Trial Outcomes & Findings for Efficacy and Safety Evaluation of Recombinant Human Growth Hormone (r-hGH), Saizen®, on a Population of Children With Hypochondroplasia, Treated at Least 3 Years or Until Near Final Height, When Applicable, in Comparison With a Historic Cohort of Non-treated Children (NCT NCT01111019)
NCT ID: NCT01111019
Last Updated: 2019-02-15
Results Overview
Height was calculated in standardized units expressed as the standard deviation score (SDS), where SDS = (x - m)/sigma, in which x represents the height variable measured by sex and age, and m and sigma are the statistical parameters (mean and standard deviation) for the Sempe reference population. The reference population was the historical cohort consisting of non-treated subjects with Hypochondroplasia (HCH). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) a participant's value was relative to the mean of the reference population. The scores were centered around zero. Negative score indicated smaller height for the respective age/gender.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
19 participants
Primary outcome timeframe
Baseline (Month 0), Year 3
Results posted on
2019-02-15
Participant Flow
The study was conducted between March 21, 2006 and January 17, 2017.
Participant milestones
| Measure |
r-hGH (Saizen®)
Subjects received a single subcutaneous injection of recombinant human growth hormone (r-hGH) equivalent to dose of 0.057 milligram per kilogram per day (mg/kg/day). The dose was subsequently reduced to 0.035 mg/kg/day for subjects, who continued treatment after 31 January 2011. Subjects were treated for at least 3 years or until near final height was reached.
|
|---|---|
|
Overall Study
STARTED
|
19
|
|
Overall Study
COMPLETED
|
16
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
r-hGH (Saizen®)
Subjects received a single subcutaneous injection of recombinant human growth hormone (r-hGH) equivalent to dose of 0.057 milligram per kilogram per day (mg/kg/day). The dose was subsequently reduced to 0.035 mg/kg/day for subjects, who continued treatment after 31 January 2011. Subjects were treated for at least 3 years or until near final height was reached.
|
|---|---|
|
Overall Study
Withdrew Prematurely
|
2
|
|
Overall Study
Other
|
1
|
Baseline Characteristics
Efficacy and Safety Evaluation of Recombinant Human Growth Hormone (r-hGH), Saizen®, on a Population of Children With Hypochondroplasia, Treated at Least 3 Years or Until Near Final Height, When Applicable, in Comparison With a Historic Cohort of Non-treated Children
Baseline characteristics by cohort
| Measure |
r-hGH (Saizen®)
n=19 Participants
Subjects received a single subcutaneous injection of recombinant human growth hormone (r-hGH) equivalent to dose of 0.057 milligram per kilogram per day (mg/kg/day). The dose was subsequently reduced to 0.035 mg/kg/day for subjects, who continued treatment after 31 January 2011. Subjects were treated for at least 3 years or until near final height was reached.
|
|---|---|
|
Age, Continuous
|
9.0 Years
STANDARD_DEVIATION 3.12 • n=5 Participants
|
|
Sex/Gender, Customized
Female
|
11 Participants
n=5 Participants
|
|
Sex/Gender, Customized
Male
|
8 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline (Month 0), Year 3Population: Intention to Treat (ITT) population included all subjects who received at least one dose of treatment with at least an efficacy assessment available during the treatment period.
Height was calculated in standardized units expressed as the standard deviation score (SDS), where SDS = (x - m)/sigma, in which x represents the height variable measured by sex and age, and m and sigma are the statistical parameters (mean and standard deviation) for the Sempe reference population. The reference population was the historical cohort consisting of non-treated subjects with Hypochondroplasia (HCH). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) a participant's value was relative to the mean of the reference population. The scores were centered around zero. Negative score indicated smaller height for the respective age/gender.
Outcome measures
| Measure |
r-hGH (Saizen®)
n=19 Participants
Subjects received a single subcutaneous injection of recombinant human growth hormone (r-hGH) equivalent to dose of 0.057 milligram per kilogram per day (mg/kg/day). The dose was subsequently reduced to 0.035 mg/kg/day for subjects, who continued treatment after 31 January 2011. Subjects were treated for at least 3 years or until near final height was reached.
|
|---|---|
|
Change From Baseline in Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects at Year 3
|
1.39 Standard Deviation Score (SDS)
Standard Deviation 0.9
|
PRIMARY outcome
Timeframe: Year 4Population: Intention to Treat (ITT) population included all subjects who received at least one dose of treatment with at least an efficacy assessment available during the treatment period.
Height was calculated in standardized units expressed as the standard deviation score (SDS), where SDS = (x - m)/sigma, in which x represents the height variable measured by sex and age, and m and sigma are the statistical parameters (mean and standard deviation) for the Sempe reference population. The reference population was the historical cohort consisting of non-treated subjects with Hypochondroplasia (HCH). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) a participant's value was relative to the mean of the reference population. The scores were centered around zero. Negative score indicated smaller height for the respective age/gender.
Outcome measures
| Measure |
r-hGH (Saizen®)
n=19 Participants
Subjects received a single subcutaneous injection of recombinant human growth hormone (r-hGH) equivalent to dose of 0.057 milligram per kilogram per day (mg/kg/day). The dose was subsequently reduced to 0.035 mg/kg/day for subjects, who continued treatment after 31 January 2011. Subjects were treated for at least 3 years or until near final height was reached.
|
|---|---|
|
Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects at Year 4
|
25.6 Standard Deviation Score (SDS)
Standard Deviation 3.5
|
SECONDARY outcome
Timeframe: Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 8.5 (only for female), 9 (only for male) and 9.5 (only for male)Population: Intention to Treat (ITT) population included all subjects who received at least one dose of treatment with at least an efficacy assessment available during the treatment period. Here "Number analyzed" signifies those subjects who were evaluable for this outcome measure for specified category.
Height was calculated in standardized units expressed as the standard deviation score (SDS), where SDS = (x - m)/sigma, in which x represents the height variable measured by sex and age, and m and sigma are the statistical parameters (mean and standard deviation) for the Sempe reference population. The reference population was the historical cohort consisting of non-treated subjects with Hypochondroplasia (HCH). Data was reported by gender. Male subjects were analyzed till 9.5 years and female subjects were analyzed till 8.5 years. SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) a participant's value was relative to the mean of the reference population. The scores were centered around zero. Negative score indicated smaller height for the respective age/gender.
