Hypochondroplasia
Conditions
Keywords
Hypochondroplasia, Skeletal dysplasia, Growth, Growth Hormone, Recombinant-human Growth Hormone
Brief summary
This study is conducted to describe the efficacy and safety of recombinant human growth hormone (r-hGH) treatment Saizen® on children with hypochondroplasia.
Interventions
Subjects will receive a single subcutaneous injection of recombinant human growth hormone (r-hGH) equivalent to a dose of 0.057 milligram per kilogram per day (mg/kg/day). The dose will be subsequently adjusted during the trial and subjects will be treated for at least 3 years or until near final height is reached.
Sponsors
Merck Serono S.A.S, France
Merck KGaA, Darmstadt, Germany
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male or female children with hypochondroplasia defined by a disproportional short limb height and a X-ray evidence of shortening of the long bones and failure of increase in the interpedicular distance between lumbar vertebrae L1 and L5 * Result of genetic analysis for mutation of gene FGFR3 already known or ongoing analysis at the beginning of the study * Chronological age greater than or equal to 3 years * Height for chronological age less than or equal to - 2 SDS * Bone age less than or equal to 11 years for girls and 13 years for boys * A written informed consent at the beginning of the pre-treatment period must be obtained from the parent(s)/legal guardian(s). Children able to understand the trial should personally sign and date the written informed consent Additional inclusion criteria for each study prolongation: * Bone age at Month 36 or Month 60 is compatible with treatment prolongation according to investigator opinion * Subject is still under r-hGH treatment with Saizen® at Month 36 or Month 60 * Height gain greater than or equal to + 1 SDS after the 2 first years of treatment for treatment prolongation at Month 36 and growth velocity greater than or equal to 5 centimeter (cm) per year, with bone age less than 14 years for females or less than 16 years for males for treatment prolongation at Month 60 * According to investigator opinion, gene mutations of the subjects are not in connection with observed side effects during the 3 or 5 first years of treatment * An updated written informed consent must be obtained from the parent(s)/legal guardian(s) before the start of each study prolongation. Children able to understand the trial should personally sign and date the written informed consent
Exclusion criteria
* Turner's Syndrome in girls * Active malignant neoplastic disease * Severe congenital malformations * Proliferative or preproliferative diabetic retinopathy * Evidence of any progression or recurrence of an underlying intra-cranial space occupying lesion * Severe psychomotor retardation * Diabetes mellitus or history of significant glucose intolerance as defined by a fasting blood glucose greater than 6.4 millimole per liter (mmol/L) * Known renal insufficiency as defined by serum creatinine level 1.0 milligram per deciliter (mg/dL) (88 micromole per liter \[mcmol/L\]) * Known hepatic disease as defined by elevated liver enzymes or total bilirubin (\* 2 Normal) * Current congestive heart failure, untreated hypertension, serious chronic edema of any cause * Chronic infectious disease * History of intracranial hypertension with papilledema * Previous or ongoing treatment with sex steroid therapy such as estrogens or testosterone * Previous or ongoing treatment with any therapy that may directly influence growth, including Growth Hormone (GH), Growth Hormone Releasing Hormone (GHRH) and long duration corticosteroids therapy * Known hypersensitivity to somatropin or any of the excipients * Epiphyseal fusion * Participation to any clinical study within the 30 days preceding study entry * Pregnant females
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects at Year 3 | Baseline (Month 0), Year 3 | 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. |
| Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects at Year 4 | Year 4 | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Upper Segment (Superior) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years | 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) | 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. |
| Change From Baseline in Weight of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years | 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) | 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. |
| 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 | 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) | 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. |
| Change From Baseline in Bone Mineral Density (BMD) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years | Baseline (Month 0), Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 9 (only for male) | 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. |
| Change From Baseline in Percent Body Fat Mass of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years | Baseline (Month 0), Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 9 (only for male) | 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. |
| Change From Baseline in Lean Body Mass (LBM) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years | Baseline (Month 0), Year 1, 2, 3, 4, 5, 6, 7, 8 (both male and female); 9 (only for male) | 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. |
| Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | 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) | 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. |
| Head Circumference Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years | 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) | 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. |
| Osteocalcin Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years | 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) | 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. |
| 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 | 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) | 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. |
| Number of Subjects With Fibroblast Growth Factor Receptor (FGFR3) Mutation | Baseline up to 3 years | 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. |
| Number of Subjects With Adverse Event (AE) and Serious Adverse Event (SAE) | Baseline up to 9.5 years | 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. |
| Growth (Height) Velocity of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years | 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) | 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. |
| Change From Baseline in Height of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years | 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) | 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. |
Countries
France
Participant flow
Recruitment details
The study was conducted between March 21, 2006 and January 17, 2017.
