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Safety and efficacy of Somatropin will be evaluated in short children born with a list weight below 1500 g and that did not catch up to normal height at the age of 4.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment Arm | Active Comparator |
| |
| Control Arm | No Intervention |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Somatropin | Drug | Controlled, prospective, randomized, multicenter study with an untreated (control) group during the first year. The children will be randomized into treatment or untreated (control) group. After one year the control group will undergo GH-therapy, too. Children randomized to the control group will get the possibility to continue treatment for a further year. The study will end after 2 and 3 years of observation, respectively. |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Height Standard Deviation Score (SDS) After 1 Year | Change in Height SDS after 1 year where SDS=height minus mean (age-and sex-matched reference) divided by SD (age and sex-matched reference). | Baseline to 1 year (Month 12) |
| Change in Growth Velocity Standard Deviation Score (SDS) After 1 Year | Change in Growth Velocity (GV) SDS after 1 year where SDS=GV minus mean (age-and sex-matched reference) divided by SD (age and sex-matched reference). | Baseline to 1 year (Month 12) |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Growth Velocity After 1 Year and After 2 Years | Growth velocity measured as centimeters per year. | Baseline, Month 12, Month 24 |
| Change From Baseline in Growth Velocity SDS After 2 Years |
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Inclusion Criteria:
Subjects must meet all of the following inclusion criteria to be eligible for enrollment into the trial:
Exclusion Criteria:
Subjects presenting with any of the following will not be included in the trial:
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Pfizer Investigational Site | Chemnitz | 09009 | Germany | |||
| Pfizer Investigational Site |
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| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
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| ID | Title | Description |
|---|---|---|
| FG000 | Somatropin | The children were randomized into a treated group receiving 0.068 mg/kg/day (0.48mg/kg/week) subcutaneous somatropin according to exact body weight specific calculation. Dose adjustments were made at 6 month intervals. (mg=milligrams, kg=kilograms) |
| FG001 | Control Arm | The children were randomized into control group and after 1 year underwent Growth Hormone (GH) therapy, with 0.068 mg/kg/day (0.48mg/kg/week) subcutaneous somatropin according to exact body weight specific calculation. Dose adjustments were made at 6 month intervals. mg=milligram, kg=kilogram. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
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|
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| ID | Title | Description |
|---|---|---|
| BG000 | Somatropin | The children were randomized into a treated group receiving 0.068 mg/kg/day (0.48mg/kg/week) subcutaneous somatropin according to exact body weight specific calculation. Dose adjustments were made at 6 month intervals. (mg=milligrams, kg=kilograms) |
| BG001 | Control Arm |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change in Height Standard Deviation Score (SDS) After 1 Year | Change in Height SDS after 1 year where SDS=height minus mean (age-and sex-matched reference) divided by SD (age and sex-matched reference). | Full Analysis Set (FAS; all randomized subjects who had at least 1 post-baseline efficacy measurement); Control group received Somatropin from Month 12 onwards. | Posted | Least Squares Mean | Standard Error | centimeters | Baseline to 1 year (Month 12) |
|
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Safety population=all participants who received at least 1 dose of study treatment. An Adverse Event (AE) term may be reported as both a serious and non-serious AE, but are distinct events. AE may = serious for 1 subject and = non-serious for another subject or subject may have experienced both a serious and non-serious episode of the same event.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Somatropin | The children were randomized into a treated group receiving 0.068 mg/kg/day (0.48mg/kg/week) subcutaneous somatropin according to exact body weight specific calculation. Dose adjustments were made at 6 month intervals. (mg=milligrams, kg=kilograms) |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pyrexia | General disorders | MedDRA (13.0) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Autoimmune thyroiditis | Endocrine disorders | MedDRA (13.0) | Systematic Assessment |
Result values for the primary outcome measures were revised at the final analysis due to programmatic corrections: age rounded up if >6 months past last birthday. Height and height SDS not rounded for final analysis; rounded only for the reports.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer ClinicalTrials.gov Call Center | Pfizer, Inc. | 1-800-718-1021 | ClinicalTrials.gov_Inquiries@pfizer.com |
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| ID | Term |
|---|---|
| D019382 | Human Growth Hormone |
| ID | Term |
|---|---|
| D013006 | Growth Hormone |
| D010908 | Pituitary Hormones, Anterior |
| D010907 | Pituitary Hormones |
| D036361 | Peptide Hormones |
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|
| Control Arm | Other | Controlled, prospective, randomized, multicenter study with an untreated (control) group during the first year. The children will be randomized into treatment or untreated (control) group. After one year the control group will undergo GH-therapy, too. The study will end after 2 and 3 years of observation, respectively. Children randomized to the control group will get the possibility to continue treatment for a further year. |
|
Change in Growth Velocity SDS after 2 years (24 months) where SDS = growth velocity minus mean (age-and sex-matched reference) divided by SD (age and sex-matched reference).
