Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

Study Aimed At Improving Height With Genotonorm In Children Born Little And/Or Light With Growth Retardation At The Age

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00174252
Recruitment Status : Completed
First Posted : September 15, 2005
Results First Posted : June 4, 2010
Last Update Posted : June 15, 2010
Sponsor:
Information provided by:
Pfizer

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Fetal Growth Retardation
Intervention Drug: Genotonorm (Somatropin)
Enrollment 57
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Genotonorm
Hide Arm/Group Description Initiated at a dose of 0.40 milligrams (mg)/kilograms (kg) of Genotonorm divided in 7 daily subcutaneous injections and titrated as needed.
Period Title: Overall Study
Started 57
Completed 55
Not Completed 2
Reason Not Completed
Protocol Violation             2
Arm/Group Title Genotonorm
Hide Arm/Group Description Initiated at a dose of 0.40 milligrams (mg)/kilograms (kg) of Genotonorm divided in 7 daily subcutaneous injections and titrated as needed.
Overall Number of Baseline Participants 57
Hide Baseline Analysis Population Description
[Not Specified]
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 57 participants
<4 years 1
Between 4 and 8 years 49
Between 8 and 12 years 7
>=12 years 0
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 57 participants
Female
34
  59.6%
Male
23
  40.4%
1.Primary Outcome
Title Percentage of Children With Insulin Growth Factor-1 (IGF-1) > 2 Standard Deviation (SD) at 9 and 12 Months
Hide Description Percentage of children with serum IGF-1 > 2 SD (compared to a child of the same gender and age and without growth hormone (GH) deficiency) 9 months and 12 months after initiation of GH treatment. 9 months and 12 months are combined.
Time Frame 9 and 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
The safety analysis set (SAS) was defined as all patients who received at least one dose of GH treatment. Number of Participants Analyzed = Number of children treated.
Arm/Group Title Genotonorm
Hide Arm/Group Description:
Initiated at a dose of 0.40 milligrams (mg)/kilograms (kg) of Genotonorm divided in 7 daily subcutaneous injections and titrated as needed.
Overall Number of Participants Analyzed 57
Measure Type: Number
Unit of Measure: percentage of participants
No 86.0
Yes 14.0
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Genotonorm
Comments Applies to "no".
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Method of Estimation Estimation Parameter Percentage
Estimated Value 86.0
Confidence Interval (2-Sided) 95%
74.2 to 93.7
Estimation Comments 95% CI was estimated using the F-distribution.
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Genotonorm
Comments Applies to "yes".
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Method of Estimation Estimation Parameter Percentage
Estimated Value 14.0
Confidence Interval 95%
6.3 to 25.8
Estimation Comments 95% confidence interval (CI) was estimated using the F-distribution.
2.Secondary Outcome
Title Change in Height SD Chronological Age (CA) From Baseline at 12 Months in Children With IGF-1 < = 2 SD at 9 or 12 Months
Hide Description The height in SD was calculated using Sempe reference means and standard deviations for height. Change in height SD was calculated as height in SD at "12 months” minus height in SD at "Baseline".
Time Frame Baseline, 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis set (FAS) included all patients who received at least one dose of assigned treatment and had at least one subsequent rating of IGF-1. Number of Participants Analyzed = Number of children with IGF-1 < = 2 SD at 9 or 12 months with evaluable height SD CA data at 12 months.
Arm/Group Title Genotonorm
Hide Arm/Group Description:
Initiated at a dose of 0.40 milligrams (mg)/kilograms (kg) of Genotonorm divided in 7 daily subcutaneous injections and titrated as needed.
Overall Number of Participants Analyzed 46
Mean (Standard Deviation)
Unit of Measure: SD for CA
0.81  (0.26)
3.Secondary Outcome
Title Change in Height SD CA From Baseline at 24 Months in Children With IGF-1 < = 2 SD at 9 or 12 Months