Outcome measures
| Measure |
r-hGH (Saizen®)
n=19 Participants
Subjects received a single subcutaneous injection of recombinant human growth hormone (r-hGH) equivalent to dose of 0.057 milligram per kilogram per day (mg/kg/day). The dose was subsequently reduced to 0.035 mg/kg/day for subjects, who continued treatment after 31 January 2011. Subjects were treated for at least 3 years or until near final height was reached.
|
|---|---|
|
Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects
Male: Year 1
|
-1.82 Standard Deviation Score (SDS)
Interval -3.4 to -1.4
|
|
Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects
Male: Year 4
|
-1.49 Standard Deviation Score (SDS)
Interval -3.7 to -1.0
|
|
Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects
Male: Year 5
|
-1.51 Standard Deviation Score (SDS)
Interval -3.5 to -1.0
|
|
Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects
Male: Year 2
|
-1.51 Standard Deviation Score (SDS)
Interval -3.4 to -1.1
|
|
Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects
Male: Year 3
|
-1.30 Standard Deviation Score (SDS)
Interval -3.7 to -1.0
|
|
Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects
Male: Year 6
|
-2.88 Standard Deviation Score (SDS)
Interval -3.8 to -2.0
|
|
Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects
Male: Year 7
|
-3.9 Standard Deviation Score (SDS)
Interval -3.9 to -3.9
|
|
Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects
Male: Year 8
|
-3.90 Standard Deviation Score (SDS)
Interval -3.9 to -3.9
|
|
Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects
Male: Year 9
|
-5.0 Standard Deviation Score (SDS)
Interval -5.0 to -5.0
|
|
Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects
Male: Year 9.5
|
-5.6 Standard Deviation Score (SDS)
Interval -5.6 to -5.6
|
|
Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects
Female: Year 1
|
-2.69 Standard Deviation Score (SDS)
Interval -5.3 to -0.6
|
|
Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects
Female: Year 2
|
-2.52 Standard Deviation Score (SDS)
Interval -5.8 to -0.7
|
|
Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects
Female: Year 3
|
-2.61 Standard Deviation Score (SDS)
Interval -6.0 to -0.6
|
|
Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects
Female: Year 4
|
-1.09 Standard Deviation Score (SDS)
Interval -1.5 to -0.9
|
|
Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects
Female: Year 5
|
-1.63 Standard Deviation Score (SDS)
Interval -1.9 to -1.4
|
|
Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects
Female: Year 6
|
-2.51 Standard Deviation Score (SDS)
Interval -3.4 to -1.6
|
|
Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects
Female: Year 7
|
-2.74 Standard Deviation Score (SDS)
Interval -3.7 to -1.8
|
|
Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects
Female: Year 8
|
-2.8 Standard Deviation Score (SDS)
Interval -2.8 to -2.8
|
|
Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects
Female: Year 8.5
|
-3.1 Standard Deviation Score (SDS)
Interval -3.1 to -3.1
|
SECONDARY outcome
Timeframe: Baseline (Month 0), Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 8.5 (only for female), 9 (only for male) and 9.5 (only for male)Population: Intention to Treat (ITT) population included all subjects who received at least one dose of treatment with at least an efficacy assessment available during the treatment period. Here "Number Analyzed" signifies those subjects who were evaluable for the specified category.
Body proportion was measured in terms of height. Data was reported by gender. Male subjects were analyzed till 9.5 years and female subjects were analyzed till 8.5 years.
Outcome measures
| Measure |
r-hGH (Saizen®)
n=19 Participants
Subjects received a single subcutaneous injection of recombinant human growth hormone (r-hGH) equivalent to dose of 0.057 milligram per kilogram per day (mg/kg/day). The dose was subsequently reduced to 0.035 mg/kg/day for subjects, who continued treatment after 31 January 2011. Subjects were treated for at least 3 years or until near final height was reached.
|
|---|---|
|
Change From Baseline in Height of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 4
|
26.96 Centimeter (cm)
Standard Deviation 2.394
|
|
Change From Baseline in Height of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 5
|
30.08 Centimeter (cm)
Standard Deviation 3.791
|
|
Change From Baseline in Height of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 6
|
32.50 Centimeter (cm)
Standard Deviation 0.566
|
|
Change From Baseline in Height of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 7
|
36.92 Centimeter (cm)
Standard Deviation 0.024
|
|
Change From Baseline in Height of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 8
|
39.47 Centimeter (cm)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Change From Baseline in Height of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 8.5
|
40.87 Centimeter (cm)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Change From Baseline in Height of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 1
|
8.10 Centimeter (cm)
Standard Deviation 1.867
|
|
Change From Baseline in Height of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 2
|
14.59 Centimeter (cm)
Standard Deviation 2.527
|
|
Change From Baseline in Height of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 3
|
20.00 Centimeter (cm)
Standard Deviation 3.398
|
|
Change From Baseline in Height of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 4
|
23.74 Centimeter (cm)
Standard Deviation 4.136
|
|
Change From Baseline in Height of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 5
|
29.06 Centimeter (cm)
Standard Deviation 4.809
|
|
Change From Baseline in Height of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 6
|
31.35 Centimeter (cm)
Standard Deviation 8.556
|
|
Change From Baseline in Height of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 7
|
28.40 Centimeter (cm)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Change From Baseline in Height of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 8
|
34.73 Centimeter (cm)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Change From Baseline in Height of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 9
|
37.98 Centimeter (cm)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Change From Baseline in Height of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 9.5
|
38.37 Centimeter (cm)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Change From Baseline in Height of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 1
|
7.72 Centimeter (cm)
Standard Deviation 1.642
|
|
Change From Baseline in Height of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 2
|
13.76 Centimeter (cm)
Standard Deviation 3.505
|
|
Change From Baseline in Height of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 3
|
17.95 Centimeter (cm)
Standard Deviation 5.189
|
SECONDARY outcome
Timeframe: Baseline (Month 0), Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 8.5 (only for female), 9 (only for male) and 9.5 (only for male)Population: ITT population. Here "Number analyzed" signifies those subjects who were evaluable for this outcome measure for specified category. Number Analyzed was "0" at certain time points because there was no data collected at the specified time points.
Body proportion was measured in terms of upper segment for standing and sitting position. Data was reported by gender. Male subjects were analyzed till 9.5 years and female subjects were analyzed till 8.5 years.