| Baseline, Month 24 |
| Change From Baseline in Height After 1 Year and After 2 Years | Baseline, Month 12, Month 24 |
| Change From Baseline in Height SDS After 2 Years | Change in Height SDS after 2 years (24 months) where SDS = height minus mean (age-and sex-matched reference) divided by SD (age and sex-matched reference). | Baseline, Month 24 |
| Change From Baseline in Body Composition (Skinfold Thickness) After 1 Year and After 2 Years: Triceps | Body composition measured as skinfold thickness at tricep in millimeters (mm); measured halfway down the left upper arm with arm hanging in relaxed position at participant's side. | Baseline, Month 12, Month 24 |
| Change From Baseline in Body Composition (Skinfold Thickness) After 1 Year and After 2 Years: Subscapular | Body composition measured as subscapular skinfold thickness in millimeters (mm); measured laterally just below the angle of the left scapula. | Baseline, Month 12, Month 24 |
| Change From Baseline in Body Composition (Skinfold Thickness) After 1 Year and After 2 Years: Suprailiac | Body composition measured as suprailiac skinfold thickness in millimeters (mm); measured just above the iliac crest in the middle-axillary line. | Baseline, Month 12, Month 24 |
| Change From Baseline in Volumetric Cortical Bone Mineral Density (BMD) Using Peripheral Quantitative Computed Tomography (pQCT) After 1 Year and After 2 Years | Volumetric Cortical BMD measured as milligrams per cubic millimeter (mg/mm3). BMD (proximal radius) SDS (number of standard deviations a participant's BMD differs from the average BMD of their age and sex). Baseline and post-baseline SDS values transformed to age and sex specific z-score (Ln(test result/M)]/S); Ln=natural logarithm; M=age- or height-) and sex-specific mean value; S=age-(or height-) and sex-specific coefficient of variation) then change from baseline is calculated. Positive values are above the average for participant's age and sex; negative values are below the average. | Baseline, Month 12, Month 24 |
| Change From Baseline in Bone Structure Using pQCT After 1 Year and 2 Years: Cortical Cross-sectional Area (CSA) | Bone structure Cortical CSA measured as millimeters squared (mm2). CSA (proximal radius) SDS (number of standard deviations a participant's CSA differs from the average CSA of their age and sex). Baseline and post-baseline SDS values transformed to age and sex specific z-score (Ln(test result/M)]/S); Ln=natural logarithm; M=age- or height-) and sex-specific mean value; S=age-(or height-) and sex-specific coefficient of variation) then change from baseline is calculated. Positive values are above the average for participant's age and sex; negative values are below the average. | Baseline, Month 12, Month 24 |
| Change From Baseline in Bone Structure Using Peripheral Quantitative Computed Tomography (pQCT) After 1 Year and 2 Years: Total Cross-sectional Area (CSA) | Bone structure Total CSA measured as millimeters squared (mm2). Total CSA (proximal radius) SDS (number of standard deviations a participant's CSA differs from the average CSA of their age and sex). Baseline and post-baseline SDS values transformed to age and sex specific z-score (Ln(test result/M)]/S); Ln=natural logarithm; M=age- or height-) and sex-specific mean value; S=age-(or height-) and sex-specific coefficient of variation) then change from baseline is calculated. Positive values are above the average for participant's age and sex; negative values are below the average. | Baseline, Month 12, Month 24 |
| Change From Baseline in Bone Structure Using pQCT After 1 Year and 2 Years: Muscle Cross-sectional Area (CSA) | Bone structure Muscle CSA measured as millimeters squared (mm2). CSA (proximal radius) SDS (number of standard deviations a participant's CSA differs from the average CSA of their age and sex). Baseline and post-baseline SDS values transformed to age and sex specific z-score (Ln(test result/M)]/S); Ln=natural logarithm; M=age- or height-) and sex-specific mean value; S=age-(or height-) and sex-specific coefficient of variation) then change from baseline is calculated. Positive values are above the average for participant's age and sex; negative values are below the average. | Baseline, Month 12, Month 24 |
| Change From Baseline in Bone Structure Using pQCT After 1 Year and 2 Years: Cortical Thickness (CT) | Cortical Thickness measured as millimeters (mm). CT (proximal radius) SDS (number of standard deviations a participant's CT differs from the average CT of their age and sex). Baseline and post-baseline SDS values transformed to age and sex specific z-score (Ln(test result/M)]/S); Ln=natural logarithm; M=age- or height-) and sex-specific mean value; S=age-(or height-) and sex-specific coefficient of variation) then change from baseline is calculated. Positive values are above the average for participant's age and sex; negative values are below the average. | Baseline, Month 12, Month 24 |
