Back to resultsTreatment With Recombinant Human Growth Hormone (GH) in Children With Short Stature Secondary to a Long Term Corticoid Therapy
Primary Purpose
Endocrine System Diseases
Status
Terminated
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: Children with juvenile arthritis or nephrotic syndrome Before or during puberty Exclusion Criteria: Diabetes Type 1 and 2 Endocrine disease, except well substituted hypothyroidism
Sites / Locations
- 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 Height Standard Deviation Score According to Chronological Age (SDS/CA) at Year 3
Height was measured using a wall mounted device (example, Harpenden stadiometer). Height SDS/CA was obtained by measuring the height, subtracting chronological age- and gender-appropriate mean height and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Change From Baseline in Height Standard Deviation Score According to Chronological Age (SDS/CA) at Final Height
Height was measured using a wall mounted device (example, Harpenden stadiometer). Height SDS/CA was obtained by measuring the height, subtracting chronological age- and gender-appropriate mean height and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Change From Baseline in Weight Standard Deviation Score (SDS) at Final Height
Body weight was measured using a balance scale. Weight SDS was obtained by measuring the weight, subtracting age- and gender-appropriate mean weight and dividing the result by standard deviation of that mean (as obtained from age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Puberty Stage at Final Height
Pubertal stage (graded from I to V for breast development and pubic hair development) according to the Tanner's method was collected. A low stage (Stage I) corresponds to a pre-pubertal stage and a high stage (Stage V) to an adult stage.
Secondary Outcome Measures
Bone Age
Bone age was determined by the Greulich and Pyle method using left wrist and hand X-ray.
Lean Body Mass
Lean body mass, a measurement of body composition, was assessed by Dual Energy X-ray Absorptiometry (DEXA) scan.
Annual Percent Change in Lean Body Mass at Year 1, 2 and 3
Lean body mass, a measurement of body composition, was assessed by DEXA scan. Annual percent change: (Lean body mass at current year minus lean body mass at previous year) divided by lean body mass at previous year, multiplied by 100.
Percent Change From Baseline in Lean Body Mass at Year 3
Lean body mass, a measurement of body composition, was assessed by DEXA scan. Percent change: (Lean body mass at Year 3 minus lean body mass at baseline) divided by lean body mass at baseline, multiplied by 100.
Lean Body Mass as Percentage of Total Weight
Lean body mass, a measurement of body composition, was assessed by DEXA scan.
Lean Body Mass Standard Deviation Score According to Chronological Age (SDS/CA)
Lean body mass was assessed by DEXA scan. Lean body mass SDS/CA was obtained by measuring lean body mass, subtracting the chronological age- and gender-appropriate mean lean body mass and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Fat Mass
Fat mass, a measurement of body composition, was assessed by DEXA scan.
Annual Percent Change in Fat Mass at Year 1, 2 and 3
Fat mass, a measurement of body composition, was assessed by DEXA scan. Annual percent change: (Fat mass at current year minus fat mass at previous year) divided by fat mass at previous year, multiplied by 100.
Percent Change From Baseline in Fat Mass at Year 3
Fat mass, a measurement of body composition, was assessed by DEXA scan. Percent change: (Fat mass at Year 3 minus fat mass at baseline) divided by fat mass at baseline, multiplied by 100.
Fat Mass as Percentage of Total Weight
Fat mass, a measurement of body composition, was assessed by DEXA scan.
Fat Mass Standard Deviation Score According to Chronological Age (SDS/CA)
Fat mass was assessed by DEXA scan. Fat mass SDS/CA was obtained by measuring fat mass, subtracting chronological age- and gender-appropriate mean fat mass and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Apparent Bone Mineral Density of Lumbar Spine (BMAD [LS])
BMAD (LS) was assessed by DEXA scan.
Apparent Bone Mineral Density Standard Deviation Score of Lumbar Spine According to Chronological Age (BMAD [LS] [SDS/CA])
BMAD (LS) was assessed by DEXA scan. BMAD (LS) (SDS/CA) was obtained by measuring the BMAD (LS), subtracting chronological age- and gender-appropriate mean BMAD (LS) and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Apparent Bone Mineral Density Standard Deviation Score of Lumber Spine According to Tanner Puberty Stage (BMAD [LS] [SDS/Tanner Puberty Stage])
BMAD (LS) was assessed by DEXA scan. BMAD (LS) (SDS/Tanner Puberty Stage) was obtained by measuring BMAD (LS), subtracting Tanner puberty stage- and gender-appropriate mean BMAD (LS) and dividing the result by standard deviation of that mean (as obtained from Tanner puberty stage- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Bone Mineral Density of Total Body (BMD [TB])
BMD (TB) was assessed by DEXA scan.