Hide Description The height in SD was calculated using Sempe reference means and SDs for height. Change in height SD was calculated as height in SD at “24 months” minus height in SD at "Baseline".
Time Frame Baseline, 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
FAS. Number of Participants Analyzed = Number of children with IGF-1 < = 2 SD at 9 or 12 months with evaluable height SD CA data at 24 months.
Arm/Group Title Genotonorm
Hide Arm/Group Description:
Initiated at a dose of 0.40 milligrams (mg)/kilograms (kg) of Genotonorm divided in 7 daily subcutaneous injections and titrated as needed.
Overall Number of Participants Analyzed 47
Mean (Standard Deviation)
Unit of Measure: SD for CA
1.24  (0.48)
4.Secondary Outcome
Title Change in Height SD CA From Baseline at 12 and 24 Months in Children With IGF-1 > 2 SD at 9 and 12 Months
Hide Description The height in SD was calculated using Sempe reference means and SDs for height. Change in height SD was calculated as height in SD at “12 or 24 months” minus height in SD at “Baseline".
Time Frame Baseline, 12 and 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
FAS. Number of Participants Analyzed = Number of children with IGF-1 > 2 SD at 9 and 12 months with evaluable Height SD CA data at 12 and 24 months.
Arm/Group Title Genotonorm
Hide Arm/Group Description:
Initiated at a dose of 0.40 milligrams (mg)/kilograms (kg) of Genotonorm divided in 7 daily subcutaneous injections and titrated as needed.
Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: SD for BA
12 Months 1.13  (0.35)
24 Months 1.53  (0.20)
5.Secondary Outcome
Title Change in Height SD Bone Age (BA) From Baseline at 12 Months in Children With IGF-1 < = 2 SD at 9 or 12 Months
Hide Description The height in SD was calculated using Sempe reference means and SDs for height. Change in height SD was calculated as height in SD at “12 months” minus height in SD at "Baseline".
Time Frame Baseline, 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
FAS. Number of Participants Analyzed = Number of children with IGF-1 < = 2 SD at 9 or 12 months with evaluable height SD BA data at 12 months.
Arm/Group Title Genotonorm
Hide Arm/Group Description:
Initiated at a dose of 0.40 milligrams (mg)/kilograms (kg) of Genotonorm divided in 7 daily subcutaneous injections and titrated as needed.
Overall Number of Participants Analyzed 33
Mean (Standard Deviation)
Unit of Measure: SD for BA
0.44  (1.34)
6.Secondary Outcome
Title Change in Height SD BA From Baseline at 24 Months in Children With IGF-1 < = 2 SD at 9 or 12 Months
Hide Description The height in SD was calculated using Sempe reference means and SDs for height. Change in height SD was calculated as height in SD at “24 months” minus height in SD at “Baseline".
Time Frame Baseline, 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
FAS. Number of Participants Analyzed = Number of children with IGF-1 < = 2 SD at 9 or 12 months with evaluable height SD BA data at 24 months.
Arm/Group Title Genotonorm
Hide Arm/Group Description:
Initiated at a dose of 0.40 milligrams (mg)/kilograms (kg) of Genotonorm divided in 7 daily subcutaneous injections and titrated as needed.
Overall Number of Participants Analyzed 31
Mean (Standard Deviation)
Unit of Measure: Height in SD
0.12  (1.86)
7.Secondary Outcome
Title Change in Height SD BA From Baseline at 12 and 24 Months in Children With IGF-1 > 2 SD at 9 and 12 Months
Hide Description The height in SD was calculated using Sempe reference means and SDs for height. Change in height SD was calculated as height in SD at “12 or 24 months” minus height in SD at “Baseline".
Time Frame Baseline, 12 and 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
FAS. Number of Participants Analyzed = Number of children with IGF-1 > 2 SD at 9 or 12 months with evaluable height SD BA data at 12 and 12 months.
Arm/Group Title Genotonorm
Hide Arm/Group Description:
Initiated at a dose of 0.40 milligrams (mg)/kilograms (kg) of Genotonorm divided in 7 daily subcutaneous injections and titrated as needed.
Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: SD for BA
12 Months 0.64  (1.19)
24 Months 0.64  (1.30)
8.Secondary Outcome
Title Summary of Growth Rate SD (CA) at 12 and 24 Months in Children With IGF-1 < = 2 SD at 9 or 12 Months
Hide Description