| Change From Baseline in Bone Structure Using pQCT After 1 Year and 2 Years: Marrow Area (MA) | Marrow Area measured as millimeters squared (mm2). MA (proximal radius) SDS (number of standard deviations a participant's MA differs from the average MA of their age and sex). Baseline and post-baseline SDS values transformed to age and sex specific z-score (Ln(test result/M)]/S); Ln=natural logarithm; M=age- or height-) and sex-specific mean value; S=age-(or height-) and sex-specific coefficient of variation) then change from baseline is calculated. Positive values are above the average for participant's age and sex; negative values are below the average. | Baseline, Month 12, Month 24 |
| Change From Baseline in Bone Stability Using pQCT After 1 Year and After 2 Years: Strength-strain Index (SSI) | Bone stability expressed as polar SSI in cubic millimeters (mm3). SSI (proximal radius) SDS (number of standard deviations a participant's SSI differs from the average SSI of their age and sex). Baseline and post-baseline SDS values transformed to age and sex specific z-score (Ln(test result/M)]/S); Ln=natural logarithm; M=age- or height-) and sex-specific mean value; S=age-(or height-) and sex-specific coefficient of variation) then change from baseline is calculated. Positive values are above the average for participant's age and sex; negative values are below the average. | Baseline, Month 12, Month 24 |
| Change From Baseline in Muscle Strength: Hand Grip SDS After 1 Year and After 2 Years | Muscle strength determined by measuring grip force (kilograms) using hand grip dynamometer for participants ≥6 years of age. Baseline and post-baseline SDS values transformed to age and sex specific z-score. Change in hand grip calculated as SDS where SDS = hand grip minus mean (age- and sex-matched reference) divided by SD (age- and sex-matched reference). Positive values are above the average for participant's age and sex; negative values are below the average. | Baseline, Month 12, Month 24 |
| Number of Participants With Change in Insulin Sensitivity: Somatropin | Insulin sensitivity calculated as incidence of pathological glucose intolerance assessed prior to randomization (Screening Day -3 to Baseline Day 0) and at final visit (final visit: Somatropin treatment group=Month 24). Pathological glucose intolerance (oral glucose tolerance test) measured as venous (blood or plasma) with range minimum 120 milligrams per deciliter (mg/dL) to >140 mg/dL; capillary (blood) with range minimum 120 mg/dL to >120 mg/dL; or method not known with range minimum 120 mg/dL to >120 mg/dL. | Baseline, Month 24 |
| Number of Participants With Change in Insulin Sensitivity: Control Arm | Insulin sensitivity calculated as incidence of pathological glucose intolerance assessed prior to randomization (Screening Day -3 to Baseline Day 0) and at final visit (final visit: Control Arm=Month 36). Pathological glucose intolerance (oral glucose tolerance test) measured as venous (blood or plasma) with range minimum 120 milligrams per deciliter (mg/dL) to >140 mg/dL; capillary (blood) with range minimum 120 mg/dL to >120 mg/dL; or method not known with range minimum 120 mg/dL to >120 mg/dL. | Baseline, Month 36 |
| Growth Curve Comparison Based on Height SDS | Growth curve comparison with height SDS in centimeters as the dependent variable; SDS = height minus mean (age-and sex-matched reference) divided by SD (age and sex-matched reference). | Month 12, Month 24 |
| Growth Curve Comparison Based on Height SDS: Control Arm | Growth curve comparison with height SDS in centimeters as the dependent variable; SDS = height minus mean (age-and sex-matched reference) divided by SD (age and sex-matched reference). Control Arm final visit=Month 36. | Month 36 |
| Growth Curve Comparison Based on Height | Growth curve comparison with height in centimeters as the dependent variable. | Month 12, Month 24 |
| Growth Curve Comparison Based on Height: Control Arm | Growth curve comparison with height in centimeters as the dependent variable. | Month 36 |
| Cologne |
| 50937 |
| Germany |
| Pfizer Investigational Site | Erlangen | 91054 | Germany |
| Pfizer Investigational Site | Freiburg im Breisgau | 79106 | Germany |
| Pfizer Investigational Site | Heidelberg | 69120 | Germany |
| Pfizer Investigational Site | Homburg | 66424 | Germany |
| Pfizer Investigational Site | Leipzig | 04103 | Germany |
| Pfizer Investigational Site | Tübingen | 72076 | Germany |