Outcome measures
| Measure |
r-hGH (Saizen®)
n=19 Participants
Subjects received a single subcutaneous injection of recombinant human growth hormone (r-hGH) equivalent to dose of 0.057 milligram per kilogram per day (mg/kg/day). The dose was subsequently reduced to 0.035 mg/kg/day for subjects, who continued treatment after 31 January 2011. Subjects were treated for at least 3 years or until near final height was reached.
|
|---|---|
|
Change From Baseline in Upper Segment (Superior) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 1
|
3.80 Centimeter (cm)
Standard Deviation 1.656
|
|
Change From Baseline in Upper Segment (Superior) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 2
|
7.96 Centimeter (cm)
Standard Deviation 4.172
|
|
Change From Baseline in Upper Segment (Superior) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 3
|
8.93 Centimeter (cm)
Standard Deviation 9.504
|
|
Change From Baseline in Upper Segment (Superior) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 4
|
16.50 Centimeter (cm)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Change From Baseline in Upper Segment (Superior) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 5
|
16.98 Centimeter (cm)
Standard Deviation 2.161
|
|
Change From Baseline in Upper Segment (Superior) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 6
|
17.05 Centimeter (cm)
Standard Deviation 6.576
|
|
Change From Baseline in Upper Segment (Superior) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 9
|
18.70 Centimeter (cm)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Change From Baseline in Upper Segment (Superior) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 9.5
|
27.80 Centimeter (cm)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Change From Baseline in Upper Segment (Superior) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 1
|
4.19 Centimeter (cm)
Standard Deviation 0.909
|
|
Change From Baseline in Upper Segment (Superior) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 2
|
9.21 Centimeter (cm)
Standard Deviation 2.450
|
|
Change From Baseline in Upper Segment (Superior) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 3
|
13.41 Centimeter (cm)
Standard Deviation 1.978
|
|
Change From Baseline in Upper Segment (Superior) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 4
|
15.70 Centimeter (cm)
Standard Deviation 2.546
|
|
Change From Baseline in Upper Segment (Superior) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 5
|
15.20 Centimeter (cm)
Standard Deviation 0.849
|
|
Change From Baseline in Upper Segment (Superior) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 6
|
18.35 Centimeter (cm)
Standard Deviation 0.354
|
|
Change From Baseline in Upper Segment (Superior) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 7
|
21.90 Centimeter (cm)
Standard Deviation 0.707
|
|
Change From Baseline in Upper Segment (Superior) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 8
|
24.40 Centimeter (cm)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
SECONDARY outcome
Timeframe: Baseline (Month 0), Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 8.5 (only for female), 9 (only for male) and 9.5 (only for male)Population: ITT population. Here "Number analyzed" signifies those subjects who were evaluable for this outcome measure for specified category.
The body weight was measured on a certified and calibrated hospital scale. Data was reported by gender. Male subjects were analyzed till 9.5 years and female subjects were analyzed till 8.5 years.
Outcome measures
| Measure |
r-hGH (Saizen®)
n=19 Participants
Subjects received a single subcutaneous injection of recombinant human growth hormone (r-hGH) equivalent to dose of 0.057 milligram per kilogram per day (mg/kg/day). The dose was subsequently reduced to 0.035 mg/kg/day for subjects, who continued treatment after 31 January 2011. Subjects were treated for at least 3 years or until near final height was reached.
|
|---|---|
|
Change From Baseline in Weight of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 1
|
4.58 Kilogram (kg)
Standard Deviation 2.715
|
|
Change From Baseline in Weight of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 2
|
9.95 Kilogram (kg)
Standard Deviation 4.577
|
|
Change From Baseline in Weight of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 3
|
15.18 Kilogram (kg)
Standard Deviation 6.259
|
|
Change From Baseline in Weight of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 4
|
14.25 Kilogram (kg)
Standard Deviation 3.948
|
|
Change From Baseline in Weight of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 5
|
20.00 Kilogram (kg)
Standard Deviation 7.024
|
|
Change From Baseline in Weight of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 6
|
25.00 Kilogram (kg)
Standard Deviation 11.314
|
|
Change From Baseline in Weight of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 7
|
19.00 Kilogram (kg)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Change From Baseline in Weight of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 8
|
23.00 Kilogram (kg)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Change From Baseline in Weight of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 9
|
28.90 Kilogram (kg)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Change From Baseline in Weight of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 9.5
|
31.00 Kilogram (kg)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Change From Baseline in Weight of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 1
|
4.72 Kilogram (kg)
Standard Deviation 1.537
|
|
Change From Baseline in Weight of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 2
|
9.85 Kilogram (kg)
Standard Deviation 3.946
|
|
Change From Baseline in Weight of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 3
|
14.37 Kilogram (kg)
Standard Deviation 4.905
|
|
Change From Baseline in Weight of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 4
|
14.70 Kilogram (kg)
Standard Deviation 5.510
|
|
Change From Baseline in Weight of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 5
|
14.93 Kilogram (kg)
Standard Deviation 3.101
|
|
Change From Baseline in Weight of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 6
|
21.25 Kilogram (kg)
Standard Deviation 1.061
|
|
Change From Baseline in Weight of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 7
|
26.25 Kilogram (kg)
Standard Deviation 1.061
|
|
Change From Baseline in Weight of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 8
|
35.20 Kilogram (kg)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Change From Baseline in Weight of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 8.5
|
38.00 Kilogram (kg)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
SECONDARY outcome
Timeframe: Baseline (Month 0), Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 8.5 (only for female), 9 (only for male) and 9.5 (only for male)Population: Intention to Treat (ITT) population included all subjects who received at least one dose of treatment with at least an efficacy assessment available during the treatment period. Here "Number analyzed" signifies those subjects who were evaluable for this outcome measure for specified category.
Body proportion measured as BMI. It was calculated according to the formula BMI = Weight (kilogram)/Height square (meter square). Data was reported by gender. Male subjects were analyzed till 9.5 years and female subjects were analyzed till 8.5 years.
Outcome measures
| Measure |
r-hGH (Saizen®)
n=19 Participants
Subjects received a single subcutaneous injection of recombinant human growth hormone (r-hGH) equivalent to dose of 0.057 milligram per kilogram per day (mg/kg/day). The dose was subsequently reduced to 0.035 mg/kg/day for subjects, who continued treatment after 31 January 2011. Subjects were treated for at least 3 years or until near final height was reached.