Bone Mineral Density of Lumbar Spine (BMD [LS])
BMD (LS) was assessed by DEXA scan.
Bone Mineral Content of Total Body (BMC [TB])
DEXA scan of BMC was used to evaluate potential bone effects of treatment. BMC is an estimate of the amount of mineral (such as calcium) in the bone.
Annual Percent Change in Bone Mineral Content of Total Body (BMC [TB]) at Year 1, 2 and 3
BMC is an estimate of the amount of mineral (such as calcium) in the bone. Annual percent change: (BMC [TB] at current year minus BMC [TB] at previous year) divided by BMC [TB] at previous year, multiplied by 100.
Percent Change From Baseline in Bone Mineral Content of Total Body (BMC [TB]) at Year 3
BMC is an estimate of the amount of mineral (such as calcium) in the bone. Percent change: (BMC [TB] at Year 3 minus BMC [TB] at baseline) divided by BMC [TB] at baseline, multiplied by 100.
Bone Mineral Content Standard Deviation Score of Total Body According to Chronological Age (BMC [TB] [SDS/CA])
BMC (TB) was measured by DEXA scan. BMC (TB) (SDS/CA) was obtained by measuring BMC (TB), subtracting the chronological age- and gender-appropriate mean BMC (TB) and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Bone Mineral Content Standard Deviation Score of Total Body According to Tanner Puberty Stage (BMC [TB] [SDS/Tanner Puberty Stage])
BMC (TB) was measured by DEXA scan. BMC (TB) (SDS/Tanner Puberty Stage) was obtained by measuring BMC (TB), subtracting the Tanner puberty stage- and gender-appropriate mean BMC (TB) and dividing the result by standard deviation of that mean (as obtained from Tanner puberty stage- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00174187
Brief Title
Treatment With Recombinant Human Growth Hormone (GH) in Children With Short Stature Secondary to a Long Term Corticoid Therapy
Official Title
Treatment With Recombinant Human Growth Hormone Genotonorm (Registered) in Children With Short Stature Secondary to a Long Term Corticoid Therapy. A Study of Efficacy and Safety.
Study Type
Interventional
2. Study Status
Record Verification Date
November 2012
Overall Recruitment Status
Terminated
Why Stopped
See termination reason in detailed description.
Study Start Date
September 2000 (undefined)
Primary Completion Date
September 2011 (Actual)
Study Completion Date
September 2011 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Pfizer
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
To assess the effect of a long-term treatment by Genotonorm on linear growth in children with short stature receiving steroid therapy
To assess the effect of a long term treatment with Genotonorm on bone mineralisation
To assess the effect of a long term treatment with Genotonorm on body composition
Detailed Description
This trial terminated on 10-Jun-2011 due to prolonged issues with drug accountability and data collection discrepancies. The decision to terminate was not based on any safety concerns.
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
30 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Somatropin
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Somatropin
Intervention Description
liquid, daily, until final height Dosage: 0,46 mg/kg/week . The maximum dose should not exceed 50 µg/Kg/day
Primary Outcome Measure Information:
Title
Change From Baseline in Height Standard Deviation Score According to Chronological Age (SDS/CA) at Year 3
Description
Height was measured using a wall mounted device (example, Harpenden stadiometer). Height SDS/CA was obtained by measuring the height, subtracting chronological age- and gender-appropriate mean height and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Time Frame
Baseline, Year 3
Title
Change From Baseline in Height Standard Deviation Score According to Chronological Age (SDS/CA) at Final Height
Description
Height was measured using a wall mounted device (example, Harpenden stadiometer). Height SDS/CA was obtained by measuring the height, subtracting chronological age- and gender-appropriate mean height and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Time Frame
Baseline, when final height was reached (assessed up to Year 11)
Title
Change From Baseline in Weight Standard Deviation Score (SDS) at Final Height
Description
Body weight was measured using a balance scale. Weight SDS was obtained by measuring the weight, subtracting age- and gender-appropriate mean weight and dividing the result by standard deviation of that mean (as obtained from age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Time Frame
Baseline, when final height was reached (assessed up to Year 11)
Title
Puberty Stage at Final Height
Description
Pubertal stage (graded from I to V for breast development and pubic hair development) according to the Tanner's method was collected. A low stage (Stage I) corresponds to a pre-pubertal stage and a high stage (Stage V) to an adult stage.