The children were randomized into control group and after 1 year underwent Growth Hormone (GH) therapy, with 0.068 mg/kg/day (0.48mg/kg/week) subcutaneous somatropin according to exact body weight specific calculation. Dose adjustments were made at 6 month intervals. mg=milligram, kg=kilogram. |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Age, Customized | 1 subject in each treatment group was below the age of 4 at screening. However, both of them were 4 years old when treatment began; they did not violate the corresponding inclusion criterion. | Number | Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
The children were randomized into control group and after 1 year underwent Growth Hormone (GH) therapy, with 0.068 mg/kg/day (0.48mg/kg/week) subcutaneous somatropin according to exact body weight specific calculation. Dose adjustments were made at 6 month intervals. mg=milligram, kg=kilogram. |
|
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|
| Secondary | Change From Baseline in Growth Velocity After 1 Year and After 2 Years | Growth velocity measured as centimeters per year. | FAS; Control group received Somatropin from Month 12 onwards. | Posted | Least Squares Mean | Standard Error | centimeters per year | Baseline, Month 12, Month 24 |
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|
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| Secondary | Change From Baseline in Growth Velocity SDS After 2 Years | Change in Growth Velocity SDS after 2 years (24 months) where SDS = growth velocity minus mean (age-and sex-matched reference) divided by SD (age and sex-matched reference). | FAS; Control group received Somatropin from Month 12 onwards. | Posted | Least Squares Mean | Standard Error | centimeters per year | Baseline, Month 24 |
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| Secondary | Change From Baseline in Height After 1 Year and After 2 Years | FAS; Control group received Somatropin from Month 12 onwards. | Posted | Mar 2011 | Least Squares Mean | Standard Error | centimeters | Baseline, Month 12, Month 24 |
|
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| Secondary | Change From Baseline in Height SDS After 2 Years | Change in Height SDS after 2 years (24 months) where SDS = height minus mean (age-and sex-matched reference) divided by SD (age and sex-matched reference). | FAS; Control group received Somatropin from Month 12 onwards. | Posted | Least Squares Mean | Standard Error | centimeters | Baseline, Month 24 |
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| Secondary | Change From Baseline in Body Composition (Skinfold Thickness) After 1 Year and After 2 Years: Triceps | Body composition measured as skinfold thickness at tricep in millimeters (mm); measured halfway down the left upper arm with arm hanging in relaxed position at participant's side. | FAS; Control group received Somatropin from Month 12 onwards. N=number of participants with evaluable data at observation. | Posted | Least Squares Mean | Standard Error | millimeters | Baseline, Month 12, Month 24 |
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|
| Secondary | Change From Baseline in Body Composition (Skinfold Thickness) After 1 Year and After 2 Years: Subscapular | Body composition measured as subscapular skinfold thickness in millimeters (mm); measured laterally just below the angle of the left scapula. | FAS; Control group received Somatropin from Month 12 onwards. N=number of participants with evaluable data at observation. | Posted | Least Squares Mean | Standard Error | millimeters | Baseline, Month 12, Month 24 |
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| Secondary | Change From Baseline in Body Composition (Skinfold Thickness) After 1 Year and After 2 Years: Suprailiac | Body composition measured as suprailiac skinfold thickness in millimeters (mm); measured just above the iliac crest in the middle-axillary line. | FAS; Control group received Somatropin from Month 12 onwards. N=number of participants with evaluable data at observation. | Posted | Least Squares Mean | Standard Error | millimeters | Baseline, Month 12, Month 24 |
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| Secondary | Change From Baseline in Volumetric Cortical Bone Mineral Density (BMD) Using Peripheral Quantitative Computed Tomography (pQCT) After 1 Year and After 2 Years | Volumetric Cortical BMD measured as milligrams per cubic millimeter (mg/mm3). BMD (proximal radius) SDS (number of standard deviations a participant's BMD differs from the average BMD of their age and sex). Baseline and post-baseline SDS values transformed to age and sex specific z-score (Ln(test result/M)]/S); Ln=natural logarithm; M=age- or height-) and sex-specific mean value; S=age-(or height-) and sex-specific coefficient of variation) then change from baseline is calculated. Positive values are above the average for participant's age and sex; negative values are below the average. | FAS; Control group received Somatropin from Month 12 onwards; (n)=number of participants from FAS with evaluable pQCT data for somatropin and control arm groups, respectively. | Posted | Least Squares Mean | Standard Error | z-score | Baseline, Month 12, Month 24 |