Participants by arm
| Arm | Count |
|---|---|
| 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. | 19 |
| Total | 19 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Other | 1 |
| Overall Study | Withdrew Prematurely | 2 |
Baseline characteristics
| Characteristic | r-hGH (Saizen®) |
|---|---|
| Age, Continuous | 9.0 Years STANDARD_DEVIATION 3.12 |
| Sex/Gender, Customized Female | 11 Participants |
| Sex/Gender, Customized Male | 8 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 19 |
| other Total, other adverse events | 18 / 19 |
| serious Total, serious adverse events | 1 / 19 |
Outcome results
Primary
Change From Baseline in Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects at Year 3
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.
Time frame: Baseline (Month 0), Year 3
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| r-hGH (Saizen®) | 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
Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects at Year 4
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.
Time frame: Year 4
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| r-hGH (Saizen®) | 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
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
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.
Time frame: 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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 2.24 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
Secondary
Change From Baseline in Bone Mineral Density (BMD) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
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.
Time frame: 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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| r-hGH (Saizen®) | 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 0.05 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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) | — |
Secondary
Change From Baseline in Height of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
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.
Time frame: 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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
Secondary
Change From Baseline in Lean Body Mass (LBM) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
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.
Time frame: 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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 14985.5 |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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
Change From Baseline in Percent Body Fat Mass of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9 Years
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.
Time frame: 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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 | — |
| r-hGH (Saizen®) | 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 | — |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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
Change From Baseline in Upper Segment (Superior) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
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.
Time frame: 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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 2.45 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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) | — |
Secondary
Change From Baseline in Weight of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) up to 9.5 Years
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.
Time frame: 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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 5.51 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
Secondary
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
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.
Time frame: 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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 4000.81 |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 931.26 |
| r-hGH (Saizen®) | 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) | — |
Secondary
Growth (Height) Velocity of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
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.
Time frame: 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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 1.06 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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 1.46 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 0.12 |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
Secondary
Head Circumference Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
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.
Time frame: 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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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) | — |
Secondary
Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects
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.
Time frame: 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.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| r-hGH (Saizen®) | Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | Male: Year 1 | -1.82 Standard Deviation Score (SDS) |
| r-hGH (Saizen®) | Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | Male: Year 2 | -1.51 Standard Deviation Score (SDS) |
| r-hGH (Saizen®) | Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | Male: Year 3 | -1.30 Standard Deviation Score (SDS) |
| r-hGH (Saizen®) | Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | Male: Year 4 | -1.49 Standard Deviation Score (SDS) |
| r-hGH (Saizen®) | Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | Male: Year 5 | -1.51 Standard Deviation Score (SDS) |
| r-hGH (Saizen®) | Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | Male: Year 6 | -2.88 Standard Deviation Score (SDS) |
| r-hGH (Saizen®) | Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | Male: Year 7 | -3.9 Standard Deviation Score (SDS) |
| r-hGH (Saizen®) | Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | Male: Year 8 | -3.90 Standard Deviation Score (SDS) |
| r-hGH (Saizen®) | Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | Male: Year 9 | -5.0 Standard Deviation Score (SDS) |
| r-hGH (Saizen®) | 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) |
| r-hGH (Saizen®) | Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | Female: Year 1 | -2.69 Standard Deviation Score (SDS) |
| r-hGH (Saizen®) | Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | Female: Year 2 | -2.52 Standard Deviation Score (SDS) |
| r-hGH (Saizen®) | Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | Female: Year 3 | -2.61 Standard Deviation Score (SDS) |
| r-hGH (Saizen®) | Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | Female: Year 4 | -1.09 Standard Deviation Score (SDS) |
| r-hGH (Saizen®) | Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | Female: Year 5 | -1.63 Standard Deviation Score (SDS) |
| r-hGH (Saizen®) | Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | Female: Year 6 | -2.51 Standard Deviation Score (SDS) |
| r-hGH (Saizen®) | Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | Female: Year 7 | -2.74 Standard Deviation Score (SDS) |
| r-hGH (Saizen®) | Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects | Female: Year 8 | -2.8 Standard Deviation Score (SDS) |
| r-hGH (Saizen®) | 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) |
Secondary
Number of Subjects With Adverse Event (AE) and Serious Adverse Event (SAE)
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.
Time frame: Baseline up to 9.5 years
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.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| r-hGH (Saizen®) | Number of Subjects With Adverse Event (AE) and Serious Adverse Event (SAE) | AEs | 18 Subjects |
| r-hGH (Saizen®) | Number of Subjects With Adverse Event (AE) and Serious Adverse Event (SAE) | SAEs | 1 Subjects |
Secondary
Number of Subjects With Fibroblast Growth Factor Receptor (FGFR3) Mutation
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.
Time frame: Baseline up to 3 years
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.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| r-hGH (Saizen®) | Number of Subjects With Fibroblast Growth Factor Receptor (FGFR3) Mutation | 14 Subjects |
Secondary
Osteocalcin Values of Recombinant Human Growth Hormone (r-hGH) Treated Subjects With Hypochondroplasia (HCH) From Year 1 up to 9.5 Years
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.
Time frame: 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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| r-hGH (Saizen®) | 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 55.82 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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) | — |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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 |
| r-hGH (Saizen®) | 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) | — |