|
|---|---|
|
Change From Baseline in Body Mass Index (BMI) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 3
|
2.71 Kilogram per meter square (kg/m^2)
Standard Deviation 1.302
|
|
Change From Baseline in Body Mass Index (BMI) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 1
|
0.43 Kilogram per meter square (kg/m^2)
Standard Deviation 0.579
|
|
Change From Baseline in Body Mass Index (BMI) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 2
|
1.56 Kilogram per meter square (kg/m^2)
Standard Deviation 0.968
|
|
Change From Baseline in Body Mass Index (BMI) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 4
|
2.21 Kilogram per meter square (kg/m^2)
Standard Deviation 1.147
|
|
Change From Baseline in Body Mass Index (BMI) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 5
|
3.60 Kilogram per meter square (kg/m^2)
Standard Deviation 2.240
|
|
Change From Baseline in Body Mass Index (BMI) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 6
|
5.02 Kilogram per meter square (kg/m^2)
Standard Deviation 1.039
|
|
Change From Baseline in Body Mass Index (BMI) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 7
|
4.43 Kilogram per meter square (kg/m^2)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Change From Baseline in Body Mass Index (BMI) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 8
|
4.57 Kilogram per meter square (kg/m^2)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Change From Baseline in Body Mass Index (BMI) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 9
|
6.63 Kilogram per meter square (kg/m^2)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Change From Baseline in Body Mass Index (BMI) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Male: Year 9.5
|
7.60 Kilogram per meter square (kg/m^2)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Change From Baseline in Body Mass Index (BMI) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 1
|
0.85 Kilogram per meter square (kg/m^2)
Standard Deviation 1.112
|
|
Change From Baseline in Body Mass Index (BMI) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 2
|
2.12 Kilogram per meter square (kg/m^2)
Standard Deviation 1.978
|
|
Change From Baseline in Body Mass Index (BMI) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 3
|
3.49 Kilogram per meter square (kg/m^2)
Standard Deviation 2.341
|
|
Change From Baseline in Body Mass Index (BMI) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 4
|
2.12 Kilogram per meter square (kg/m^2)
Standard Deviation 1.778
|
|
Change From Baseline in Body Mass Index (BMI) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 5
|
2.36 Kilogram per meter square (kg/m^2)
Standard Deviation 2.207
|
|
Change From Baseline in Body Mass Index (BMI) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 6
|
4.90 Kilogram per meter square (kg/m^2)
Standard Deviation 0.634
|
|
Change From Baseline in Body Mass Index (BMI) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 7
|
6.19 Kilogram per meter square (kg/m^2)
Standard Deviation 0.408
|
|
Change From Baseline in Body Mass Index (BMI) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 8
|
9.83 Kilogram per meter square (kg/m^2)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Change From Baseline in Body Mass Index (BMI) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
Female: Year 8.5
|
10.71 Kilogram per meter square (kg/m^2)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
SECONDARY outcome
Timeframe: Baseline (Month 0), Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 9 (only for male)Population: ITT population. Here "Number analyzed" signifies those subjects who were evaluable for this outcome measure for specified category. Number Analyzed was "0" at certain time points because there was no data collected at the specified time points.
Body composition measured as BMD. It was examined at the lumbar spine (L1-L4) and determined annually by dual-energy X-ray absorptiometry. Data was reported by gender. Male subjects were analyzed till 9 years and female subjects were analyzed till 8 years.
Outcome measures
| Measure |
r-hGH (Saizen®)
n=19 Participants
Subjects received a single subcutaneous injection of recombinant human growth hormone (r-hGH) equivalent to dose of 0.057 milligram per kilogram per day (mg/kg/day). The dose was subsequently reduced to 0.035 mg/kg/day for subjects, who continued treatment after 31 January 2011. Subjects were treated for at least 3 years or until near final height was reached.
|
|---|---|
|
Change From Baseline in Bone Mineral Density (BMD) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Male: Year 1
|
0.05 Gram per square centimeter (g/cm^2)
Standard Deviation 0.050
|
|
Change From Baseline in Bone Mineral Density (BMD) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Male: Year 2
|
0.11 Gram per square centimeter (g/cm^2)
Standard Deviation 0.063
|
|
Change From Baseline in Bone Mineral Density (BMD) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Male: Year 3
|
0.14 Gram per square centimeter (g/cm^2)
Standard Deviation 0.059
|
|
Change From Baseline in Bone Mineral Density (BMD) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Male: Year 4
|
0.11 Gram per square centimeter (g/cm^2)
Standard Deviation 0.054
|
|
Change From Baseline in Bone Mineral Density (BMD) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Male: Year 5
|
0.15 Gram per square centimeter (g/cm^2)
Standard Deviation 0.064
|
|
Change From Baseline in Bone Mineral Density (BMD) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Male: Year 6
|
0.18 Gram per square centimeter (g/cm^2)
Standard Deviation 0.182
|
|
Change From Baseline in Bone Mineral Density (BMD) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Male: Year 8
|
0.06 Gram per square centimeter (g/cm^2)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Change From Baseline in Bone Mineral Density (BMD) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Male: Year 9
|
0.12 Gram per square centimeter (g/cm^2)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Change From Baseline in Bone Mineral Density (BMD) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Female: Year 1
|
0.05 Gram per square centimeter (g/cm^2)
Standard Deviation 0.042
|
|
Change From Baseline in Bone Mineral Density (BMD) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Female: Year 2
|
0.09 Gram per square centimeter (g/cm^2)
Standard Deviation 0.053
|
|
Change From Baseline in Bone Mineral Density (BMD) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Female: Year 3
|
0.15 Gram per square centimeter (g/cm^2)
Standard Deviation 0.089
|
|
Change From Baseline in Bone Mineral Density (BMD) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Female: Year 4
|
0.22 Gram per square centimeter (g/cm^2)
Standard Deviation 0.104
|
|
Change From Baseline in Bone Mineral Density (BMD) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Female: Year 5
|
0.17 Gram per square centimeter (g/cm^2)
Standard Deviation 0.006
|
|
Change From Baseline in Bone Mineral Density (BMD) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Female: Year 6
|
0.19 Gram per square centimeter (g/cm^2)
Standard Deviation 0.004
|
|
Change From Baseline in Bone Mineral Density (BMD) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Female: Year 7
|
0.26 Gram per square centimeter (g/cm^2)
Standard Deviation 0.028
|
|
Change From Baseline in Bone Mineral Density (BMD) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Female: Year 8
|
0.40 Gram per square centimeter (g/cm^2)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
SECONDARY outcome
Timeframe: Baseline (Month 0), Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 9 (only for male)Population: ITT population. Here "Number analyzed" signifies those subjects who were evaluable for this outcome measure for specified category. Number Analyzed was "0" at certain time points because there was no data collected at the specified time points.
Body composition measured as percentage of body fat mass. It was measured by Dual X-ray absorptiometry. Data was reported by gender. Male subjects were analyzed till 9 years and female subjects were analyzed till 8 years.