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| Primary | Change in Growth Velocity Standard Deviation Score (SDS) After 1 Year | Change in Growth Velocity (GV) SDS after 1 year where SDS=GV minus mean (age-and sex-matched reference) divided by SD (age and sex-matched reference). | FAS; Control group received Somatropin from Month 12 onwards. | Posted | Least Squares Mean | Standard Error | centimeters per year | Baseline to 1 year (Month 12) |
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| Secondary | Change From Baseline in Bone Structure Using pQCT After 1 Year and 2 Years: Cortical Cross-sectional Area (CSA) | Bone structure Cortical CSA measured as millimeters squared (mm2). CSA (proximal radius) SDS (number of standard deviations a participant's CSA differs from the average CSA of their age and sex). Baseline and post-baseline SDS values transformed to age and sex specific z-score (Ln(test result/M)]/S); Ln=natural logarithm; M=age- or height-) and sex-specific mean value; S=age-(or height-) and sex-specific coefficient of variation) then change from baseline is calculated. Positive values are above the average for participant's age and sex; negative values are below the average. | FAS; Control group received Somatropin from Month 12 onwards; (n)=number of participants from FAS with evaluable pQCT data for somatropin and control arm groups, respectively. | Posted | Least Squares Mean | Standard Error | z-score | Baseline, Month 12, Month 24 |
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| Secondary | Change From Baseline in Bone Structure Using Peripheral Quantitative Computed Tomography (pQCT) After 1 Year and 2 Years: Total Cross-sectional Area (CSA) | Bone structure Total CSA measured as millimeters squared (mm2). Total CSA (proximal radius) SDS (number of standard deviations a participant's CSA differs from the average CSA of their age and sex). Baseline and post-baseline SDS values transformed to age and sex specific z-score (Ln(test result/M)]/S); Ln=natural logarithm; M=age- or height-) and sex-specific mean value; S=age-(or height-) and sex-specific coefficient of variation) then change from baseline is calculated. Positive values are above the average for participant's age and sex; negative values are below the average. | FAS; Control group received Somatropin from Month 12 onwards; (n)=number of participants from FAS with evaluable pQCT data for somatropin and control arm groups, respectively. | Posted | Least Squares Mean | Standard Error | z-score | Baseline, Month 12, Month 24 |
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| Secondary | Change From Baseline in Bone Structure Using pQCT After 1 Year and 2 Years: Muscle Cross-sectional Area (CSA) | Bone structure Muscle CSA measured as millimeters squared (mm2). CSA (proximal radius) SDS (number of standard deviations a participant's CSA differs from the average CSA of their age and sex). Baseline and post-baseline SDS values transformed to age and sex specific z-score (Ln(test result/M)]/S); Ln=natural logarithm; M=age- or height-) and sex-specific mean value; S=age-(or height-) and sex-specific coefficient of variation) then change from baseline is calculated. Positive values are above the average for participant's age and sex; negative values are below the average. | FAS; Control group received Somatropin from Month 12 onwards; (n)=number of participants from FAS with evaluable pQCT data for somatropin and control arm groups, respectively. | Posted | Least Squares Mean | Standard Error | z-score | Baseline, Month 12, Month 24 |
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| Secondary | Change From Baseline in Bone Structure Using pQCT After 1 Year and 2 Years: Cortical Thickness (CT) | Cortical Thickness measured as millimeters (mm). CT (proximal radius) SDS (number of standard deviations a participant's CT differs from the average CT of their age and sex). Baseline and post-baseline SDS values transformed to age and sex specific z-score (Ln(test result/M)]/S); Ln=natural logarithm; M=age- or height-) and sex-specific mean value; S=age-(or height-) and sex-specific coefficient of variation) then change from baseline is calculated. Positive values are above the average for participant's age and sex; negative values are below the average. | FAS; Control group received Somatropin from Month 12 onwards. Cortical thickness was not analyzed as planned. | Posted | Baseline, Month 12, Month 24 |