Growth rate SD CA at 12 months was calculated as:(Growth rate at 12 months minus reference mean for CA at 12 months) divided by reference SD for CA at 12 months

Growth rate SD CA at 24 months was calculated as:(Growth rate at 24 months minus reference mean for CA at 24 months) divided by reference SD for CA at 24 months

Time Frame 12 and 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
FAS. Number of Participants Analyzed = Number of children with IGF-1 < = 2 SD at 9 or 12 months with evaluable growth rate SD CA data at 12 months. n = Number of children with IGF-1 < = 2 SD at 9 or 12 months with evaluable growth rate SD CA data at 24 months.
Arm/Group Title Genotonorm
Hide Arm/Group Description:
Initiated at a dose of 0.40 milligrams (mg)/kilograms (kg) of Genotonorm divided in 7 daily subcutaneous injections and titrated as needed.
Overall Number of Participants Analyzed 49
Mean (Standard Deviation)
Unit of Measure: SD for CA
12 Months 3.77  (1.68)
24 Months (n=47) 1.74  (1.51)
9.Secondary Outcome
Title Summary of Growth Rate SD (CA) at 12 and 24 Months in Children With IGF-1 > 2 SD at 9 and 12 Months
Hide Description

Growth rate SD CA at 12 months was calculated as: (Growth rate at 12 months minus reference mean for CA at 12 months) divided by reference SD for CA at 12 months

Growth rate SD CA at 24 months was calculated as: (Growth rate at 24 months minus reference mean for CA at 24 months) divided by reference SD for CA at 24 months

Time Frame 12 and 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
FAS. Number of Participants Analyzed = Number of children with IGF-1 > 2 SD at 9 and 12 months with evaluable growth rate SD CA data at 12 and 24 months.
Arm/Group Title Genotonorm
Hide Arm/Group Description:
Initiated at a dose of 0.40 milligrams (mg)/kilograms (kg) of Genotonorm divided in 7 daily subcutaneous injections and titrated as needed.
Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: SD for CA
12 Months 5.24  (1.64)
24 Months 2.15  (2.21)
10.Secondary Outcome
Title Summary of Growth Rate SD (BA) at 12 Months in Children With IGF-1 < = 2 SD at 9 or 12 Months
Hide Description Growth rate SD BA at 12 months was calculated as: (Growth rate at 12 months minus reference mean for BA at 12 months) divided by reference SD for BA at 12 months
Time Frame Baseline, 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
FAS. Number of Participants Analyzed = Number of children with IGF-1 < = 2 SD at 9 or 12 months with evaluable growth rate SD BA data at 12 months.
Arm/Group Title Genotonorm
Hide Arm/Group Description:
Initiated at a dose of 0.40 milligrams (mg)/kilograms (kg) of Genotonorm divided in 7 daily subcutaneous injections and titrated as needed.
Overall Number of Participants Analyzed 33
Mean (Standard Deviation)
Unit of Measure: SD for CA
2.38  (1.88)
11.Secondary Outcome
Title Summary of Growth Rate SD (BA) at 24 Months in Children With IGF-1 < = 2 SD at 9 or 12 Months
Hide Description Growth rate SD BA at 24 months was calculated as: (Growth rate at 24 months minus reference mean for BA at 24 months) divided by reference SD for BA at 24 months
Time Frame Baseline, 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
FAS. Number of Participants Analyzed = Number of children with IGF-1 < = 2 SD at 9 or 12 months with evaluable growth rate SD BA data at 24 months.
Arm/Group Title Genotonorm
Hide Arm/Group Description:
Initiated at a dose of 0.40 milligrams (mg)/kilograms (kg) of Genotonorm divided in 7 daily subcutaneous injections and titrated as needed.
Overall Number of Participants Analyzed 39
Mean (Standard Deviation)
Unit of Measure: SD for BA
1.26  (1.62)
12.Secondary Outcome
Title Summary of Growth Rate SD (BA) at 12 and 24 Months in Children With IGF-1 > 2 SD at 9 and 12 Months
Hide Description

