Outcome
| Type |
Measure |
Description |
Time frame |
Safety issue |
| Other |
Number of Participants With Treatment-Emergent Adverse Events (AEs) or Serious Adverse Events (SAEs) |
An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent are events between first dose of study drug and up to 28 days after last dose that were absent before treatment or that worsened relative to pretreatment state. AEs include both SAEs and non-SAEs. |
Baseline up to 28 days after last study treatment |
|
| Other |
Number of Participants With Significant Changes in Physical Examinations |
Number of participants with clinically significant physical examinations changes since previous visit were reported. Physical examination including estimation of pubertal stage and blood pressure measurement; |
Baseline, Month 12, 24, 36, 48, 60, End of Treatment (EOT) |
|
| Other |
Number of Participants With at Least 1 Medical or Surgical History |
|
Screening |
|
| Other |
Number of Participants Who Received Concomitant Medications |
|
Baseline up to Month 60 |
|
| Other |
Fasting Serum Insulin Like Growth Factor-1 (IGF-1) Levels |
|
Screening, Month 6, 12, 18, 24, 30, 36, 42, 48, 54, 60 |
|
| Other |
Fasting and Postprandial Plasma Glucose Levels at Month 12, 24, 36, 48 and 60 |
Fasting and 2 hours plasma glucose levels were assessed using standard oral glucose tolerance test (OGTT). |
Screening, Month 12, 24, 36, 48, 60 |
|
| Primary |
Change From Baseline in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Month 36: Full Analysis Population |
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; Yx refers to the value at particular timepoint x. 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. |
Baseline, Month 36 |
|
| Primary |
Change From Baseline in Height Standard Deviation Score (SD) for Chronological Age (CA) at Month 36: Per Protocol (PP) Population |
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; Yx refers to the value at particular timepoint x. 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. |
Baseline, Month 36 |
|
| Secondary |
Mean Height |
The standing height measurements were performed 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. |
Baseline, Month 12, 24, 36, 48, 60 |
|
| Secondary |
Mean Height Standard Deviation Score (SDS) for Bone Age (BA) |
The standing height measurements were performed 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 BA Yx = (height Yx - reference mean for BA Yx) / reference SD for BA Yx; Yx refers to the value at particular timepoint x. 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. |
Baseline, Month 12, 24, 36, 48, 60 |
|
| Secondary |
Annual Growth Rate (AGR) |
AGR at Yx was derived by subtracting AGR at baseline from Yx value. AGR was calculated each year and re scaled 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). Yx refers to the value at particular timepoint x. |
Baseline, Month 12, 24, 36, 48, 60 |
|
| Secondary |
Growth Rate (GR) Standard Deviation Score (SDS) for Bone Age (BA) |
GR SDS BA Yx = (GR Yx - reference mean for BA Yx) / reference SD for BA Yx; Yx refers to the value at particular timepoint x. GR in SDS was calculated using Sempe reference means and SD for GR. BA was estimated locally using an X-ray from the left wrist and hand. |
Month 12, 24, 36, 48, 60 |
|
| Secondary |
Growth Rate (GR) Standard Deviation Score (SDS) for Chronological Age (CA) |
GR SDS CA Yx = (GR Yx - reference mean for CA Yx) / reference SD for CA Yx; Yx refers to the value at particular timepoint x. GR in SDS was calculated using Sempe reference means and SD for GR. CA calculated as integer (Date of height measurement-Date of birth)/365.25*12. |
Baseline, Month 12, 24, 36, 48, 60 |
|
| Secondary |
Body Mass Index (BMI) |
BMI was calculated by weight divided by height squared and measured as kilogram per square meter (kg/m^2). |
Baseline, Month 12, 24, 36, 48, 60 |
|
| Secondary |
Change From Baseline in Height at Month 12, 24, 36, 48 and 60 |
The standing height measurements were performed 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. |
Baseline, Month 12, 24, 36, 48, 60 |
|
| Secondary |
Change From Baseline in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12, 24, 48 and 60 |
The standing height measurements were performed 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; Yx refers to the value at particular timepoint x. 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. |
Baseline, Month 12, 24, 48, 60 |
|
| Secondary |
Change From Baseline in Height Standard Deviation Score (SDS) for Bone Age (BA) at Month 12, 24, 36, 48 and 60 |
The standing height measurements were performed 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 BA Yx = (height Yx - reference mean for BA Yx) / reference SD for BA Yx; Yx refers to the value at particular timepoint x. 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. |
Baseline, Month 12, 24, 36, 48, 60 |
|
| Secondary |
Change From Baseline in Bone Age (BA) at Month 12, 24, 36, 48 and 60 |
BA was estimated locally using an X-ray from the left wrist and hand. |
Baseline, Month 12, 24, 36, 48, 60 |
|
| Secondary |
Ratio of Bone Age (BA) to Chronological Age (CA) |
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. |
Baseline, Month 12, 24, 36, 48, 60 |
|