Outcome measures
| Measure |
r-hGH (Saizen®)
n=19 Participants
Subjects received a single subcutaneous injection of recombinant human growth hormone (r-hGH) equivalent to dose of 0.057 milligram per kilogram per day (mg/kg/day). The dose was subsequently reduced to 0.035 mg/kg/day for subjects, who continued treatment after 31 January 2011. Subjects were treated for at least 3 years or until near final height was reached.
|
|---|---|
|
Change From Baseline in Percent Body Fat Mass of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Female: Year 5
|
-2.50 Percentage (%) of body fat mass
Standard Deviation 1.131
|
|
Change From Baseline in Percent Body Fat Mass of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Male: Year 5
|
-2.33 Percentage (%) of body fat mass
Standard Deviation 5.708
|
|
Change From Baseline in Percent Body Fat Mass of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Male: Year 1
|
-4.54 Percentage (%) of body fat mass
Standard Deviation 2.049
|
|
Change From Baseline in Percent Body Fat Mass of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Male: Year 2
|
-4.49 Percentage (%) of body fat mass
Standard Deviation 3.007
|
|
Change From Baseline in Percent Body Fat Mass of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Male: Year 3
|
-2.80 Percentage (%) of body fat mass
Standard Deviation 3.967
|
|
Change From Baseline in Percent Body Fat Mass of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Male: Year 4
|
-3.40 Percentage (%) of body fat mass
Standard Deviation 4.493
|
|
Change From Baseline in Percent Body Fat Mass of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Male: Year 6
|
-0.65 Percentage (%) of body fat mass
Standard Deviation 11.384
|
|
Change From Baseline in Percent Body Fat Mass of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Male: Year 8
|
5.70 Percentage (%) of body fat mass
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Change From Baseline in Percent Body Fat Mass of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Male: Year 9
|
1.90 Percentage (%) of body fat mass
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Change From Baseline in Percent Body Fat Mass of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Female: Year 1
|
-3.50 Percentage (%) of body fat mass
Standard Deviation 3.185
|
|
Change From Baseline in Percent Body Fat Mass of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Female: Year 2
|
-2.27 Percentage (%) of body fat mass
Standard Deviation 4.441
|
|
Change From Baseline in Percent Body Fat Mass of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Female: Year 3
|
-2.74 Percentage (%) of body fat mass
Standard Deviation 5.227
|
|
Change From Baseline in Percent Body Fat Mass of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Female: Year 4
|
-1.40 Percentage (%) of body fat mass
Standard Deviation 3.536
|
|
Change From Baseline in Percent Body Fat Mass of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Female: Year 6
|
0.05 Percentage (%) of body fat mass
Standard Deviation 6.576
|
|
Change From Baseline in Percent Body Fat Mass of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Female: Year 7
|
-0.50 Percentage (%) of body fat mass
Standard Deviation 5.798
|
SECONDARY outcome
Timeframe: Baseline (Month 0), Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 9 (only for male)Population: ITT population. Here "Number analyzed" signifies those subjects who were evaluable for this outcome measure for specified category. Number Analyzed was "0" at certain time points because there was no data collected at the specified time points.
Body composition measured as LBM. It was measured by Dual X-ray absorptiometry. Data was reported by gender. Male subjects were analyzed till 9 years and female subjects were analyzed till 8 years.
Outcome measures
| Measure |
r-hGH (Saizen®)
n=19 Participants
Subjects received a single subcutaneous injection of recombinant human growth hormone (r-hGH) equivalent to dose of 0.057 milligram per kilogram per day (mg/kg/day). The dose was subsequently reduced to 0.035 mg/kg/day for subjects, who continued treatment after 31 January 2011. Subjects were treated for at least 3 years or until near final height was reached.
|
|---|---|
|
Change From Baseline in Lean Body Mass (LBM) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Male: Year 1
|
4966.46 Gram (g)
Standard Deviation 2670.694
|
|
Change From Baseline in Lean Body Mass (LBM) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Male: Year 2
|
9134.35 Gram (g)
Standard Deviation 4747.742
|
|
Change From Baseline in Lean Body Mass (LBM) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Male: Year 3
|
11182.24 Gram (g)
Standard Deviation 4126.876
|
|
Change From Baseline in Lean Body Mass (LBM) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Male: Year 4
|
11599.80 Gram (g)
Standard Deviation 4560.877
|
|
Change From Baseline in Lean Body Mass (LBM) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Male: Year 5
|
15729.38 Gram (g)
Standard Deviation 6102.608
|
|
Change From Baseline in Lean Body Mass (LBM) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Male: Year 6
|
20667.65 Gram (g)
Standard Deviation 14985.50
|
|
Change From Baseline in Lean Body Mass (LBM) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Male: Year 8
|
15773.80 Gram (g)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Change From Baseline in Lean Body Mass (LBM) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Male: Year 9
|
21713.10 Gram (g)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Change From Baseline in Lean Body Mass (LBM) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Female: Year 1
|
4384.54 Gram (g)
Standard Deviation 1400.497
|
|
Change From Baseline in Lean Body Mass (LBM) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Female: Year 2
|
6720.74 Gram (g)
Standard Deviation 2597.295
|
|
Change From Baseline in Lean Body Mass (LBM) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Female: Year 3
|
9229.21 Gram (g)
Standard Deviation 2740.883
|
|
Change From Baseline in Lean Body Mass (LBM) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Female: Year 4
|
11791.85 Gram (g)
Standard Deviation 1173.161
|
|
Change From Baseline in Lean Body Mass (LBM) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Female: Year 5
|
12989.50 Gram (g)
Standard Deviation 83.439
|
|
Change From Baseline in Lean Body Mass (LBM) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Female: Year 6
|
14860.75 Gram (g)
Standard Deviation 2140.483
|
|
Change From Baseline in Lean Body Mass (LBM) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
Female: Year 7
|
18111.60 Gram (g)
Standard Deviation 1239.275
|
SECONDARY outcome
Timeframe: Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 8.5 (only for female), 9 (only for male) and 9.5 (only for male)Population: Intention to Treat (ITT) population included all subjects who received at least one dose of treatment with at least an efficacy assessment available during the treatment period. Here "Number analyzed" signifies those subjects who were evaluable for this outcome measure for specified category.
Growth velocity was calculated in terms of height velocity. It corresponded to a difference in height between current and baseline values divided by the time interval between both evaluations. Data was reported by gender. Male subjects were analyzed till 9.5 years and female subjects were analyzed till 8.5 years.
