Growth Retardation In Children With Special Pathological Conditions Or Disease
Primary Purpose
Endocrine System Diseases
Status
Completed
Phase
Phase 3
Locations
France
Study Type
Interventional
Intervention
Somatropin
Sponsored by
About this trial
This is an interventional treatment trial for Endocrine System Diseases
Eligibility Criteria
Inclusion Criteria: Bone age < 13 years for a boy and < 11 years for a girl Naive child: Measured Height < -2.5 SD for CA Child currently treated by GH Exclusion Criteria: Idiopathic short stature Syndrome known to be associated with an increased risk of cancer e.g. family history of adenomatous polyposis
Sites / Locations
- Pfizer Investigational Site
- Pfizer Investigational Site
- Pfizer Investigational Site
- Pfizer Investigational Site
- Pfizer Investigational Site
- Pfizer Investigational Site
- Pfizer Investigational Site
- Pfizer Investigational Site
- Pfizer Investigational Site
- Pfizer Investigational Site
- Pfizer Investigational Site
- Pfizer Investigational Site
- Pfizer Investigational Site
- Pfizer Investigational Site
- Pfizer Investigational Site
- Pfizer Investigational Site
- Pfizer Investigational Site
- Pfizer Investigational Site
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Somatropin
Arm Description
Outcomes
Primary Outcome Measures
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.
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.
Secondary Outcome Measures
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).
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).
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00163215
Brief Title
Growth Retardation In Children With Special Pathological Conditions Or Disease
Official Title
Evolution Of Growth Rate In Children Suffering From A Disease Associated With Growth Retardation and Treated By Genotonorm. A Pilot Study.
Study Type
Interventional
2. Study Status
Record Verification Date
November 2012
Overall Recruitment Status
Completed
Study Start Date
January 2005 (undefined)
Primary Completion Date
October 2011 (Actual)
Study Completion Date
October 2011 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Pfizer
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Endocrine System Diseases
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
46 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Somatropin
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Somatropin
Intervention Description
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
Primary Outcome Measure Information:
Title
Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 36 in Intent-to-Treat (ITT) Population
Description
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
Title
Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 36 in Per-Protocol (PP) Population
Description
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
Secondary Outcome Measure Information:
Title
Change From Baseline in Annual Growth Rate at Month 12, Month 24 and Month 36 in ITT Population
Description
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
Title
Change From Baseline in Annual Growth Rate at Month 12, Month 24, Month 36 in PP Population
Description
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
Title
Height
Description
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
Title
Change From Baseline in Height at Month 12, Month 24 and Month 36
Description
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
Title
Mean Height Standard Deviation Score (SDS) for Chronological Age (CA)
Description
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
Title
Change From Baseline in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12, Month 24 and Month 36
Description
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
Title
Mean Height Standard Deviation Score (SDS) for Bone Age (BA)
Description
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
Title
Change From Baseline in Height Standard Deviation Score (SDS) for Bone Age (BA) at Month 12, Month 24 and Month 36
Description
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
Title
Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12 and Month 24 in ITT Population
Description
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
Title
Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12 and Month 24 in PP Population
Description
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
Title
Mean Growth Rate Standard Deviation Score (SDS) for Bone Age (BA)
Description
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
Title
Body Mass Index (BMI)
Description
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
Title
Change From Baseline in Body Mass Index (BMI) at Month 12, Month 24 and Month 36
Description
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
Title
Change From Baseline in Bone Age (BA) at Month 12, Month 24 and Month 36
Description
BA was estimated locally using an X-ray from the left wrist and hand.
Time Frame
Baseline, Month 12, Month 24, Month 36
Title
Ratio of Bone Age (BA) to Chronological Age (CA)
Description
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
10. Eligibility
Sex
All
Minimum Age & Unit of Time
11 Years
Maximum Age & Unit of Time
13 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Bone age < 13 years for a boy and < 11 years for a girl
Naive child: Measured Height < -2.5 SD for CA
Child currently treated by GH
Exclusion Criteria:
Idiopathic short stature
Syndrome known to be associated with an increased risk of cancer e.g. family history of adenomatous polyposis
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Pfizer CT.gov Call Center
Organizational Affiliation
Pfizer
Official's Role
Study Director
Facility Information:
Facility Name
Pfizer Investigational Site
City
Annemasse Cedex
ZIP/Postal Code
74107
Country
France
Facility Name
Pfizer Investigational Site
City
Besancon
ZIP/Postal Code
25030
Country
France
Facility Name
Pfizer Investigational Site
City
Bordeaux
ZIP/Postal Code
33000
Country
France
Facility Name
Pfizer Investigational Site
City
Bordeaux
ZIP/Postal Code
33076
Country
France
Facility Name
Pfizer Investigational Site
City
Bron Cedex
ZIP/Postal Code
69677
Country
France
Facility Name
Pfizer Investigational Site
City
Dijon
ZIP/Postal Code
21000
Country
France
Facility Name
Pfizer Investigational Site
City
Grenoble Cedex
ZIP/Postal Code
38043
Country
France
Facility Name
Pfizer Investigational Site
City
Lorient
ZIP/Postal Code
56100
Country
France
Facility Name
Pfizer Investigational Site
City
Lyon Cedex 05
ZIP/Postal Code
69322
Country
France
Facility Name
Pfizer Investigational Site
City
Marseille
ZIP/Postal Code
13385
Country
France
Facility Name
Pfizer Investigational Site
City
Montpellier
ZIP/Postal Code
34059
Country
France
Facility Name
Pfizer Investigational Site
City
Paris
ZIP/Postal Code
75015
Country
France
Facility Name
Pfizer Investigational Site
City
Paris
ZIP/Postal Code
75019
Country
France
Facility Name
Pfizer Investigational Site
City
Paris
ZIP/Postal Code
75571
Country
France
Facility Name
Pfizer Investigational Site
City
Paris
ZIP/Postal Code
75674 Cedex
Country
France
Facility Name
Pfizer Investigational Site
City
PRINGY Cedex
ZIP/Postal Code
74374
Country
France
Facility Name
Pfizer Investigational Site
City
Toulouse Cedex
ZIP/Postal Code
31026
Country
France
Facility Name
Pfizer Investigational Site
City
Vandoeuvre Les Nancy Cedex
ZIP/Postal Code
54511
Country
France
12. IPD Sharing Statement
Links:
URL
https://trialinfoemail.pfizer.com/pages/landing.aspx?StudyID=A6281269&StudyName=Growth%20Retardation%20In%20Children%20With%20Special%20Pathological%20Conditions%20Or%20Disease%20
Description
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Learn more about this trial
Growth Retardation In Children With Special Pathological Conditions Or Disease
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