Time Frame
When final height was reached (assessed up to Year 11)
Secondary Outcome Measure Information:
Title
Bone Age
Description
Bone age was determined by the Greulich and Pyle method using left wrist and hand X-ray.
Time Frame
Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
Title
Lean Body Mass
Description
Lean body mass, a measurement of body composition, was assessed by Dual Energy X-ray Absorptiometry (DEXA) scan.
Time Frame
Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
Title
Annual Percent Change in Lean Body Mass at Year 1, 2 and 3
Description
Lean body mass, a measurement of body composition, was assessed by DEXA scan. Annual percent change: (Lean body mass at current year minus lean body mass at previous year) divided by lean body mass at previous year, multiplied by 100.
Time Frame
Baseline, Year 1, 2, 3
Title
Percent Change From Baseline in Lean Body Mass at Year 3
Description
Lean body mass, a measurement of body composition, was assessed by DEXA scan. Percent change: (Lean body mass at Year 3 minus lean body mass at baseline) divided by lean body mass at baseline, multiplied by 100.
Time Frame
Baseline, Year 3
Title
Lean Body Mass as Percentage of Total Weight
Description
Lean body mass, a measurement of body composition, was assessed by DEXA scan.
Time Frame
Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
Title
Lean Body Mass Standard Deviation Score According to Chronological Age (SDS/CA)
Description
Lean body mass was assessed by DEXA scan. Lean body mass SDS/CA was obtained by measuring lean body mass, subtracting the chronological age- and gender-appropriate mean lean body mass and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Time Frame
Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
Title
Fat Mass
Description
Fat mass, a measurement of body composition, was assessed by DEXA scan.
Time Frame
Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
Title
Annual Percent Change in Fat Mass at Year 1, 2 and 3
Description
Fat mass, a measurement of body composition, was assessed by DEXA scan. Annual percent change: (Fat mass at current year minus fat mass at previous year) divided by fat mass at previous year, multiplied by 100.
Time Frame
Baseline, Year 1, 2, 3
Title
Percent Change From Baseline in Fat Mass at Year 3
Description
Fat mass, a measurement of body composition, was assessed by DEXA scan. Percent change: (Fat mass at Year 3 minus fat mass at baseline) divided by fat mass at baseline, multiplied by 100.
Time Frame
Baseline, Year 3
Title
Fat Mass as Percentage of Total Weight
Description
Fat mass, a measurement of body composition, was assessed by DEXA scan.
Time Frame
Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
Title
Fat Mass Standard Deviation Score According to Chronological Age (SDS/CA)
Description
Fat mass was assessed by DEXA scan. Fat mass SDS/CA was obtained by measuring fat mass, subtracting chronological age- and gender-appropriate mean fat mass and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Time Frame
Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
Title
Apparent Bone Mineral Density of Lumbar Spine (BMAD [LS])
Description
BMAD (LS) was assessed by DEXA scan.
Time Frame
Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
Title
Apparent Bone Mineral Density Standard Deviation Score of Lumbar Spine According to Chronological Age (BMAD [LS] [SDS/CA])
Description
BMAD (LS) was assessed by DEXA scan. BMAD (LS) (SDS/CA) was obtained by measuring the BMAD (LS), subtracting chronological age- and gender-appropriate mean BMAD (LS) and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Time Frame
Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
Title
Apparent Bone Mineral Density Standard Deviation Score of Lumber Spine According to Tanner Puberty Stage (BMAD [LS] [SDS/Tanner Puberty Stage])
Description
BMAD (LS) was assessed by DEXA scan. BMAD (LS) (SDS/Tanner Puberty Stage) was obtained by measuring BMAD (LS), subtracting Tanner puberty stage- and gender-appropriate mean BMAD (LS) and dividing the result by standard deviation of that mean (as obtained from Tanner puberty stage- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Time Frame
Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
Title
Bone Mineral Density of Total Body (BMD [TB])
Description
BMD (TB) was assessed by DEXA scan.
Time Frame
Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
Title
Bone Mineral Density of Lumbar Spine (BMD [LS])
Description
BMD (LS) was assessed by DEXA scan.
