month 144 (Increasing: n=4, Remaining: n=2) | -0.70 SDS | Standard Deviation 0.42 |
| Dose-Remaining Group | Height SDS for Chronological Age | Month 24 (Increasing: n=28, Remaining: n=32) | -1.70 SDS | Standard Deviation 1.03 |
| Dose-Remaining Group | Height SDS for Chronological Age | Month 96 (Increasing: n=11, Remaining: n=14) | -1.87 SDS | Standard Deviation 1.36 |
| Dose-Remaining Group | Height SDS for Chronological Age | Month 36 (Increasing: n=26, Remaining: n=28) | -1.53 SDS | Standard Deviation 1.1 |
| Dose-Remaining Group | Height SDS for Chronological Age | Month 132 (Increasing: n=5, Remaining: n=4) | -0.98 SDS | Standard Deviation 0.51 |
| Dose-Remaining Group | Height SDS for Chronological Age | Month 48 (Increasing: n=24, Remaining: n=23) | -1.49 SDS | Standard Deviation 1.15 |
| Dose-Remaining Group | Height SDS for Chronological Age | Month 108 (Increasing: n=9, Remaining: n=8) | -1.63 SDS | Standard Deviation 1.48 |
| Dose-Remaining Group | Height SDS for Chronological Age | Month 60 (Increasing: n=21, Remaining: n=20) | -1.44 SDS | Standard Deviation 1.1 |
| Dose-Remaining Group | Height SDS for Chronological Age | Month 156 (Increasing: n=3, Remaining: n=0) | NA SDS | — |
| Dose-Remaining Group | Height SDS for Chronological Age | Month 72 (Increasing: n=20, Remaining: n=16) | -1.43 SDS | Standard Deviation 1.06 |
Secondary
Height Velocity
Height velocity is the yearly height gain
Time frame: Month 12 (at the end of previous study) to 156
Population: Full Analysis Set (FAS) included participants who received at least 1 dose of study medication and had at least one observation after enrollment of this study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Dose-Increasing Group | Height Velocity | Month 72 to 84 (Increasing: n=15, Remaining: n=16) | 4.89 cm/year | Standard Deviation 2.18 |
| Dose-Increasing Group | Height Velocity | Month 12 to 24 (Increasing: n=28, Remaining: n=32) | 7.83 cm/year | Standard Deviation 1.33 |
| Dose-Increasing Group | Height Velocity | Month 24 to 36 (Increasing: n=26, Remaining: n=28) | 6.88 cm/year | Standard Deviation 0.94 |
| Dose-Increasing Group | Height Velocity | Month 36 to 48 (Increasing: n=24, Remaining: n=23) | 6.68 cm/year | Standard Deviation 0.98 |
| Dose-Increasing Group | Height Velocity | Month 48 to 60 (Increasing: n=21, Remaining: n=20) | 6.06 cm/year | Standard Deviation 1.49 |
| Dose-Increasing Group | Height Velocity | Month 60 to 72 (Increasing: n=20, Remaining: n=16) | 6.08 cm/year | Standard Deviation 1.45 |
| Dose-Increasing Group | Height Velocity | Month 84 to 96 (Increasing: n=11, Remaining: n=14) | 5.16 cm/year | Standard Deviation 1.2 |
| Dose-Increasing Group | Height Velocity | Month 96 to 108 (Increasing: n=9, Remaining: n=8) | 5.18 cm/year | Standard Deviation 2.16 |
| Dose-Increasing Group | Height Velocity | Month 108 to 120 (Increasing: n=6, Remaining: n=6) | 5.72 cm/year | Standard Deviation 2.24 |
| Dose-Increasing Group | Height Velocity | Month 120 to 132 (Increasing: n=5, Remaining: n=4) | 4.84 cm/year | Standard Deviation 1.3 |
| Dose-Increasing Group | Height Velocity | Month 132 to 144 (Increasing: n=4, Remaining: n=2) | 4.38 cm/year | Standard Deviation 1.1 |
| Dose-Increasing Group | Height Velocity | Month 144 to 156(Increasing: n=3, Remaining: n=0) | 3.10 cm/year | Standard Deviation 1.92 |
