To show an increase in annual growth rate 3 years after Visit 2. Annual growth rate in standard deviation (SD) after 3 years will be compared to growth rate before the start of GH treatment.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
46
Adapted dosage based on IGF 1 level and weight Form: liquid; Dosage and Frequency: from 0.0033mg/kg/day to 0.0067 mg/kg/day; Duration: 3 years
Pfizer Investigational Site
Annemasse, France
Pfizer Investigational Site
Besançon, France
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 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.
Time frame: Baseline, Month 36
Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 36 in Per-Protocol (PP) Population
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.
Time frame: Baseline, Month 36
Change From Baseline in Annual Growth Rate at Month 12, Month 24 and Month 36 in ITT Population
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).
Time frame: Baseline, Month 12, Month 24, Month 36
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Pfizer Investigational Site
Bordeaux, France
Pfizer Investigational Site
Bordeaux, France
Pfizer Investigational Site
Bron, France
Pfizer Investigational Site
Dijon, France
Pfizer Investigational Site
Grenoble, France
Pfizer Investigational Site
Lorient, France
Pfizer Investigational Site
Lyon, France
Pfizer Investigational Site
Marseille, France
...and 8 more locations
Change From Baseline in Annual Growth Rate at Month 12, Month 24, Month 36 in PP Population
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).
Time frame: Baseline, Month 12, Month 24, Month 36
Height
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.
Time frame: Baseline, Month 12, Month 24, Month 36
Change From Baseline in Height at Month 12, Month 24 and Month 36
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.
Time frame: Baseline, Month 12, Month 24, Month 36
Mean Height Standard Deviation Score (SDS) for Chronological Age (CA)
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.
Time frame: Baseline, Month 12, Month 24, Month 36
Change From Baseline in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12, Month 24 and Month 36
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.
Time frame: Baseline, Month 12, Month 24, Month 36
Mean Height Standard Deviation Score (SDS) for Bone Age (BA)
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.
Time frame: Baseline, Month 12, Month 24, Month 36
Change From Baseline in Height Standard Deviation Score (SDS) for Bone Age (BA) at Month 12, Month 24 and Month 36
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.
Time frame: Baseline, Month 12, Month 24, Month 36
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 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.
Time frame: Baseline, Month 12, Month 24
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 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.
Time frame: Baseline, Month 12, Month 24
Mean Growth Rate Standard Deviation Score (SDS) for Bone Age (BA)
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.
Time frame: Month 12, Month 24, Month 36
Body Mass Index (BMI)
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.
Time frame: Baseline, Month 12, Month 24, Month 36
Change From Baseline in Body Mass Index (BMI) at Month 12, Month 24 and Month 36
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.
Time frame: Baseline, Month 12, Month 24, Month 36
Change From Baseline in Bone Age (BA) at Month 12, Month 24 and Month 36
BA was estimated locally using an X-ray from the left wrist and hand.
Time frame: Baseline, Month 12, Month 24, Month 36
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.
Time frame: Baseline, Month 12, Month 24, Month 36