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| Secondary | Change From Baseline in Bone Structure Using pQCT After 1 Year and 2 Years: Marrow Area (MA) | Marrow Area measured as millimeters squared (mm2). MA (proximal radius) SDS (number of standard deviations a participant's MA differs from the average MA of their age and sex). Baseline and post-baseline SDS values transformed to age and sex specific z-score (Ln(test result/M)]/S); Ln=natural logarithm; M=age- or height-) and sex-specific mean value; S=age-(or height-) and sex-specific coefficient of variation) then change from baseline is calculated. Positive values are above the average for participant's age and sex; negative values are below the average. | FAS; Control group received Somatropin from Month 12 onwards. Marrow Area was not analyzed as planned. | Posted | Baseline, Month 12, Month 24 |
|
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| Secondary | Change From Baseline in Bone Stability Using pQCT After 1 Year and After 2 Years: Strength-strain Index (SSI) | Bone stability expressed as polar SSI in cubic millimeters (mm3). SSI (proximal radius) SDS (number of standard deviations a participant's SSI differs from the average SSI of their age and sex). Baseline and post-baseline SDS values transformed to age and sex specific z-score (Ln(test result/M)]/S); Ln=natural logarithm; M=age- or height-) and sex-specific mean value; S=age-(or height-) and sex-specific coefficient of variation) then change from baseline is calculated. Positive values are above the average for participant's age and sex; negative values are below the average. | FAS; Control group received Somatropin from Month 12 onwards; (n)=number of participants from FAS with evaluable pQCT data for somatropin and control arm groups, respectively. | Posted | Least Squares Mean | Standard Error | z-score | Baseline, Month 12, Month 24 |
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| Secondary | Change From Baseline in Muscle Strength: Hand Grip SDS After 1 Year and After 2 Years | Muscle strength determined by measuring grip force (kilograms) using hand grip dynamometer for participants ≥6 years of age. Baseline and post-baseline SDS values transformed to age and sex specific z-score. Change in hand grip calculated as SDS where SDS = hand grip minus mean (age- and sex-matched reference) divided by SD (age- and sex-matched reference). Positive values are above the average for participant's age and sex; negative values are below the average. | FAS; Control group received Somatropin from Month 12 onwards; (n)=number of participants ≥6 years of age with evaluable data at observation for Somatropin and Control Arm, respectively. SDS reference values used were for the right hand but the hand grip strength measured for this study was for the dominant hand (may not have been the right hand). | Posted | Least Squares Mean | Standard Error | z-score | Baseline, Month 12, Month 24 |
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| Secondary | Number of Participants With Change in Insulin Sensitivity: Somatropin | Insulin sensitivity calculated as incidence of pathological glucose intolerance assessed prior to randomization (Screening Day -3 to Baseline Day 0) and at final visit (final visit: Somatropin treatment group=Month 24). Pathological glucose intolerance (oral glucose tolerance test) measured as venous (blood or plasma) with range minimum 120 milligrams per deciliter (mg/dL) to >140 mg/dL; capillary (blood) with range minimum 120 mg/dL to >120 mg/dL; or method not known with range minimum 120 mg/dL to >120 mg/dL. | FAS | Posted | Number | participants | Baseline, Month 24 |
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| Secondary | Number of Participants With Change in Insulin Sensitivity: Control Arm | Insulin sensitivity calculated as incidence of pathological glucose intolerance assessed prior to randomization (Screening Day -3 to Baseline Day 0) and at final visit (final visit: Control Arm=Month 36). Pathological glucose intolerance (oral glucose tolerance test) measured as venous (blood or plasma) with range minimum 120 milligrams per deciliter (mg/dL) to >140 mg/dL; capillary (blood) with range minimum 120 mg/dL to >120 mg/dL; or method not known with range minimum 120 mg/dL to >120 mg/dL. | FAS; Control group received Somatropin from Month 12 onwards. | Posted | Number | participants | Baseline, Month 36 |
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| Secondary | Growth Curve Comparison Based on Height SDS | Growth curve comparison with height SDS in centimeters as the dependent variable; SDS = height minus mean (age-and sex-matched reference) divided by SD (age and sex-matched reference). | FAS; Control group received Somatropin from Month 12 onwards. Data for Month 36 (applicable only to the Control Arm) is reported in a separate outcome measure. | Posted | Least Squares Mean | Standard Error | centimeters | Month 12, Month 24 |