Time Frame
Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
Title
Bone Mineral Content of Total Body (BMC [TB])
Description
DEXA scan of BMC was used to evaluate potential bone effects of treatment. BMC is an estimate of the amount of mineral (such as calcium) in the bone.
Time Frame
Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
Title
Annual Percent Change in Bone Mineral Content of Total Body (BMC [TB]) at Year 1, 2 and 3
Description
BMC is an estimate of the amount of mineral (such as calcium) in the bone. Annual percent change: (BMC [TB] at current year minus BMC [TB] at previous year) divided by BMC [TB] at previous year, multiplied by 100.
Time Frame
Baseline, Year 1, 2, 3
Title
Percent Change From Baseline in Bone Mineral Content of Total Body (BMC [TB]) at Year 3
Description
BMC is an estimate of the amount of mineral (such as calcium) in the bone. Percent change: (BMC [TB] at Year 3 minus BMC [TB] at baseline) divided by BMC [TB] at baseline, multiplied by 100.
Time Frame
Baseline, Year 3
Title
Bone Mineral Content Standard Deviation Score of Total Body According to Chronological Age (BMC [TB] [SDS/CA])
Description
BMC (TB) was measured by DEXA scan. BMC (TB) (SDS/CA) was obtained by measuring BMC (TB), subtracting the chronological age- and gender-appropriate mean BMC (TB) and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Time Frame
Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
Title
Bone Mineral Content Standard Deviation Score of Total Body According to Tanner Puberty Stage (BMC [TB] [SDS/Tanner Puberty Stage])
Description
BMC (TB) was measured by DEXA scan. BMC (TB) (SDS/Tanner Puberty Stage) was obtained by measuring BMC (TB), subtracting the Tanner puberty stage- and gender-appropriate mean BMC (TB) and dividing the result by standard deviation of that mean (as obtained from Tanner puberty stage- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Time Frame
Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
Other Pre-specified Outcome Measures:
Title
Growth Velocity (GV)
Description
Growth velocity measures the annual rate of increase in height.
Time Frame
Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
Title
Growth Velocity Standard Deviation Score According to Chronological Age (GV [SDS/CA])
Description
GV measures the annual rate of increase in height. GV (SDS/CA) was obtained by measuring GV, subtracting the chronological age- and gender-appropriate mean GV and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Time Frame
Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
Title
Growth Velocity Standard Deviation Score According to Bone Age (GV [SDS/BA])
Description
GV measures the annual rate of increase in height. GV (SDS/BA) was obtained by measuring GV, subtracting the bone age- and gender-appropriate mean GV and dividing the result by standard deviation of that mean (as obtained from bone age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Time Frame
Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11
Title
Insulin-like Growth Factor-1 (IGF-1) Concentration up to Year 3
Time Frame
Baseline, Year 1, 2, 3
Title
Insulin-like Growth Factor-1 (IGF-1) Concentration After Year 3
Time Frame
Year 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5, 9, 9.5, 10; 0.5 and 1 year after somatropin discontinuation, Final Height (assessed up to Year 11)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
11 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Children with juvenile arthritis or nephrotic syndrome
Before or during puberty
Exclusion Criteria:
Diabetes Type 1 and 2
Endocrine disease, except well substituted hypothyroidism
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
Paris
ZIP/Postal Code
75019
Country
France
Facility Name
Pfizer Investigational Site
City
Paris
ZIP/Postal Code
75743
Country
France
12. IPD Sharing Statement
Citations:
PubMed Identifier
29029101
Citation
David H, Aupiais C, Louveau B, Quartier P, Jacqz-Aigrain E, Carel JC, Simon D. Growth Outcomes After GH Therapy of Patients Given Long-Term Corticosteroids for Juvenile Idiopathic Arthritis. J Clin Endocrinol Metab. 2017 Dec 1;102(12):4578-4587. doi: 10.1210/jc.2017-01455.
Results Reference
derived
Links:
URL
https://trialinfoemail.pfizer.com/pages/landing.aspx?StudyID=307-MET-9002-0009&StudyName=Treatment%20with%20Recombinant%20Human%20Growth%20Hormone%20%28GH%29%20in%20Children%20with%20Short%20Stature%20Secondary%20to%20a%20Long%20Term%20Corticoid%20Therap
Description
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Treatment With Recombinant Human Growth Hormone (GH) in Children With Short Stature Secondary to a Long Term Corticoid Therapy
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