Growth rate SD BA at 12 months was calculated as: (Growth rate at 12 months minus reference mean for BA at 12 months) divided by reference SD for CA at 12 months

Growth rate SD BA at 24 months was calculated as: (Growth rate at 24 months minus reference mean for BA at 24 months) divided by reference SD for CA at 24 months

Time Frame 12 and 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
FAS. Number of Participants Analyzed = Number of children with IGF-1 > 2 SD at 9 and 12 months with evaluable growth rate SD BA data at 12 months. n = Number of children with IGF-1 > 2 SD at 9 and 12 months with evaluable growth rate SD BA data at 24 months.
Arm/Group Title Genotonorm
Hide Arm/Group Description:
Initiated at a dose of 0.40 milligrams (mg)/kilograms (kg) of Genotonorm divided in 7 daily subcutaneous injections and titrated as needed.
Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: SD for BA
12 Months 4.02  (2.37)
24 Months (n=6) 1.71  (2.26)
13.Secondary Outcome
Title Analysis of Covariance (ANCOVA) for Height SD CA at 12 Months in Children With IGF-1 < = 2 SD at 9 or 12 Months and IGF-1 > 2 SD at 9 and 12 Months
Hide Description

Change in Height SD (CA) was calculated as:

Height SD (CA) at 12 months minus Height SD (CA) at Baseline

Time Frame Baseline, 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
FAS. Number of Participants Analyzed = Number of children with IGF-1 < = 2 SD at 9 or 12 months or with IGF-1 > 2 SD at 9 and 12 months and with evaluable height SD CA data at 12 months.
Arm/Group Title Genotonorm (IGF-1 < = 2 SD at 9 or 12 Months) Genotonorm (IGF-1 > 2 SD at 9 and 12 Months)
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 46 8
Mean (Standard Error)
Unit of Measure: SD for CA
-1.94  (0.04) -1.59  (0.10)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Genotonorm (IGF-1 < = 2 SD at 9 or 12 Months), Genotonorm (IGF-1 > 2 SD at 9 and 12 Months)
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.002
Comments [Not Specified]
Method ANCOVA
Comments Adjusted for height SD at baseline.
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.35
Estimation Comments [Not Specified]
14.Secondary Outcome
Title ANCOVA for Height SD CA at 24 Months in Children With IGF-1 < = 2 SD at 9 or 12 Months and IGF-1 > 2 SD at 9 and 12 Months
Hide Description Height SD (CA) at 24 months.
Time Frame Baseline, 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
FAS. Number of Participants Analyzed = Number of children with IGF-1 < = 2 SD at 9 or 12 months or with IGF-1 > 2 SD at 9 and 12 months and with evaluable height SD CA data at 24 months.
Arm/Group Title Genotonorm (IGF-1 < = 2 SD at 9 or 12 Months) Genotonorm (IGF-1 > 2 SD at 9 and 12 Months)
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 44 8
Mean (Standard Error)
Unit of Measure: SD for CA
-1.53  (0.06) -1.18  (0.14)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Genotonorm (IGF-1 < = 2 SD at 9 or 12 Months), Genotonorm (IGF-1 > 2 SD at 9 and 12 Months)
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.023
Comments Bilateral test multiple comparison threshold for statistical significance = 0.05
Method ANCOVA
Comments Adjusted for height SD at baseline.
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.35
Estimation Comments [Not Specified]
15.Secondary Outcome
Title ANCOVA for Height SD BA at 12 Months in Children With IGF-1 < = 2 SD at 9 or 12 Months and IGF-1 > 2 SD at 9 and 12 Months
Hide Description

Change in Height SD (BA) was calculated as:

Height SD (BA) at 12 months minus Height SD (BA) at Baseline

Time Frame Baseline, 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
FAS. Number of Participants Analyzed = Number of children with IGF-1 < = 2 SD at 9 or 12 months or with IGF-1 > 2 SD at 9 and 12 months and with evaluable height SD BA data at 12 months.
Arm/Group Title Genotonorm (IGF-1 < = 2 SD at 9 or 12 Months) Genotonorm (IGF-1 > 2 SD at 9 and 12 Months)
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 33 7
Mean (Standard Error)
Unit of Measure: SD for BA
0.56  (0.22) 0.76  (0.48)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Genotonorm (IGF-1 < = 2 SD at 9 or 12 Months), Genotonorm (IGF-1 > 2 SD at 9 and 12 Months)
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.708
Comments [Not Specified]
Method ANCOVA
Comments Adjusted for height SD at baseline.
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value -0.20
Estimation Comments [Not Specified]
16.Secondary Outcome
Title ANCOVA for Height SD BA at 24 Months in Children With IGF-1 <= 2 SD at 9 or 12 Months and IGF-1 > 2 SD at 9 and 12 Months
Hide Description Height SD (BA) at 24 months
Time Frame Baseline, 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
FAS. Number of Participants Analyzed = Number of children with IGF-1 < = 2 SD at 9 or 12 months or with IGF-1 > 2 SD at 9 and 12 months and with evaluable height SD BA data at 24 months.
Arm/Group Title Genotonorm (IGF-1 < = 2 SD at 9 or 12 Months) Genotonorm (IGF-1 > 2 SD at 9 and 12 Months)
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 31 7
Mean (Standard Error)
Unit of Measure: SD for BA
0.05  (0.25) 0.64  (0.53)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Genotonorm (IGF-1 < = 2 SD at 9 or 12 Months), Genotonorm (IGF-1 > 2 SD at 9 and 12 Months)
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.325
Comments [Not Specified]
Method ANCOVA
Comments Adjusted for height SD at baseline.
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.59
Estimation Comments [Not Specified]
17.Secondary Outcome
Title Summary of IGF-1 SD at 6, 9, 12, 15, 18, 21, and 24 Months in Children With IGF-1 < = 2 SD at 9 or 12 Months
Hide Description IGF-1 SD was calculated at each study time point using gender specific IGF-1 reference mean and SD for the CA of the child on the date of the corresponding IGF-1 blood sample: IGF-1 SD = (IGF-1 minus reference mean) divided by reference SD
Time Frame 6, 9, 12, 15, 18, 21, and 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
SAS. Number of Participants Analyzed = Number of children treated. n = Number of children with IGF-1 < = 2 SD at 9 or 12 months with evaluable IGF-1 SD data at 6, 9, 12, 15, 18, 21, and 24 months.
Arm/Group Title Genotonorm
Hide Arm/Group Description:
Initiated at a dose of 0.40 milligrams (mg)/kilograms (kg) of Genotonorm divided in 7 daily subcutaneous injections and titrated as needed.
Overall Number of Participants Analyzed 57
Mean (Standard Deviation)
Unit of Measure: SD
6 Months (n=49) 0.65  (1.11)
9 Months (n=46) 0.93  (1.26)
12 Months (n=44) 1.04  (0.96)
15 Months (n=40) 0.88  (1.28)
18 Months (n=43) 0.88  (1.17)
21 Months (n=41) 0.82  (0.93)
24 Months (n=45) 0.85  (1.17)
18.Secondary Outcome
Title Summary of IGF-1 SD at 6, 9, 12, 15, 18, 21, and 24 Months in Children With IGF-1 > 2 SD at 9 and 12 Months
Hide Description IGF-1 SD was calculated at each study time point using these gender specific IGF-1 reference means and SDs for the CA of the child on the date of the corresponding IGF-1 blood sample: IGF-1 SD = (IGF-1 – reference mean) / reference SD
Time Frame 6, 9, 12, 15, 18, 21, and 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
SAS. Number of Participants Analyzed = Number of children treated. n = Number of children with IGF-1 > 2 SD at 9 and 12 months with evaluable IGF-1 SD data at 6, 9, 12, 15, 18, 21, and 24 months.
Arm/Group Title Genotonorm
Hide Arm/Group Description:
Initiated at a dose of 0.40 milligrams (mg)/kilograms (kg) of Genotonorm divided in 7 daily subcutaneous injections and titrated as needed.
Overall Number of Participants Analyzed 57
Mean (Standard Deviation)
Unit of Measure: SD
6 Months (n=7) 2.32  (0.68)
9 Months (n=6) 2.35  (0.19)
12 Months (n=8) 2.50  (0.27)
15 Months (n=7) 1.40  (1.08)
18 Months (n=7) 2.12  (1.18)
21 Months (n=7) 1.82  (1.09)
24 Months (n=8) 1.81  (1.03)
19.Secondary Outcome
Title IGF-1/Insulin-Like Growth Factor Binding Protein 3 (IGFBP-3) Ratio at 12 and 24 Months in Children With IGF-1 < = 2 SD at 9 or 12 Months
Hide Description [Not Specified]
Time Frame 12 and 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
SAS. Number of Participants Analyzed = Number of children treated. n = Number of children with IGF-1 < = 2 SD at 9 or 12 months with evaluable IGF-1/IGFBP-3 data at 12 and 24 months.
Arm/Group Title Genotonorm
Hide Arm/Group Description:
Initiated at a dose of 0.40 milligrams (mg)/kilograms (kg) of Genotonorm divided in 7 daily subcutaneous injections and titrated as needed.
Overall Number of Participants Analyzed 57
Mean (Standard Deviation)
Unit of Measure: ratio
12 Months (n=44) 0.17  (0.06)
24 Months (n=45) 0.19  (0.07)
20.Secondary Outcome
Title IGF-1/IGFBP-3 Ratio at 12 and 24 Months in Children With IGF-1 > 2 SD at 9 and 12 Months
Hide Description [Not Specified]
Time Frame 12 and 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
SAS. Number of Participants Analyzed = Number of children with IGF-1 > 2 SD at 9 and 12 months with evaluable IGF-1/IGFBP-3 data at 12 and 24 months.
Arm/Group Title Genotonorm
Hide Arm/Group Description:
Initiated at a dose of 0.40 milligrams (mg)/kilograms (kg) of Genotonorm divided in 7 daily subcutaneous injections and titrated as needed.
Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: ratio
12 Months 0.23  (0.03)
24 Months 0.21  (0.06)
21.Other Pre-specified Outcome
Title Change in Height From Baseline
Hide Description The standing height measurements were performed at the same time of the day by using a wallmounted device (e.g. Harpenden Stadiometer) at each study visit. The pre-specified clinical outcomes were analyzed at 12 and 24 months.
Time Frame Baseline, 12 and 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
FAS. Number of Participants Analyzed = Number of children with evaluable height data at 12 and 24 months.
Arm/Group Title Genotonorm
Hide Arm/Group Description:
Initiated at a dose of 0.40 milligrams (mg)/kilograms (kg) of Genotonorm divided in 7 daily subcutaneous injections and titrated as needed.
Overall Number of Participants Analyzed 55
Mean (Standard Deviation)
Unit of Measure: centimeters (cm)
12 Months 9.0  (1.3)
24 Months 16.4  (2.2)
22.Other Pre-specified Outcome
Title Growth Rate at 12 and 24 Months
Hide Description

Growth Rate was calculated at 12 months as:

(Height at 12 months minus Height at Day 0) divided by {(Date of 12 months minus Date of Day 0) divided by 365.25}

Growth Rate was calculated at 24 months as:

(Height at 24 months minus Height at 12 months) divided by {(Date of 24 months minus Date of 12 months) divided by 365.25}

Time Frame 12 and 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
FAS. Number of Participants Analyzed = Number of children treated. n = Number of children with evaluable growth rate data at 12 and 24 months.
Arm/Group Title Genotonorm
Hide Arm/Group Description:
Initiated at a dose of 0.40 milligrams (mg)/kilograms (kg) of Genotonorm divided in 7 daily subcutaneous injections and titrated as needed.
Overall Number of Participants Analyzed 57
Mean (Standard Deviation)
Unit of Measure: cm/year
12 Months 9.06  (1.40)
24 Months (n=55) 7.23  (1.38)
23.Other Pre-specified Outcome
Title Summary of Body Mass Index (BMI) at 12 and 24 Months
Hide Description BMI was calculated at 12 months and 24 months as: (Weight at 12 or 24 months divided by Height at 12 or 24 months) squared
Time Frame 12 and 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
FAS. Number of Participants Analyzed = Number of children treated. n = Number of children with evaluable BMI data at 12 and 24 months.
Arm/Group Title Genotonorm
Hide Arm/Group Description:
Initiated at a dose of 0.40 milligrams (mg)/kilograms (kg) of Genotonorm divided in 7 daily subcutaneous injections and titrated as needed.
Overall Number of Participants Analyzed 57
Mean (Standard Deviation)
Unit of Measure: kg/meters squared (m2)
12 Months (n=54) 14.7  (1.5)
24 Months (n=55) 14.9  (1.4)
24.Other Pre-specified Outcome
Title Change in BA From Baseline at 12 and 24 Months
Hide Description Change in BA was calculated as: (12 or 24 months minus Screening)
Time Frame Baseline, 12 and 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
FAS. Number of Participants Analyzed = Number of children treated. n = Number of children with evaluable BA data at 12 and 24 months.
Arm/Group Title Genotonorm
Hide Arm/Group Description:
Initiated at a dose of 0.40 milligrams (mg)/kilograms (kg) of Genotonorm divided in 7 daily subcutaneous injections and titrated as needed.
Overall Number of Participants Analyzed 57
Mean (Standard Deviation)
Unit of Measure: year
12 Months (n=33) 1.2  (0.9)
24 Months (n=31) 2.5  (1.1)
25.Other Pre-specified Outcome
Title BA/CA at 12 and 24 Months in Children With IGF-1 < = 2 SD at 9 or 12 Months
Hide Description The BA/CA ratio was calculated at Screening, 12 and 24 months. The CA was the age on the date that the corresponding BA X-ray was performed.
Time Frame 12 and 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
FAS. Number of Participants Analyzed = Number of children with IGF-1 < = 2 SD at 9 or 12 months with evaluable BA/CA data at 12 and 24 months.
Arm/Group Title Genotonorm
Hide Arm/Group Description:
Initiated at a dose of 0.40 milligrams (mg)/kilograms (kg) of Genotonorm divided in 7 daily subcutaneous injections and titrated as needed.
Overall Number of Participants Analyzed 43
Mean (Standard Deviation)
Unit of Measure: ratio
12 Months 0.74  (0.17)
24 Months 0.82  (0.16)
26.Other Pre-specified Outcome
Title BA/CA at 12 and 24 Months in Children With IGF-1 > 2 SD at 9 and 12 Months
Hide Description The BA/CA ratio was calculated at Screening, 12 and 24 months. The CA was the age on the date that the corresponding BA X-ray was performed.
Time Frame 12 and 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
FAS. Number of Participants Analyzed = Number of children with IGF-1 > 2 SD at 9 and 12 months with evaluable BA/CA data at 12 and 24 months.
Arm/Group Title Genotonorm
Hide Arm/Group Description:
Initiated at a dose of 0.40 milligrams (mg)/kilograms (kg) of Genotonorm divided in 7 daily subcutaneous injections and titrated as needed.
Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: ratio
12 Months 0.80  (0.21)
24 Months 0.83  (0.09)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Genotonorm
Hide Arm/Group Description Initiated at a dose of 0.40 milligrams (mg)/kilograms (kg) of Genotonorm divided in 7 daily subcutaneous injections and titrated as needed.
All-Cause Mortality
Genotonorm
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Genotonorm
Affected / at Risk (%)
Total   6/57 (10.53%) 
Congenital, familial and genetic disorders   
Amniotic band syndrome  1  1/57 (1.75%) 
Ear and labyrinth disorders   
Deafness  1  1/57 (1.75%) 
Infections and infestations   
Pneumonia  1  1/57 (1.75%) 
Staphylococcal infection  1  1/57 (1.75%) 
Renal and urinary disorders   
Dysuria  1  1/57 (1.75%) 
Haematuria  1  1/57 (1.75%) 
Respiratory, thoracic and mediastinal disorders   
Pleurisy  1  1/57 (1.75%) 
Tonsillar hypertrophy  1  1/57 (1.75%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (12.1)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Genotonorm
Affected / at Risk (%)
Total   30/57 (52.63%) 
Blood and lymphatic system disorders   
Microcytosis  1  1/57 (1.75%) 
Ear and labyrinth disorders   
Tympanic membrane perforation  1  1/57 (1.75%) 
Eye disorders   
Conjunctivitis  1  1/57 (1.75%) 
Conjunctivitis allergic  1  1/57 (1.75%) 
Eyelid oedema  1  1/57 (1.75%) 
Gastrointestinal disorders   
Constipation  1  4/57 (7.02%) 
Diarrhoea  1  1/57 (1.75%) 
General disorders   
Fatigue  1  1/57 (1.75%) 
Pain  1  1/57 (1.75%) 
Pyrexia  1  1/57 (1.75%) 
Immune system disorders   
House dust allergy  1  1/57 (1.75%) 
Infections and infestations   
Bronchiolitis  1  1/57 (1.75%) 
Bronchitis  1  5/57 (8.77%) 
Ear infection  1  3/57 (5.26%) 
Furuncle  1  1/57 (1.75%) 
Gastroenteritis  1  3/57 (5.26%) 
Influenza  1  1/57 (1.75%) 
Laryngitis  1  1/57 (1.75%) 
Lyme disease  1  1/57 (1.75%) 
Nasopharyngitis  1  4/57 (7.02%) 
Oral herpes  1  1/57 (1.75%) 
Otitis media  1  1/57 (1.75%) 
Pharyngitis  1  1/57 (1.75%) 
Pneumonia  1  1/57 (1.75%) 
Rhinitis  1  4/57 (7.02%) 
Skin infection  1  1/57 (1.75%) 
Tinea infection  1  1/57 (1.75%) 
Tonsillitis  1  2/57 (3.51%) 
Upper respiratory tract infection  1  1/57 (1.75%) 
Varicella  1  6/57 (10.53%) 
Viral infection  1  1/57 (1.75%) 
Vulvitis  1  1/57 (1.75%) 
Investigations   
Insulin-like growth factor increased  1  3/57 (5.26%) 
Musculoskeletal and connective tissue disorders   
Arthralgia  1  1/57 (1.75%) 
Pain in extremity  1  2/57 (3.51%) 
Nervous system disorders   
Headache  1  5/57 (8.77%) 
Psychiatric disorders   
Sleep disorder  1  1/57 (1.75%) 
Sleep terror  1  1/57 (1.75%) 
Renal and urinary disorders   
Enuresis  1  1/57 (1.75%) 
Respiratory, thoracic and mediastinal disorders   
Cough  1  3/57 (5.26%) 
Nasal polyps  1  1/57 (1.75%) 
Pharyngeal erythema  1  1/57 (1.75%) 
Productive cough  1  1/57 (1.75%) 
Respiratory disorder  1  1/57 (1.75%) 
Skin and subcutaneous tissue disorders   
Eczema  1  1/57 (1.75%) 
Lipodystrophy acquired  1  1/57 (1.75%) 
Rash  1  1/57 (1.75%) 
Surgical and medical procedures   
Adenotonsillectomy  1  1/57 (1.75%) 
Tonsillectomy  1  1/57 (1.75%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (12.1)
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Pfizer ClinicalTrials.gov Call Center
Organization: Pfizer, Inc.
Phone: 1-800-718-1021
Responsible Party: Director, Clinical Trial Disclosure Group, Pfizer, Inc.
ClinicalTrials.gov Identifier: NCT00174252     History of Changes
Other Study ID Numbers: A6281234
First Submitted: September 9, 2005
First Posted: September 15, 2005
Results First Submitted: April 2, 2010
Results First Posted: June 4, 2010
Last Update Posted: June 15, 2010