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| Secondary | Growth Curve Comparison Based on Height SDS: Control Arm | Growth curve comparison with height SDS in centimeters as the dependent variable; SDS = height minus mean (age-and sex-matched reference) divided by SD (age and sex-matched reference). Control Arm final visit=Month 36. | FAS; Control group received Somatropin from Month 12 onwards. N=number of subjects with evaluable data at observation. Month 36 visit not applicable to Somatropin treatment group. | Posted | Least Squares Mean | Standard Error | centimeters | Month 36 |
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| Secondary | Growth Curve Comparison Based on Height | Growth curve comparison with height in centimeters as the dependent variable. | FAS; Control group received Somatropin from Month 12 onwards. Data for Month 36 (applicable only to the Control Arm) is reported in a separate outcome measure. | Posted | Least Squares Mean | Standard Error | centimeters | Month 12, Month 24 |
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| Secondary | Growth Curve Comparison Based on Height: Control Arm | Growth curve comparison with height in centimeters as the dependent variable. | FAS; Control group received Somatropin from Month 12 onwards. N=number of subjects with evaluable data at observation. Month 36 visit not applicable to Somatropin treatment group. | Posted | Least Squares Mean | Standard Error | centimeters | Month 36 |
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|
| 5 |
| 18 |
| 13 |
| 18 |
| EG001 | Control Arm | The children were randomized into control group and after 1 year underwent Growth Hormone (GH) therapy, with 0.068 mg/kg/day (0.48mg/kg/week) subcutaneous somatropin according to exact body weight specific calculation. Dose adjustments were made at 6 month intervals. mg=milligram, kg=kilogram. | 1 | 15 | 11 | 15 |
| Acute tonsillitis | Infections and infestations | MedDRA (13.0) | Systematic Assessment |
|
| Bronchitis viral | Infections and infestations | MedDRA (13.0) | Systematic Assessment |
|
| Bronchopneumonia | Infections and infestations | MedDRA (13.0) | Systematic Assessment |
|
| Ear infection | Infections and infestations | MedDRA (13.0) | Systematic Assessment |
|
| Tonsillitis | Infections and infestations | MedDRA (13.0) | Systematic Assessment |
|
| Facial palsy | Nervous system disorders | MedDRA (13.0) | Systematic Assessment |
|
| Febrile convulsion | Nervous system disorders | MedDRA (13.0) | Systematic Assessment |
|
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA (13.0) | Systematic Assessment |
|
| Hypothyroidism | Endocrine disorders | MedDRA (13.0) | Systematic Assessment |
|
| Visual impairment | Eye disorders | MedDRA (13.0) | Systematic Assessment |
|
| Inguinal hernia | Gastrointestinal disorders | MedDRA (13.0) | Systematic Assessment |
|
| Tooth disorder | Gastrointestinal disorders | MedDRA (13.0) | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA (13.0) | Systematic Assessment |
|
| Acute tonsillitis | Infections and infestations | MedDRA (13.0) | Systematic Assessment |
|
| Bronchitis | Infections and infestations | MedDRA (13.0) | Systematic Assessment |
|
| Ear infection | Infections and infestations | MedDRA (13.0) | Systematic Assessment |
|
| Erythema infectiosum | Infections and infestations | MedDRA (13.0) | Systematic Assessment |
|
| Febrile infection | Infections and infestations | MedDRA (13.0) | Systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA (13.0) | Systematic Assessment |
|
| Gastrointestinal infection | Infections and infestations | MedDRA (13.0) | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA (13.0) | Systematic Assessment |
|
| Oral herpes | Infections and infestations | MedDRA (13.0) | Systematic Assessment |
|
| Otitis media | Infections and infestations | MedDRA (13.0) | Systematic Assessment |
|
| Otitis media acute | Infections and infestations | MedDRA (13.0) | Systematic Assessment |
|
| Pharyngitis | Infections and infestations | MedDRA (13.0) | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA (13.0) | Systematic Assessment |
|
| Rhinitis | Infections and infestations | MedDRA (13.0) | Systematic Assessment |
|
| Scarlet fever | Infections and infestations | MedDRA (13.0) | Systematic Assessment |
|
| Sinusitis | Infections and infestations | MedDRA (13.0) | Systematic Assessment |
|
| Tonsillitis | Infections and infestations | MedDRA (13.0) | Systematic Assessment |
|
| Tooth abscess | Infections and infestations | MedDRA (13.0) | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA (13.0) | Systematic Assessment |
|
| Varicella | Infections and infestations | MedDRA (13.0) | Systematic Assessment |
|
| Open wound | Injury, poisoning and procedural complications | MedDRA (13.0) | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (13.0) | Systematic Assessment |
|
| Arthritis allergic | Musculoskeletal and connective tissue disorders | MedDRA (13.0) | Systematic Assessment |
|
| Nuchal rigidity | Musculoskeletal and connective tissue disorders | MedDRA (13.0) | Systematic Assessment |