Outcome measures
| Measure |
r-hGH (Saizen®)
n=19 Participants
Subjects received a single subcutaneous injection of recombinant human growth hormone (r-hGH) equivalent to dose of 0.057 milligram per kilogram per day (mg/kg/day). The dose was subsequently reduced to 0.035 mg/kg/day for subjects, who continued treatment after 31 January 2011. Subjects were treated for at least 3 years or until near final height was reached.
|
|---|---|
|
Growth (Height) Velocity of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 5
|
5.77 Centimeter per year (cm/year)
Standard Deviation 0.968
|
|
Growth (Height) Velocity of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 1
|
8.21 Centimeter per year (cm/year)
Standard Deviation 2.001
|
|
Growth (Height) Velocity of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 2
|
7.26 Centimeter per year (cm/year)
Standard Deviation 1.292
|
|
Growth (Height) Velocity of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 3
|
6.55 Centimeter per year (cm/year)
Standard Deviation 1.104
|
|
Growth (Height) Velocity of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 4
|
5.88 Centimeter per year (cm/year)
Standard Deviation 1.060
|
|
Growth (Height) Velocity of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 6
|
4.98 Centimeter per year (cm/year)
Standard Deviation 1.313
|
|
Growth (Height) Velocity of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 7
|
3.92 Centimeter per year (cm/year)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Growth (Height) Velocity of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 8
|
4.25 Centimeter per year (cm/year)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Growth (Height) Velocity of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 9
|
4.11 Centimeter per year (cm/year)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Growth (Height) Velocity of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 9.5
|
3.94 Centimeter per year (cm/year)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Growth (Height) Velocity of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 1
|
7.53 Centimeter per year (cm/year)
Standard Deviation 1.460
|
|
Growth (Height) Velocity of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 2
|
6.64 Centimeter per year (cm/year)
Standard Deviation 1.713
|
|
Growth (Height) Velocity of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 3
|
5.86 Centimeter per year (cm/year)
Standard Deviation 1.677
|
|
Growth (Height) Velocity of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 4
|
6.57 Centimeter per year (cm/year)
Standard Deviation 0.572
|
|
Growth (Height) Velocity of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 5
|
5.89 Centimeter per year (cm/year)
Standard Deviation 0.787
|
|
Growth (Height) Velocity of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 6
|
5.13 Centimeter per year (cm/year)
Standard Deviation 0.006
|
|
Growth (Height) Velocity of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 7
|
5.06 Centimeter per year (cm/year)
Standard Deviation 0.120
|
|
Growth (Height) Velocity of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 8
|
4.69 Centimeter per year (cm/year)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Growth (Height) Velocity of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 8.5
|
4.54 Centimeter per year (cm/year)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
SECONDARY outcome
Timeframe: Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 8.5 (only for female), 9 (only for male) and 9.5 (only for male)Population: ITT population. Here "Number analyzed" signifies those subjects who were evaluable for this outcome measure for specified category. Number Analyzed was "0" at certain time points because there was no data collected at the specified time points.
Body proportion was measured in terms of head circumference with a tape measure. Data was reported by gender. Male subjects were analyzed till 9.5 years and female subjects were analyzed till 8.5 years.
Outcome measures
| Measure |
r-hGH (Saizen®)
n=19 Participants
Subjects received a single subcutaneous injection of recombinant human growth hormone (r-hGH) equivalent to dose of 0.057 milligram per kilogram per day (mg/kg/day). The dose was subsequently reduced to 0.035 mg/kg/day for subjects, who continued treatment after 31 January 2011. Subjects were treated for at least 3 years or until near final height was reached.
|
|---|---|
|
Head Circumference Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 6
|
57.25 Centimeter (cm)
Standard Deviation 1.768
|
|
Head Circumference Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 1
|
56.11 Centimeter (cm)
Standard Deviation 1.268
|
|
Head Circumference Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 2
|
56.53 Centimeter (cm)
Standard Deviation 1.359
|
|
Head Circumference Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 3
|
57.38 Centimeter (cm)
Standard Deviation 1.734
|
|
Head Circumference Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 4
|
57.33 Centimeter (cm)
Standard Deviation 2.021
|
|
Head Circumference Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 5
|
56.88 Centimeter (cm)
Standard Deviation 1.887
|
|
Head Circumference Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 6
|
55.50 Centimeter (cm)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Head Circumference Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 7
|
57.00 Centimeter (cm)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Head Circumference Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 9
|
57.50 Centimeter (cm)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Head Circumference Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 9.5
|
58.00 Centimeter (cm)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Head Circumference Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 1
|
55.20 Centimeter (cm)
Standard Deviation 2.379
|
|
Head Circumference Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 2
|
56.06 Centimeter (cm)
Standard Deviation 1.841
|
|
Head Circumference Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 3
|
56.23 Centimeter (cm)
Standard Deviation 2.453
|
|
Head Circumference Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 4
|
58.00 Centimeter (cm)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Head Circumference Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 5
|
54.33 Centimeter (cm)
Standard Deviation 3.512
|
|
Head Circumference Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 7
|
56.75 Centimeter (cm)
Standard Deviation 1.061
|
|
Head Circumference Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 8
|
59.00 Centimeter (cm)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
SECONDARY outcome
Timeframe: Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 8.5 (only for female), 9 (only for male), 9.5 (only for male)Population: ITT population. Here "Number analyzed" signifies those subjects who were evaluable for this outcome measure for specified category. Number Analyzed was "0" at certain time points because there was no data collected at the specified time points.
Osteocalcin as bone marker was analyzed with the Immunology system Elecsys. Data was reported by gender. Male subjects were analyzed till 9.5 years and female subjects were analyzed till 8.5 years.
Outcome measures
| Measure |
r-hGH (Saizen®)
n=19 Participants
Subjects received a single subcutaneous injection of recombinant human growth hormone (r-hGH) equivalent to dose of 0.057 milligram per kilogram per day (mg/kg/day). The dose was subsequently reduced to 0.035 mg/kg/day for subjects, who continued treatment after 31 January 2011. Subjects were treated for at least 3 years or until near final height was reached.