| Dose-Remaining Group | Height Velocity | Month 132 to 144 (Increasing: n=4, Remaining: n=2) | 2.60 cm/year | Standard Deviation 0.14 |
| Dose-Remaining Group | Height Velocity | Month 84 to 96 (Increasing: n=11, Remaining: n=14) | 4.82 cm/year | Standard Deviation 2.12 |
| Dose-Remaining Group | Height Velocity | Month 12 to 24 (Increasing: n=28, Remaining: n=32) | 7.70 cm/year | Standard Deviation 1.19 |
| Dose-Remaining Group | Height Velocity | Month 120 to 132 (Increasing: n=5, Remaining: n=4) | 4.30 cm/year | Standard Deviation 1.84 |
| Dose-Remaining Group | Height Velocity | Month 24 to 36 (Increasing: n=26, Remaining: n=28) | 6.75 cm/year | Standard Deviation 1.52 |
| Dose-Remaining Group | Height Velocity | Month 96 to 108 (Increasing: n=9, Remaining: n=8) | 5.55 cm/year | Standard Deviation 2.42 |
| Dose-Remaining Group | Height Velocity | Month 36 to 48 (Increasing: n=24, Remaining: n=23) | 6.08 cm/year | Standard Deviation 1.14 |
| Dose-Remaining Group | Height Velocity | Month 144 to 156(Increasing: n=3, Remaining: n=0) | NA cm/year | — |
| Dose-Remaining Group | Height Velocity | Month 48 to 60 (Increasing: n=21, Remaining: n=20) | 6.30 cm/year | Standard Deviation 1.24 |
| Dose-Remaining Group | Height Velocity | Month 108 to 120 (Increasing: n=6, Remaining: n=6) | 5.03 cm/year | Standard Deviation 1.86 |
| Dose-Remaining Group | Height Velocity | Month 60 to 72 (Increasing: n=20, Remaining: n=16) | 6.49 cm/year | Standard Deviation 1.37 |
| Dose-Remaining Group | Height Velocity | Month 72 to 84 (Increasing: n=15, Remaining: n=16) | 4.88 cm/year | Standard Deviation 1.86 |
Secondary
Height Velocity SDS for Bone Age
To measure bone age, X-ray images of the left hand were centrally assessed by an independent specialist using the Tanner-Whitehouse 2 (RUS) method standardized for Japanese children. Height velocity is the yearly height gain. Height velocity SDS for bone age is calculated as following formula; Height velocity SDS = (height velocity - mean) / standard deviation, where mean and standard deviation were based on standard Japanese values corresponding to bone age and gender. The scores were centred around zero. Negative score indicated a participant was smaller for their age/gender.
Time frame: Month 12 (at the end of previous study) to 156
Population: Full Analysis Set (FAS) included participants who received at least 1 dose of study medication and had at least one observation after enrollment of this study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Dose-Increasing Group | Height Velocity SDS for Bone Age | Month 24 to 36 (Increasing: n=24, Remaining: n=27) | 1.503 SDS | Standard Deviation 1.83 |
| Dose-Increasing Group | Height Velocity SDS for Bone Age | Month 84 to 96 (Increasing: n=10, Remaining: n=13) | 0.804 SDS | Standard Deviation 3.44 |
| Dose-Increasing Group | Height Velocity SDS for Bone Age | Month 48 to 60 (Increasing: n=20, Remaining: n=19) | -0.062 SDS | Standard Deviation 1.775 |
| Dose-Increasing Group | Height Velocity SDS for Bone Age | Month 96 to 108 (Increasing: n=8, Remaining: n=7) | -0.634 SDS | Standard Deviation 2.419 |
| Dose-Increasing Group | Height Velocity SDS for Bone Age | Month 12 to 24 (Increasing: n=26, Remaining: n=31) | 2.586 SDS | Standard Deviation 2.268 |
| Dose-Increasing Group | Height Velocity SDS for Bone Age | Month 108 to 120 (Increasing: n=5, Remaining: n=5) | 0.070 SDS | Standard Deviation 4.12 |
| Dose-Increasing Group | Height Velocity SDS for Bone Age | Month 60 to 72 (Increasing: n=16, Remaining: n=14) | 0.281 SDS | Standard Deviation 2.814 |
| Dose-Increasing Group | Height Velocity SDS for Bone Age | Month 120 to 132 (Increasing: n=4, Remaining: n=4) | 1.553 SDS | Standard Deviation 3.019 |
| Dose-Increasing Group | Height Velocity SDS for Bone Age | Month 36 to 48 (Increasing: n=22, Remaining: n=22) | 1.196 SDS | Standard Deviation 1.409 |
| Dose-Increasing Group | Height Velocity SDS for Bone Age | Month 132 to 144 (Increasing: n=3, Remaining: n=2) | 2.310 SDS | Standard Deviation 1.711 |
| Dose-Increasing Group | Height Velocity SDS for Bone Age | Month 144 to 156 (Increasing: n=2, Remaining: n=0) | 2.710 SDS | Standard Deviation 2.942 |
| Dose-Increasing Group | Height Velocity SDS for Bone Age | Month 72 to 84 (Increasing: n=13, Remaining: n=13) | -1.249 SDS | Standard Deviation 2.558 |
| Dose-Remaining Group | Height Velocity SDS for Bone Age | Month 144 to 156 (Increasing: n=2, Remaining: n=0) | NA SDS | — |
| Dose-Remaining Group | Height Velocity SDS for Bone Age | Month 12 to 24 (Increasing: n=26, Remaining: n=31) | 2.461 SDS | Standard Deviation 1.99 |
| Dose-Remaining Group | Height Velocity SDS for Bone Age | Month 24 to 36 (Increasing: n=24, Remaining: n=27) | 1.091 SDS | Standard Deviation 1.852 |
| Dose-Remaining Group | Height Velocity SDS for Bone Age | Month 36 to 48 (Increasing: n=22, Remaining: n=22) | 0.510 SDS | Standard Deviation 1.809 |
| Dose-Remaining Group | Height Velocity SDS for Bone Age | Month 48 to 60 (Increasing: n=20, Remaining: n=19) | 0.913 SDS | Standard Deviation 2.146 |
| Dose-Remaining Group | Height Velocity SDS for Bone Age | Month 60 to 72 (Increasing: n=16, Remaining: n=14) | 0.949 SDS | Standard Deviation 2.729 |
| Dose-Remaining Group | Height Velocity SDS for Bone Age | Month 72 to 84 (Increasing: n=13, Remaining: n=13) | 0.932 SDS | Standard Deviation 2.545 |
| Dose-Remaining Group | Height Velocity SDS for Bone Age | Month 84 to 96 (Increasing: n=10, Remaining: n=13) | 0.286 SDS | Standard Deviation 2.351 |
| Dose-Remaining Group | Height Velocity SDS for Bone Age | Month 96 to 108 (Increasing: n=8, Remaining: n=7) | -0.223 SDS | Standard Deviation 1.352 |
| Dose-Remaining Group | Height Velocity SDS for Bone Age | Month 108 to 120 (Increasing: n=5, Remaining: n=5) | 0.532 SDS | Standard Deviation 1.809 |
| Dose-Remaining Group | Height Velocity SDS for Bone Age | Month 120 to 132 (Increasing: n=4, Remaining: n=4) | 2.718 SDS | Standard Deviation 0.864 |
| Dose-Remaining Group | Height Velocity SDS for Bone Age | Month 132 to 144 (Increasing: n=3, Remaining: n=2) | 2.185 SDS | Standard Deviation 0.304 |
Secondary
Height Velocity Standard Deviation Score (SDS) for Chronological Age
Height velocity is the yearly height gain. Height velocity SDS is calculated as following formula; Height velocity SDS = (height velocity - mean) / standard deviation, where mean and standard deviation were based on standard Japanese values of the participants age and gender. The scores were centred around zero. Negative score indicated a participant was smaller for their age/gender.
Time frame: Month 12 (at the end of previous study) to 156
Population: Full Analysis Set (FAS) included participants who received at least 1 dose of study medication and had at least one observation after enrollment of this study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Dose-Increasing Group | Height Velocity Standard Deviation Score (SDS) for Chronological Age | Month 12 to 24 (Increasing: n=28, Remaining: n=32) | 2.782 SDS | Standard Deviation 1.978 |
| Dose-Increasing Group | Height Velocity Standard Deviation Score (SDS) for Chronological Age | Month 24 to36 (Increasing:n=26, Remaining:n=28) | 1.812 SDS | Standard Deviation 1.526 |
| Dose-Increasing Group | Height Velocity Standard Deviation Score (SDS) for Chronological Age | Month 36 to 48 (Increasing: n=24, Remaining: n=23) | 1.480 SDS | Standard Deviation 1.543 |
| Dose-Increasing Group | Height Velocity Standard Deviation Score (SDS) for Chronological Age | Month 48 to 60 (Increasing: n=21, Remaining: n=20) | -0.041 SDS | Standard Deviation 2.081 |
| Dose-Increasing Group | Height Velocity Standard Deviation Score (SDS) for Chronological Age | Month 60 to 72 (Increasing: n=20, Remaining: n=16) | -0.293 SDS | Standard Deviation 1.585 |
| Dose-Increasing Group | Height Velocity Standard Deviation Score (SDS) for Chronological Age | Month 72 to 84 (Increasing: n=15, Remaining: n=16) | -0.488 SDS | Standard Deviation 3.117 |
| Dose-Increasing Group | Height Velocity Standard Deviation Score (SDS) for Chronological Age | Month 84 to 96 (Increasing: n=11, Remaining: n=14) | 0.263 SDS | Standard Deviation 1.802 |
| Dose-Increasing Group | Height Velocity Standard Deviation Score (SDS) for Chronological Age | Month 96 to 108 (Increasing: n=9, Remaining: n=8) | 0.521 SDS | Standard Deviation 2.058 |
| Dose-Increasing Group | Height Velocity Standard Deviation Score (SDS) for Chronological Age | Month 108 to 120 (Increasing: n=6, Remaining: n=6) | -0.668 SDS | Standard Deviation 2.126 |
| Dose-Increasing Group | Height Velocity Standard Deviation Score (SDS) for Chronological Age | Month 120 to 132 (Increasing: n=5, Remaining: n=4) | -1.080 SDS | Standard Deviation 1.953 |
| Dose-Increasing Group | Height Velocity Standard Deviation Score (SDS) for Chronological Age | Month 132 to 144 (Increasing: n=4, Remaining: n=2) | 2.655 SDS | Standard Deviation 4.329 |
| Dose-Increasing Group | Height Velocity Standard Deviation Score (SDS) for Chronological Age | Month 144 to 156 (Increasing: n=3, Remaining: n=0) | 3.373 SDS | Standard Deviation 1.995 |
| Dose-Remaining Group | Height Velocity Standard Deviation Score (SDS) for Chronological Age | Month 132 to 144 (Increasing: n=4, Remaining: n=2) | 0.730 SDS | Standard Deviation 2.022 |
| Dose-Remaining Group | Height Velocity Standard Deviation Score (SDS) for Chronological Age | Month 12 to 24 (Increasing: n=28, Remaining: n=32) | 2.595 SDS | Standard Deviation 1.731 |
| Dose-Remaining Group | Height Velocity Standard Deviation Score (SDS) for Chronological Age | Month 84 to 96 (Increasing: n=11, Remaining: n=14) | -0.114 SDS | Standard Deviation 1.964 |
| Dose-Remaining Group | Height Velocity Standard Deviation Score (SDS) for Chronological Age | Month 24 to36 (Increasing:n=26, Remaining:n=28) | 1.696 SDS | Standard Deviation 2.111 |
| Dose-Remaining Group | Height Velocity Standard Deviation Score (SDS) for Chronological Age | Month 120 to 132 (Increasing: n=5, Remaining: n=4) | 1.173 SDS | Standard Deviation 3.042 |
| Dose-Remaining Group | Height Velocity Standard Deviation Score (SDS) for Chronological Age | Month 36 to 48 (Increasing: n=24, Remaining: n=23) | 0.824 SDS | Standard Deviation 1.527 |
| Dose-Remaining Group | Height Velocity Standard Deviation Score (SDS) for Chronological Age | Month 96 to 108 (Increasing: n=9, Remaining: n=8) | -0.466 SDS | Standard Deviation 2.055 |
| Dose-Remaining Group | Height Velocity Standard Deviation Score (SDS) for Chronological Age | Month 48 to 60 (Increasing: n=21, Remaining: n=20) | 0.480 SDS | Standard Deviation 1.651 |
| Dose-Remaining Group | Height Velocity Standard Deviation Score (SDS) for Chronological Age | Month 144 to 156 (Increasing: n=3, Remaining: n=0) | NA SDS | — |
| Dose-Remaining Group | Height Velocity Standard Deviation Score (SDS) for Chronological Age | Month 60 to 72 (Increasing: n=20, Remaining: n=16) | -0.046 SDS | Standard Deviation 2.434 |
| Dose-Remaining Group | Height Velocity Standard Deviation Score (SDS) for Chronological Age | Month 108 to 120 (Increasing: n=6, Remaining: n=6) | -0.590 SDS | Standard Deviation 2.693 |
| Dose-Remaining Group | Height Velocity Standard Deviation Score (SDS) for Chronological Age | Month 72 to 84 (Increasing: n=15, Remaining: n=16) | -1.511 SDS | Standard Deviation 2.692 |