|
| Scoliosis | Musculoskeletal and connective tissue disorders | MedDRA (13.0) | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA (13.0) | Systematic Assessment |
|
| Attention deficit / hyperactivity disorder | Psychiatric disorders | MedDRA (13.0) | Systematic Assessment |
|
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA (13.0) | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA (13.0) | Systematic Assessment |
|
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
| D006728 |
| Hormones |
| D006730 | Hormones, Hormone Substitutes, and Hormone Antagonists |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
Treatment difference Month 24 |
| ANCOVA |
Results from ANCOVA adjusted for baseline growth velocity, target height SDS, sex, and age; LOCF. |
| 0.108 |
| Mean Difference (Final Values) |
| -0.884 |
| Standard Error of the Mean |
| 0.5327 |
| 2-Sided |
| 95 |
| -1.977 |
| 0.208 |
| Superiority or Other |
Treatment difference Month 24 |
| ANCOVA |
Results from ANCOVA adjusted for baseline height, target height SDS, sex, and age; LOCF. |
| <0.001 |
| Mean Difference (Final Values) |
| 4.064 |
| Standard Error of the Mean |
| 0.9822 |
| 2-Sided |
| 95 |
| 2.049 |
| 6.080 |
| Superiority or Other |
Treatment difference Month 24 |
| ANCOVA |
Results from ANCOVA adjusted for baseline skinfold thickness, target height SDS, age, and sex; LOCF. |
| 0.537 |
| Mean Difference (Final Values) |
| -0.48 |
| Standard Error of the Mean |
| 0.758 |
| 2-Sided |
| 95 |
| -2.04 |
| 1.09 |
| Superiority or Other |
Treatment difference Month 24 |
| ANCOVA |
Results from ANCOVA adjusted for baseline skinfold thickness, target height SDS, sex, and age; LOCF. |
| 0.506 |
| Mean Difference (Final Values) |
| -0.23 |
| Standard Error of the Mean |
| 0.345 |
| 2-Sided |
| 95 |
| -0.94 |
| 0.48 |
| Superiority or Other |
Treatment difference Month 24 |
| ANCOVA |
Results from ANCOVA adjusted for baseline skinfold thickness, target height SDS, sex, and age; LOCF. |
| 0.919 |
| Mean Difference (Final Values) |
| 0.11 |
| Standard Error of the Mean |
| 1.065 |
| 2-Sided |
| 95 |
| -2.11 |
| 2.32 |
| Superiority or Other |
Treatment difference Month 24 |
| ANCOVA |
Results from ANCOVA adjusted for baseline volumetric cortical bone mineral density SDS and target height SDS; LOCF. |
| 0.300 |
| Mean Difference (Final Values) |
| 0.717 |
| Standard Error of the Mean |
| 0.6027 |
| 2-Sided |
| 95 |
| -0.956 |
| 2.391 |
| Superiority or Other |
Treatment difference Month 24 |
| ANCOVA |
Results from ANCOVA adjusted for baseline cortical cross-sectional area SDS and target height SDS; LOCF. |
| 0.251 |
| Mean Difference (Final Values) |
| -1.205 |
| Standard Error of the Mean |
| 0.8991 |
| 2-Sided |
| 95 |
| -3.701 |
| 1.292 |
| Superiority or Other |
Treatment difference Month 24 |
| ANCOVA |
Results from ANCOVA adjusted for baseline total cross-sectional area SDS and target height SDS; LOCF. |
| 0.547 |
| Mean Difference (Final Values) |
| 0.292 |
| Standard Error of the Mean |
| 0.4432 |
| 2-Sided |
| 95 |
| -0.939 |
| 1.522 |
| Superiority or Other |
Treatment difference Month 24 |
| ANCOVA |
Results from ANCOVA adjusted for baseline muscle cross-sectional area SDS and target height SDS; LOCF. |
| 0.068 |
| Mean Difference (Final Values) |
| 1.626 |
| Standard Error of the Mean |
| 0.6536 |
| 2-Sided |
| 95 |
| -0.189 |
| 3.440 |
| Superiority or Other |
Treatment difference Month 24 |
| ANCOVA |
Results from ANCOVA adjusted for baseline strength-strain index SDS and target height SDS; LOCF. |
| 0.820 |
| Mean Difference (Final Values) |
| 0.114 |
| Standard Error of the Mean |
| 0.4703 |
| 2-Sided |
| 95 |
| -1.192 |
| 1.420 |
| Superiority or Other |
Treatment difference Month 24 |
| ANCOVA |
Results from ANCOVA adjusted for baseline hand grip strength SDS and target height SDS; LOCF. |
| 0.519 |
| Mean Difference (Final Values) |
| 0.22 |
| Standard Error of the Mean |
| 0.329 |
| 2-Sided |
| 95 |
| -0.52 |
| 0.96 |
| Superiority or Other |
| Title | Measurements |
|---|---|
|
| Month 24: intolerant |
|
| Title | Measurements |
|---|---|
|
| Month 36: intolerant |
|
Treatment difference Month 24 |
| Mixed models analysis |
Results from repeated measures mixed models analysis adjusted for baseline height SDS, visit, visit*treatment and target height SDS. |
| <0.001 |
| Mean Difference (Final Values) |
| 0.688 |
| Standard Error of the Mean |
| 0.1519 |
| 2-Sided |
| 95 |
| 0.382 |
| 0.994 |
| Superiority or Other |
Treatment difference Month 24 |
| Mixed models analysis |
Results from repeated measure mixed models analysis adjusted for baseline height, sex, age, visit, visit*treatment and target height SDS. |
| <0.001 |
| Mean Difference (Final Values) |
| 3.563 |
| Standard Error of the Mean |
| 0.7080 |
| 2-Sided |
| 95 |
| 2.134 |
| 4.992 |
| Superiority or Other |