|
|---|---|
|
Osteocalcin Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 3
|
94.73 Nanogram per milliliter (ng/mL)
Standard Deviation 33.344
|
|
Osteocalcin Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 1
|
123.06 Nanogram per milliliter (ng/mL)
Standard Deviation 55.820
|
|
Osteocalcin Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 2
|
145.00 Nanogram per milliliter (ng/mL)
Standard Deviation 35.705
|
|
Osteocalcin Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 3
|
130.13 Nanogram per milliliter (ng/mL)
Standard Deviation 36.408
|
|
Osteocalcin Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 4
|
101.00 Nanogram per milliliter (ng/mL)
Standard Deviation 34.708
|
|
Osteocalcin Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 5
|
139.25 Nanogram per milliliter (ng/mL)
Standard Deviation 76.908
|
|
Osteocalcin Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 6
|
75.50 Nanogram per milliliter (ng/mL)
Standard Deviation 47.376
|
|
Osteocalcin Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 7
|
113.00 Nanogram per milliliter (ng/mL)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Osteocalcin Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 8
|
117.00 Nanogram per milliliter (ng/mL)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Osteocalcin Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 9
|
159.80 Nanogram per milliliter (ng/mL)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Osteocalcin Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 9.5
|
131.60 Nanogram per milliliter (ng/mL)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
Osteocalcin Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 1
|
148.00 Nanogram per milliliter (ng/mL)
Standard Deviation 46.183
|
|
Osteocalcin Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 2
|
122.82 Nanogram per milliliter (ng/mL)
Standard Deviation 46.531
|
|
Osteocalcin Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 4
|
102.00 Nanogram per milliliter (ng/mL)
Standard Deviation 26.268
|
|
Osteocalcin Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 5
|
84.33 Nanogram per milliliter (ng/mL)
Standard Deviation 22.591
|
|
Osteocalcin Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 6
|
106.00 Nanogram per milliliter (ng/mL)
Standard Deviation 22.627
|
|
Osteocalcin Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 7
|
77.50 Nanogram per milliliter (ng/mL)
Standard Deviation 19.092
|
|
Osteocalcin Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 8
|
73.70 Nanogram per milliliter (ng/mL)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
SECONDARY outcome
Timeframe: Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 8.5 (only for female), 9 (only for male), 9.5 (only for male)Population: ITT population. Here "Number analyzed" signifies those subjects who were evaluable for this outcome measure for specified category. Number Analyzed was "0" at certain time points because there was no data collected at the specified time points.
CTX (Blood) as bone marker was analyzed with the Immunology system Elecsys. Data was reported by gender. Male subjects were analyzed till 9.5 years and female subjects were analyzed till 8.5 years.
Outcome measures
| Measure |
r-hGH (Saizen®)
n=19 Participants
Subjects received a single subcutaneous injection of recombinant human growth hormone (r-hGH) equivalent to dose of 0.057 milligram per kilogram per day (mg/kg/day). The dose was subsequently reduced to 0.035 mg/kg/day for subjects, who continued treatment after 31 January 2011. Subjects were treated for at least 3 years or until near final height was reached.
|
|---|---|
|
C-terminal Telopeptide (CTX) Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 5
|
12503.33 picomole per liter (pmol/L)
Standard Deviation 2867.849
|
|
C-terminal Telopeptide (CTX) Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 1
|
15784.17 picomole per liter (pmol/L)
Standard Deviation 1426.214
|
|
C-terminal Telopeptide (CTX) Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 2
|
16304.13 picomole per liter (pmol/L)
Standard Deviation 2537.725
|
|
C-terminal Telopeptide (CTX) Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 3
|
15703.50 picomole per liter (pmol/L)
Standard Deviation 3249.336
|
|
C-terminal Telopeptide (CTX) Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 4
|
15868.25 picomole per liter (pmol/L)
Standard Deviation 4485.621
|
|
C-terminal Telopeptide (CTX) Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 5
|
17088.75 picomole per liter (pmol/L)
Standard Deviation 5208.676
|
|
C-terminal Telopeptide (CTX) Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 6
|
12361.00 picomole per liter (pmol/L)
Standard Deviation 4000.810
|
|
C-terminal Telopeptide (CTX) Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 7
|
13795.00 picomole per liter (pmol/L)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
C-terminal Telopeptide (CTX) Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 8
|
15345.00 picomole per liter (pmol/L)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
C-terminal Telopeptide (CTX) Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 9
|
15655.00 picomole per liter (pmol/L)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
C-terminal Telopeptide (CTX) Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Male: Year 9.5
|
17205.00 picomole per liter (pmol/L)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
|
C-terminal Telopeptide (CTX) Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 1
|
16008.00 picomole per liter (pmol/L)
Standard Deviation 2725.874
|
|
C-terminal Telopeptide (CTX) Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 2
|
15183.09 picomole per liter (pmol/L)
Standard Deviation 4371.058
|
|
C-terminal Telopeptide (CTX) Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 3
|
12595.18 picomole per liter (pmol/L)
Standard Deviation 3172.529
|
|
C-terminal Telopeptide (CTX) Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 4
|
13911.00 picomole per liter (pmol/L)
Standard Deviation 1076.509
|
|
C-terminal Telopeptide (CTX) Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 6
|
13678.50 picomole per liter (pmol/L)
Standard Deviation 603.162
|
|
C-terminal Telopeptide (CTX) Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 7
|
13446.50 picomole per liter (pmol/L)
Standard Deviation 931.260
|
|
C-terminal Telopeptide (CTX) Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
Female: Year 8
|
11858.00 picomole per liter (pmol/L)
Standard Deviation NA
Standard deviation could not be estimated as there was only 1 subject analyzed at the specified time point.
|
SECONDARY outcome
Timeframe: Baseline up to 3 yearsPopulation: Intention to Treat (ITT) population included all subjects who received at least one dose of treatment with at least an efficacy assessment available during the treatment period.
Genetic analysis was performed to record FGFR3 gene mutation. Genomic DNA was extracted from lymphocytes of collected blood samples using standard procedures. A set of intronic primers was designed based on the genomic sequence of the human FGFR3 gene and used to amplify exons 2-19. Number of subjects with FGFR3 mutation were reported.
Outcome measures
| Measure |
r-hGH (Saizen®)
n=19 Participants
Subjects received a single subcutaneous injection of recombinant human growth hormone (r-hGH) equivalent to dose of 0.057 milligram per kilogram per day (mg/kg/day). The dose was subsequently reduced to 0.035 mg/kg/day for subjects, who continued treatment after 31 January 2011. Subjects were treated for at least 3 years or until near final height was reached.
|
|---|---|
|
Number of Subjects With Fibroblast Growth Factor Receptor (FGFR3) Mutation
|
14 Subjects
|
SECONDARY outcome
Timeframe: Baseline up to 9.5 yearsPopulation: Intention to Treat (ITT) population included all subjects who received at least one dose of treatment with at least an efficacy assessment available during the treatment period.
AEs: any new untoward medical occurrences/worsening of pre-existing medical condition, whether or not related to study drug , SAE: any AE that resulted in death; was life threatening; resulted in persistent/significant disability/incapacity; resulted in/prolonged an existing in-patient hospitalization; was a congenital anomaly/birth defect; or was an overdose.
Outcome measures
| Measure |
r-hGH (Saizen®)
n=19 Participants
Subjects received a single subcutaneous injection of recombinant human growth hormone (r-hGH) equivalent to dose of 0.057 milligram per kilogram per day (mg/kg/day). The dose was subsequently reduced to 0.035 mg/kg/day for subjects, who continued treatment after 31 January 2011. Subjects were treated for at least 3 years or until near final height was reached.
|
|---|---|
|
Number of Subjects With Adverse Event (AE) and Serious Adverse Event (SAE)
AEs
|
18 Subjects
|
|
Number of Subjects With Adverse Event (AE) and Serious Adverse Event (SAE)
SAEs
|
1 Subjects
|
Adverse Events
r-hGH (Saizen®)
Serious events: 1 serious events
Other events: 18 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
r-hGH (Saizen®)
n=19 participants at risk
Subjects received a single subcutaneous injection of recombinant human growth hormone (r-hGH) equivalent to dose of 0.057 milligram per kilogram per day (mg/kg/day). The dose was subsequently reduced to 0.035 mg/kg/day for subjects, who continued treatment after 31 January 2011. Subjects were treated for at least 3 years or until near final height was reached.
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
5.3%
1/19
|
|
Infections and infestations
Appendicitis
|
5.3%
1/19
|
Other adverse events
| Measure |
r-hGH (Saizen®)
n=19 participants at risk
Subjects received a single subcutaneous injection of recombinant human growth hormone (r-hGH) equivalent to dose of 0.057 milligram per kilogram per day (mg/kg/day). The dose was subsequently reduced to 0.035 mg/kg/day for subjects, who continued treatment after 31 January 2011. Subjects were treated for at least 3 years or until near final height was reached.
|
|---|---|
|
Blood and lymphatic system disorders
Neutropenia
|
5.3%
1/19
|
|
Blood and lymphatic system disorders
Thrombopenia
|
5.3%
1/19
|
|
Blood and lymphatic system disorders
Leucopenia
|
5.3%
1/19
|
|
Blood and lymphatic system disorders
Lymphopenia
|
5.3%
1/19
|
|
Cardiac disorders
Angina pectoris
|
5.3%
1/19
|
|
Endocrine disorders
Precocious puberty
|
5.3%
1/19
|
|
Endocrine disorders
Hypothyroidism
|
5.3%
1/19
|
|
Gastrointestinal disorders
Dental cyst
|
5.3%
1/19
|
|
Gastrointestinal disorders
Constipation
|
5.3%
1/19
|
|
Gastrointestinal disorders
Abdominal Pain
|
26.3%
5/19
|
|
Gastrointestinal disorders
Diarrhea
|
5.3%
1/19
|
|
General disorders
Pyrexia
|
15.8%
3/19
|
|
General disorders
Fatigue
|
5.3%
1/19
|
|
General disorders
Oedema peripheral
|
5.3%
1/19
|
|
Infections and infestations
Tooth abscess
|
5.3%
1/19
|
|
Infections and infestations
Pharyngitis
|
5.3%
1/19
|
|
Infections and infestations
Vulvitis
|
5.3%
1/19
|
|
Infections and infestations
Bronchitis
|
15.8%
3/19
|
|
Infections and infestations
Gastroenteritis
|
15.8%
3/19
|
|
Infections and infestations
Rhinitis
|
5.3%
1/19
|
|
Infections and infestations
Tonsillitis
|
10.5%
2/19
|
|
Infections and infestations
Meningitis viral
|
5.3%
1/19
|
|
Infections and infestations
Influenza
|
10.5%
2/19
|
|
Infections and infestations
Appendicitis
|
5.3%
1/19
|
|
Infections and infestations
Conjunctivitis
|
5.3%
1/19
|
|
Infections and infestations
Infection
|
5.3%
1/19
|
|
Infections and infestations
Herpes zoster
|
5.3%
1/19
|
|
Infections and infestations
Ear infection
|
5.3%
1/19
|
|
Infections and infestations
Nasopharyngitis
|
5.3%
1/19
|
|
Injury, poisoning and procedural complications
Meniscus injury
|
5.3%
1/19
|
|
Investigations
Insulin-like growth factor increased
|
15.8%
3/19
|
|
Metabolism and nutrition disorders
Vitamin D deficiency
|
15.8%
3/19
|
|
Metabolism and nutrition disorders
Insulin resistance
|
5.3%
1/19
|
|
Metabolism and nutrition disorders
Hypertriglyceridaemia
|
5.3%
1/19
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
5.3%
1/19
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
10.5%
2/19
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
36.8%
7/19
|
|
Musculoskeletal and connective tissue disorders
Foot deformity
|
5.3%
1/19
|
|
Musculoskeletal and connective tissue disorders
Knee deformity
|
21.1%
4/19
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
10.5%
2/19
|
|
Musculoskeletal and connective tissue disorders
Torticollis
|
5.3%
1/19
|
|
Musculoskeletal and connective tissue disorders
Scoliosis
|
26.3%
5/19
|
|
Metabolism and nutrition disorders
Limb deformity
|
5.3%
1/19
|
|
Musculoskeletal and connective tissue disorders
Deformity thorax
|
5.3%
1/19
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
5.3%
1/19
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal stiffness
|
5.3%
1/19
|
|
Musculoskeletal and connective tissue disorders
Lordosis
|
5.3%
1/19
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Skin papilloma
|
5.3%
1/19
|
|
Nervous system disorders
Headache
|
21.1%
4/19
|
|
Nervous system disorders
Sciatica
|
5.3%
1/19
|
|
Nervous system disorders
Syncope
|
5.3%
1/19
|
|
Nervous system disorders
Dizziness
|
5.3%
1/19
|
|
Nervous system disorders
Loss of consciousness
|
5.3%
1/19
|
|
Psychiatric disorders
Anger
|
5.3%
1/19
|
|
Reproductive system and breast disorders
Gynaecomastia
|
5.3%
1/19
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
15.8%
3/19
|
|
Respiratory, thoracic and mediastinal disorders
Bronchial obstruction
|
5.3%
1/19
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
5.3%
1/19
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea exertional
|
5.3%
1/19
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
10.5%
2/19
|
|
Skin and subcutaneous tissue disorders
Pigmentation disorder
|
10.5%
2/19
|
|
Skin and subcutaneous tissue disorders
Eczema
|
10.5%
2/19
|
|
Skin and subcutaneous tissue disorders
Acne
|
10.5%
2/19
|
|
Skin and subcutaneous tissue disorders
Skin lesion
|
5.3%
1/19
|
|
Skin and subcutaneous tissue disorders
Rash
|
5.3%
1/19
|
|
Social circumstances
Corrective lens user
|
5.3%
1/19
|
|
Surgical and medical procedures
Mole excision
|
5.3%
1/19
|
Additional Information
Merck KGaA Communication Center
Merck Healthcare, a business of Merck KGaA
Phone: +49 6151 72 5200
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER