Growth Hormone Deficiency in Children, Growth Hormone Disorder
Conditions
Brief summary
This trial is conducted globally. The aim of the trial is to investigate efficacy and safety of once-weekly NNC0195-0092 treatment compared to daily growth hormone treatment (Norditropin® FlexPro®) in growth hormone treatment naïve pre-pubertal children with growth hormone deficiency. The main trial period will consist of 26 weeks of treatment, followed by a 26 week extension period.
Interventions
DRUGsomapacitan
Administered subcutaneously (s.c., under the skin) once-weekly.
Administered subcutaneously (s.c., under the skin) once daily.
Sponsors
Novo Nordisk A/S
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Masking description
Sponsor staff involved in the clinical trial is masked according to company standard procedures.
Eligibility
Sex/Gender
ALL
Age
2 Years to 10 Years
Healthy volunteers
No
Inclusion criteria
* Boys: Tanner stage 1 for pubic hair and testis volume below 4 ml , age at least 2 years and 26 weeks and below or equal to 10.0 years at screening * Girls: Tanner stage 1 for breast development (no palpable glandular breast tissue) and pubic hair, age at least 2 years and 26 weeks and below or equal to 9.0 years at screening * Confirmed diagnosis of GHD (growth hormone deficiency) within 12 months prior to screening as determined by two different GH (growth hormone) stimulation tests, defined as a peak GH level of below or equal to 7.0 ng/ml. For children with three or more pituitary hormone deficiencies only one GH stimulation test is needed * No prior exposure to GH therapy and/or IGF-I (insulin-like growth factor I) treatment * Height of at least 2.0 standard deviations below the mean height for chronological age (CA) and gender according to the standards of Centers for Disease Control and Prevention 2-20 years: Girls/Boys stature-for-age and weight-for-age percentiles CDC at screening * Annualized height velocity (HV) below the 25th percentile for CA (chronological age) and gender or below -0.7 SD (standard deviation) score for CA and sex, according to the standards of Prader calculated over a time span of minimum 6 months and maximum 18 months
Exclusion criteria
* Any clinically significant abnormality likely to affect growth or the ability to evaluate * growth with standing measurements: Chromosomal aneuploidy and significant gene mutations causing medical "syndromes" with short stature, including but not limited to Turner syndrome, Laron syndrome, Noonan syndrome, or absence of GH receptors. Congenital abnormalities (causing skeletal abnormalities), including but not limited to Russell-Silver Syndrome, skeletal dysplasias. Significant spinal abnormalities including but not limited to scoliosis, kyphosis and spina bifida variants * Children born small for gestational age (SGA - birth weight and/or birth length below-2 SD for gestational age) * Concomitant administration of other treatments that may have an effect on growth, including but not limited to methylphenidate for treatment of attention deficit hyperactivity disorder (ADHD) * Prior history or presence of malignancy and/or intracranial tumour
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Height Velocity (HV) (cm/Year) During the First 26 Weeks of Treatment, Measured as Standing Height With Stadiometer | Baseline (week 0), week 26 | Height velocity (HV) was derived from height measurements taken at baseline (week 0) and the week 26 as: HV = (height at 26 weeks visit- height at baseline) / (time from baseline to 26 weeks visit in years). |
| Cohort II and Cohort III - Adverse Events Rate, Including Injection Site Reactions in Children With GHD. | From week 156 up to week 364 | This primary outcome measure was analysed by cohort using descriptive statistics. Adverse event per 100 patient years are presented in this outcome measure. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Cohort I: Change in Height Standard Deviation Score (HSDS) | Baseline (Week 0), week 26, week 52 | Change in height standard deviation score is presented from baseline (week 0) to end of the main trial period week 26 and end of extension trial period week 52. The formula to calculate HSDS is: HSDS = ((Height / M)\*\*L-1) / (L\*S). L: The gender and age-specific power in the Box-Cox transformation, M: The gender and age-specific median, S: The gender and age-specific generalized coefficient of variation. The range for HSDS was -10 to +10. Negative scores indicated a height below the mean height for a child with the same age and gender, whereas positive scores indicated a height above the mean height for a child with the same age and gender. |
| Cohort I: Change in Height Velocity Standard Deviation Score (HVSDS) | Baseline (Week 0), week 26, week 52 | Change in height velocity standard deviation score is presented from baseline (week 0) to end of main trial period week 26 and end of extension trial period week 52. HVSDS was calculated using the formula: HVSDS = (height velocity - mean)/standard deviation (SD), where height velocity was the height velocity variable measured, mean and SD of height velocity by gender and age for the reference population. The range for HVSDS was -10 to +10. Negative scores indicated a height velocity below the mean height velocity for a child with the same age and gender, whereas positive scores indicated a height velocity above the mean height velocity for a child with the same age and gender. |
| Cohort I: Adverse Events Rate, Including Injection Site Reactions | From week 0 Up to week 364 | Adverse events per 100 patient years are presented. AEs with an onset after the first administration of trial product and up until 14 days after last trial drug administration for withdrawn participants, and with an onset after the first administration of trial product and up until visit 32 (week 364) or 14 days after last trial drug administration, which ever comes first, for all other participants, are analysed. |
| Cohort I: Occurrence of Anti-NNC0195-0092 and Anti-hGH Antibodies | From week 0 Up to week 364 | Participants who developed anti-NNC0195-0092 and anti-hGH antibodies are reported in this outcome measure. |
| Change in Insulin-like Growth Factor I (IGF-I) Standard Deviation Score (SDS) | (Week 0), week 26, week 52 | Change in IGF-I SDS is presented from baseline (week 0) to end of main trial period week 26 and end of extension period week 52. The range for IGF-I SDS was from -10 to +10. Negative scores indicated a IGF-I below the mean IGF-I for a child with the same age and gender, whereas positive scores indicated a IGF-I above the mean IGF-I for a child with the same age and gender. |
| Change in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) Standard Deviation Score (SDS) | Baseline (Week 0), week 26, week 52 | Change in IGFBP-3 SDS is presented from baseline (week 0) to end of main trial period week 26 and end of extension trial period week 52. The range for IGFBP-3 SDS was from -10 to +10. Negative scores indicated a IGFBP-3 below the mean IGFBP-3 for a child with the same age and gender, whereas positive scores indicated a IGFBP-3 above the mean IGFBP-3 for a child with the same age and gender. |
| Height Velocity (HV) (cm/Year) at Weeks 52 (Derived From Standing Height) | Baseline (week 0); week 52 | HV was derived from height measurements taken at baseline (week 0) and the week 52 as: HV = (height at 52 weeks visit-height at baseline) / (time from baseline to 52 weeks visit in years) |
| Bone Age Progression vs. Chronological Age Ratio | At week 52 | The bone age vs. chronological age ratio is presented at week 52. X-Ray of left hand and wrist, central assessed according to Greulich \& Pyle atlas were taken. |
| Serum Somapacitan Concentrations | At week 52 | Serum somapacitan concentrations are presented at week 52. |
| Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Baseline (Week 0), week 26, week 52 | Change in Treatment Related Impact Measure from baseline (week 0) to week 26 and week 52 were assessed in children with growth hormone deficiency. This outcome measure was assessed using patient reported outcome (PRO) questionnaires with 3 domains, such as emotional well-being score, physical health score, social wellbeing core and total score. The total score was calculated by taking average of each domain. The scale range for each domain and total score was from 0-100 and a lower score indicates a better health state. TRIMCGHD-O was analysed using descriptive statistics. |
| Total Score of TB-CGHD-O (The Treatment Burden Measure - Child Growth Hormone Deficiency - Observer) | At week 26, at week 52 | Total score of Treatment Burden Measure (observer) was assessed at week 26 and at week 52 in children with growth hormone deficiency. This outcome measure was assessed using PRO questionnaires. The scale range for total score was from 0-100 and a lower score indicates a better health state. |
| Total Score of TB-CGHD-P (The Treatment Burden Measure - Child Growth Hormone Deficiency - Parent/Guardian) | At week 26, at week 52 | Total score of Treatment Burden Measure (parent/guardian) is reported at week 26 and at week 52 in children with growth hormone deficiency. This outcome measure was assessed using PRO questionnaires. The scale range for total score was from 0-100 and a lower score indicates a better health state. |
Countries
Austria, Belgium, Brazil, Canada, France, Germany, India, Israel, Japan, Slovenia, Sweden, Turkey (Türkiye), Ukraine, United States
Contacts
STUDY_DIRECTORGlobal Clinical Registry (GCR, 1452)
Novo Nordisk A/S
Participant flow
Recruitment details
The trial was conducted at 30 sites in 11 countries.
Pre-assignment details
The study has 3 cohorts. Cohort I participants were randomised (1:1:1:1) to receive norditropin/somapacitan(0.04/0.08/0.16 mg/kg) until week 52. After week 52, participants who were randomised to somapacitan were allocated to open labelled somapacitan and those who randomised to norditropin received same treatment. After week 156, all cohort I participants received somapacitan until August 24. Participants in cohorts II and III were given somapacitan from enrolment (week 156) until August 24.
Participants by arm
| Arm | Count |
|---|---|
| Cohort I Norditropin/Somapacitan Participants received norditropin 0.034 mg/kg subcutaneously daily in main trial period, extension trial period and safety extension trial period. After completing the safety extension trial period (week 156), participants who received norditropin were allocated to open-labelled somapacitan 0.16 mg/kg subcutaneously once weekly for the 104-week safety extension trial period, the 208-week (up till week 364) long-term safety extension period and extension after week 364 period until somapacitan was available for prescription for children with GHD in their country or until August 2024, at the latest. | 14 |
| Cohort I Somapacitan 0.04 mg/kg Participants received somapacitan 0.04 mg/kg subcutaneously once-weekly during the 26-week main trial period and the 26-week extension trial period. After completing the main and extension trial periods (week 52), participants received open-labelled somapacitan 0.16 mg/kg/week subcutaneously for the 104-week safety extension trial period, the 208-week (up till week 364) long-term safety extension period and extension after week 364 period until somapacitan was available for prescription for children with GHD in their country or until August 2024, at the latest. | 16 |
| Cohort I Somapacitan 0.08 mg/kg Participants received somapacitan 0.08 mg/kg subcutaneously once-weekly during the 26-week main trial period and the 26-week extension trial period. After completing the main and extension trial periods (week 52), participants received open-labelled somapacitan 0.16 mg/kg/week subcutaneously for the 104-week safety extension trial period, the 208-week (up till week 364) long-term safety extension period and extension after week 364 period until somapacitan was available for prescription for children with GHD in their country or until August 2024, at the latest. | 15 |
| Cohort I Somapacitan 0.16 mg/kg Participants received somapacitan 0.16 mg/kg subcutaneously once-weekly during the 26-week main trial period and the 26-week extension trial period. After completing the main and extension trial periods (week 52), participants received open-labelled somapacitan 0.16 mg/kg/week subcutaneously for the 104-week safety extension trial period, the 208-week (up till week 364) long-term safety extension period and extension after week 364 period until somapacitan was available for prescription for children with GHD in their country or until August 2024, at the latest. | 14 |
| Cohort II Somapacitan Previously Treated Participant who was previously treated with GH prior to enrollment in the trial at week 156, received somapacitan 0.16 mg/kg subcutaneously once weekly until it was available for prescription in participants' respective countries or until August 2024, at the latest. | 1 |
| Cohort III Somapacitan Treatment Naive Participants who were naive to treatment with GH prior to enrollment in the trial at week 156, received open-labelled somapacitan 0.16 mg/kg subcutaneously once weekly until it was available for prescription in participants' respective countries or until August 2024, at the latest. | 4 |
| Cohort III Somapacitan Previously Treated Participants who were previously treated with GH prior to enrollment in the trial at week 156, received open-labelled somapacitan 0.16 mg/kg subcutaneously once weekly until it was available for prescription in participants' respective countries or until August 2024, at the latest. | 12 |
| Total | 76 |
Baseline characteristics
| Characteristic | Cohort I Norditropin/Somapacitan | Cohort I Somapacitan 0.04 mg/kg | Cohort I Somapacitan 0.08 mg/kg | Cohort I Somapacitan 0.16 mg/kg | Cohort II Somapacitan Previously Treated | Cohort III Somapacitan Treatment Naive | Cohort III Somapacitan Previously Treated | Total |
|---|---|---|---|---|---|---|---|---|
| Age, Continuous | 5.89 Years STANDARD_DEVIATION 1.98 | 5.61 Years STANDARD_DEVIATION 1.81 | 5.82 Years STANDARD_DEVIATION 1.82 | 6.06 Years STANDARD_DEVIATION 2.32 | 2.5 Years STANDARD_DEVIATION 0 | 12.93 Years STANDARD_DEVIATION 2.24 | 12.69 Years STANDARD_DEVIATION 2.02 | 7.25 Years STANDARD_DEVIATION 3.47 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 1 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 1 Participants | 2 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 14 Participants | 15 Participants | 15 Participants | 14 Participants | 1 Participants | 4 Participants | 11 Participants | 74 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 6 Participants | 8 Participants | 6 Participants | 8 Participants | 1 Participants | 1 Participants | 3 Participants | 33 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 7 Participants | 8 Participants | 9 Participants | 6 Participants | 0 Participants | 3 Participants | 9 Participants | 42 Participants |
| Sex: Female, Male Female | 5 Participants | 7 Participants | 5 Participants | 6 Participants | 0 Participants | 1 Participants | 1 Participants | 25 Participants |
| Sex: Female, Male Male | 9 Participants | 9 Participants | 10 Participants | 8 Participants | 1 Participants | 3 Participants | 11 Participants | 51 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk | EG005 affected / at risk | EG006 affected / at risk | EG007 affected / at risk | EG008 affected / at risk | EG009 affected / at risk |
|---|---|---|---|---|---|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 14 | 0 / 11 | 0 / 5 | 0 / 16 | 0 / 15 | 0 / 14 | 0 / 44 | 0 / 1 | 0 / 4 | 0 / 12 |
| other Total, other adverse events | 14 / 14 | 10 / 11 | 2 / 5 | 11 / 16 | 11 / 15 | 13 / 14 | 35 / 44 | 1 / 1 | 2 / 4 | 9 / 12 |
| serious Total, serious adverse events | 2 / 14 | 0 / 11 | 0 / 5 | 0 / 16 | 1 / 15 | 1 / 14 | 6 / 44 | 1 / 1 | 0 / 4 | 0 / 12 |
Outcome results
Primary
Cohort II and Cohort III - Adverse Events Rate, Including Injection Site Reactions in Children With GHD.
This primary outcome measure was analysed by cohort using descriptive statistics. Adverse event per 100 patient years are presented in this outcome measure.
Time frame: From week 156 up to week 364
Population: The outcome measure was assessed only for Cohort II and Cohort III. All participants: all adverse events (AEs) with an onset after the first administration of trial product \& up until 14 days after last trial drug administration for withdrawn participants, \& with an onset after the first administration of trial product \& up until visit 32 (week 208) or 14 days after last trial drug administration, which ever comes first for all participants are included in the analysis.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Cohort I - Norditropin 0.034 mg/kg | Cohort II and Cohort III - Adverse Events Rate, Including Injection Site Reactions in Children With GHD. | 1047 Events per 100 patient years |
| Cohort I - Somapacitan 0.04 mg/kg | Cohort II and Cohort III - Adverse Events Rate, Including Injection Site Reactions in Children With GHD. | 115.4 Events per 100 patient years |
| Cohort I - Somapacitan 0.08 mg/kg | Cohort II and Cohort III - Adverse Events Rate, Including Injection Site Reactions in Children With GHD. | 196.9 Events per 100 patient years |
Primary
Height Velocity (HV) (cm/Year) During the First 26 Weeks of Treatment, Measured as Standing Height With Stadiometer
Height velocity (HV) was derived from height measurements taken at baseline (week 0) and the week 26 as: HV = (height at 26 weeks visit- height at baseline) / (time from baseline to 26 weeks visit in years).
Time frame: Baseline (week 0), week 26
Population: FAS was used to analyse this outcome measure. FAS is defined as all randomized participants that received at least one dose of randomized treatment. Here, Overall number of participants analysed = participants with available data for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Cohort I - Norditropin 0.034 mg/kg | Height Velocity (HV) (cm/Year) During the First 26 Weeks of Treatment, Measured as Standing Height With Stadiometer | 11.35 Centimetre per year (Cm/year) | Standard Deviation 3.27 |
| Cohort I - Somapacitan 0.04 mg/kg | Height Velocity (HV) (cm/Year) During the First 26 Weeks of Treatment, Measured as Standing Height With Stadiometer | 7.96 Centimetre per year (Cm/year) | Standard Deviation 2.04 |
| Cohort I - Somapacitan 0.08 mg/kg | Height Velocity (HV) (cm/Year) During the First 26 Weeks of Treatment, Measured as Standing Height With Stadiometer | 10.92 Centimetre per year (Cm/year) | Standard Deviation 1.9 |
| Cohort I - Somapcitan 0.16 mg/kg | Height Velocity (HV) (cm/Year) During the First 26 Weeks of Treatment, Measured as Standing Height With Stadiometer | 12.88 Centimetre per year (Cm/year) | Standard Deviation 3.46 |
Comparison: The primary analysis tested the estimated treatment difference in HV after 26 weeks of treatment between once-weekly somapacitan 0.04 mg/kg and daily dosing of norditropin 0.034 mg/kg. It was analysed using a mixed model for repeated measurements, with treatment, age group, sex, region and sex by age group interaction as factors and height at baseline as a covariate, all nested within week as a factor.95% CI: [-5.57, -1.76]
Comparison: The primary analysis tested the estimated treatment difference in HV after 26 weeks of treatment between once-weekly somapacitan 0.08 mg/kg and daily dosing of norditropin 0.034 mg/kg. It was analysed using a mixed model for repeated measurements, with treatment, age group, sex, region and sex by age group interaction as factors and height at baseline as a covariate, all nested within week as a factor.95% CI: [-2.41, 1.32]
Comparison: The primary analysis tested the estimated treatment difference in HV after 26 weeks of treatment between once-weekly somapacitan 0.16 mg/kg and daily dosing of norditropin 0.034 mg/kg. It was analysed using a mixed model for repeated measurements, with treatment, age group, sex, region and sex by age group interaction as factors and height at baseline as a covariate, all nested within week as a factor.95% CI: [-0.22, 3.56]
Secondary
Bone Age Progression vs. Chronological Age Ratio
The bone age vs. chronological age ratio is presented at week 52. X-Ray of left hand and wrist, central assessed according to Greulich & Pyle atlas were taken.
Time frame: At week 52
Population: This outcome measure was assessed only for Cohort 1 and FAS was used to analyse this outcome measure. FAS is defined as all randomized participants that received at least one dose of randomized treatment. Here, Overall number of participants analysed (N) = participants with available data for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Cohort I - Norditropin 0.034 mg/kg | Bone Age Progression vs. Chronological Age Ratio | 0.577 Ratio | Standard Deviation 0.176 |
| Cohort I - Somapacitan 0.04 mg/kg | Bone Age Progression vs. Chronological Age Ratio | 0.570 Ratio | Standard Deviation 0.147 |
| Cohort I - Somapacitan 0.08 mg/kg | Bone Age Progression vs. Chronological Age Ratio | 0.651 Ratio | Standard Deviation 0.176 |
| Cohort I - Somapcitan 0.16 mg/kg | Bone Age Progression vs. Chronological Age Ratio | 0.705 Ratio | Standard Deviation 0.207 |
Secondary
Change in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) Standard Deviation Score (SDS)
Change in IGFBP-3 SDS is presented from baseline (week 0) to end of main trial period week 26 and end of extension trial period week 52. The range for IGFBP-3 SDS was from -10 to +10. Negative scores indicated a IGFBP-3 below the mean IGFBP-3 for a child with the same age and gender, whereas positive scores indicated a IGFBP-3 above the mean IGFBP-3 for a child with the same age and gender.
Time frame: Baseline (Week 0), week 26, week 52
Population: This outcome measure was assessed only for Cohort 1 and FAS was used to analyse this outcome measure. FAS is defined as all randomised participants that received at least one dose of randomised treatment. Here, Overall number of participants analysed = participants with available data for this outcome measure. Number analysed = participants analysed for specific category for this outcome measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Cohort I - Norditropin 0.034 mg/kg | Change in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) Standard Deviation Score (SDS) | Change at week 26 | 1.58 Score on scale | Standard Deviation 0.99 |
| Cohort I - Norditropin 0.034 mg/kg | Change in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) Standard Deviation Score (SDS) | Change at week 52 | 0.95 Score on scale | Standard Deviation 1.88 |
| Cohort I - Somapacitan 0.04 mg/kg | Change in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) Standard Deviation Score (SDS) | Change at week 52 | 1.05 Score on scale | Standard Deviation 0.87 |
| Cohort I - Somapacitan 0.04 mg/kg | Change in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) Standard Deviation Score (SDS) | Change at week 26 | 0.86 Score on scale | Standard Deviation 0.73 |
| Cohort I - Somapacitan 0.08 mg/kg | Change in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) Standard Deviation Score (SDS) | Change at week 26 | 1.59 Score on scale | Standard Deviation 1.04 |
| Cohort I - Somapacitan 0.08 mg/kg | Change in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) Standard Deviation Score (SDS) | Change at week 52 | 1.54 Score on scale | Standard Deviation 1.13 |
| Cohort I - Somapcitan 0.16 mg/kg | Change in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) Standard Deviation Score (SDS) | Change at week 26 | 1.45 Score on scale | Standard Deviation 1.33 |
| Cohort I - Somapcitan 0.16 mg/kg | Change in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) Standard Deviation Score (SDS) | Change at week 52 | 1.81 Score on scale | Standard Deviation 1.39 |
Secondary
Change in Insulin-like Growth Factor I (IGF-I) Standard Deviation Score (SDS)
Change in IGF-I SDS is presented from baseline (week 0) to end of main trial period week 26 and end of extension period week 52. The range for IGF-I SDS was from -10 to +10. Negative scores indicated a IGF-I below the mean IGF-I for a child with the same age and gender, whereas positive scores indicated a IGF-I above the mean IGF-I for a child with the same age and gender.
Time frame: (Week 0), week 26, week 52
Population: This outcome measure was assessed only for Cohort 1 and FAS was used to analyse this outcome measure. FAS is defined as all randomised participants that received at least one dose of randomised treatment. Here, Overall number of participants analysed = participants with available data for this outcome measure. Number analysed = participants analysed for specific category for this outcome measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Cohort I - Norditropin 0.034 mg/kg | Change in Insulin-like Growth Factor I (IGF-I) Standard Deviation Score (SDS) | Change at week 26 | 1.86 Score on scale | Standard Deviation 0.81 |
| Cohort I - Norditropin 0.034 mg/kg | Change in Insulin-like Growth Factor I (IGF-I) Standard Deviation Score (SDS) | Change at week 52 | 1.67 Score on scale | Standard Deviation 1.78 |
| Cohort I - Somapacitan 0.04 mg/kg | Change in Insulin-like Growth Factor I (IGF-I) Standard Deviation Score (SDS) | Change at week 52 | 0.98 Score on scale | Standard Deviation 0.67 |
| Cohort I - Somapacitan 0.04 mg/kg | Change in Insulin-like Growth Factor I (IGF-I) Standard Deviation Score (SDS) | Change at week 26 | 0.95 Score on scale | Standard Deviation 0.55 |
| Cohort I - Somapacitan 0.08 mg/kg | Change in Insulin-like Growth Factor I (IGF-I) Standard Deviation Score (SDS) | Change at week 26 | 1.99 Score on scale | Standard Deviation 1.02 |
| Cohort I - Somapacitan 0.08 mg/kg | Change in Insulin-like Growth Factor I (IGF-I) Standard Deviation Score (SDS) | Change at week 52 | 2.05 Score on scale | Standard Deviation 1 |
| Cohort I - Somapcitan 0.16 mg/kg | Change in Insulin-like Growth Factor I (IGF-I) Standard Deviation Score (SDS) | Change at week 26 | 3.00 Score on scale | Standard Deviation 1.43 |
| Cohort I - Somapcitan 0.16 mg/kg | Change in Insulin-like Growth Factor I (IGF-I) Standard Deviation Score (SDS) | Change at week 52 | 3.29 Score on scale | Standard Deviation 1.73 |
Secondary
Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer)
Change in Treatment Related Impact Measure from baseline (week 0) to week 26 and week 52 were assessed in children with growth hormone deficiency. This outcome measure was assessed using patient reported outcome (PRO) questionnaires with 3 domains, such as emotional well-being score, physical health score, social wellbeing core and total score. The total score was calculated by taking average of each domain. The scale range for each domain and total score was from 0-100 and a lower score indicates a better health state. TRIMCGHD-O was analysed using descriptive statistics.
Time frame: Baseline (Week 0), week 26, week 52
Population: This outcome measure was assessed only for Cohort 1 and FAS was used to analyse this outcome measure. FAS is defined as all randomized participants that received at least one dose of randomized treatment. Here, Overall number of participants analysed = participants with available data for this outcome measure. Number analysed = participants analysed for specific category for this outcome measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Cohort I - Norditropin 0.034 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Physical health score at week 26 | 4.3 Score on scale | Standard Deviation 18.8 |
| Cohort I - Norditropin 0.034 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Total score at week 26 | -4.6 Score on scale | Standard Deviation 10.7 |
| Cohort I - Norditropin 0.034 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Social well-being score at week 52 | -9.5 Score on scale | Standard Deviation 23.9 |
| Cohort I - Norditropin 0.034 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Emotional well-being score at week 26 | -3.3 Score on scale | Standard Deviation 19.8 |
| Cohort I - Norditropin 0.034 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Social well-being score at week 26 | -9.6 Score on scale | Standard Deviation 17.5 |
| Cohort I - Norditropin 0.034 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Emotional well-being score at week 52 | -5.7 Score on scale | Standard Deviation 13.2 |
| Cohort I - Norditropin 0.034 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Total score at week 52 | -6.1 Score on scale | Standard Deviation 10.2 |
| Cohort I - Norditropin 0.034 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Physical health score at week 52 | -1.9 Score on scale | Standard Deviation 13.4 |
| Cohort I - Somapacitan 0.04 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Emotional well-being score at week 52 | 1.4 Score on scale | Standard Deviation 18.7 |
| Cohort I - Somapacitan 0.04 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Social well-being score at week 26 | 0.6 Score on scale | Standard Deviation 20.3 |
| Cohort I - Somapacitan 0.04 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Physical health score at week 26 | -4.0 Score on scale | Standard Deviation 10.3 |
| Cohort I - Somapacitan 0.04 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Physical health score at week 52 | 0.9 Score on scale | Standard Deviation 13.8 |
| Cohort I - Somapacitan 0.04 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Social well-being score at week 52 | 4.5 Score on scale | Standard Deviation 20.6 |
| Cohort I - Somapacitan 0.04 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Total score at week 26 | -0.7 Score on scale | Standard Deviation 13.6 |
| Cohort I - Somapacitan 0.04 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Total score at week 52 | 1.8 Score on scale | Standard Deviation 13.5 |
| Cohort I - Somapacitan 0.04 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Emotional well-being score at week 26 | 1.0 Score on scale | Standard Deviation 24.9 |
| Cohort I - Somapacitan 0.08 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Emotional well-being score at week 26 | 1.1 Score on scale | Standard Deviation 22 |
| Cohort I - Somapacitan 0.08 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Physical health score at week 52 | -1.0 Score on scale | Standard Deviation 20.9 |
| Cohort I - Somapacitan 0.08 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Physical health score at week 26 | -2.8 Score on scale | Standard Deviation 27 |
| Cohort I - Somapacitan 0.08 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Social well-being score at week 26 | -8.6 Score on scale | Standard Deviation 17.8 |
| Cohort I - Somapacitan 0.08 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Emotional well-being score at week 52 | -8.0 Score on scale | Standard Deviation 22.7 |
| Cohort I - Somapacitan 0.08 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Social well-being score at week 52 | -20.5 Score on scale | Standard Deviation 20.4 |
| Cohort I - Somapacitan 0.08 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Total score at week 52 | -9.4 Score on scale | Standard Deviation 17.3 |
| Cohort I - Somapacitan 0.08 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Total score at week 26 | -3.9 Score on scale | Standard Deviation 16.4 |
| Cohort I - Somapcitan 0.16 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Total score at week 52 | -12.7 Score on scale | Standard Deviation 12.3 |
| Cohort I - Somapcitan 0.16 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Physical health score at week 52 | -6.7 Score on scale | Standard Deviation 16.8 |
| Cohort I - Somapcitan 0.16 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Social well-being score at week 52 | -17.9 Score on scale | Standard Deviation 21.2 |
| Cohort I - Somapcitan 0.16 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Emotional well-being score at week 26 | 2.4 Score on scale | Standard Deviation 28.4 |
| Cohort I - Somapcitan 0.16 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Total score at week 26 | -5.9 Score on scale | Standard Deviation 12.6 |
| Cohort I - Somapcitan 0.16 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Physical health score at week 26 | -11.6 Score on scale | Standard Deviation 16.9 |
| Cohort I - Somapcitan 0.16 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Emotional well-being score at week 52 | -12.2 Score on scale | Standard Deviation 27.9 |
| Cohort I - Somapcitan 0.16 mg/kg | Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer) | Social well-being score at week 26 | -11.0 Score on scale | Standard Deviation 22.7 |
Secondary
Cohort I: Adverse Events Rate, Including Injection Site Reactions
Adverse events per 100 patient years are presented. AEs with an onset after the first administration of trial product and up until 14 days after last trial drug administration for withdrawn participants, and with an onset after the first administration of trial product and up until visit 32 (week 364) or 14 days after last trial drug administration, which ever comes first, for all other participants, are analysed.
Time frame: From week 0 Up to week 364
Population: Safety analysis set (SAS) is defined as all randomised participants that received at least one dose of randomised treatment. Data for 3 somapacitan arms (0.04/0.08/0.16 mg/kg) have been pooled from week 156-364, because it was considered appropriate to pool the data from those arms since the size of each individual cohort was small and it would give a more robust measure of long-term safety and tolerability of treatment with somapacitan 0.16 mg/kg/week using this methodology.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Cohort I - Norditropin 0.034 mg/kg | Cohort I: Adverse Events Rate, Including Injection Site Reactions | 247.7 Events per 100 patient years |
| Cohort I - Somapacitan 0.04 mg/kg | Cohort I: Adverse Events Rate, Including Injection Site Reactions | 116.3 Events per 100 patient years |
| Cohort I - Somapacitan 0.08 mg/kg | Cohort I: Adverse Events Rate, Including Injection Site Reactions | 210.4 Events per 100 patient years |
| Cohort I - Somapcitan 0.16 mg/kg | Cohort I: Adverse Events Rate, Including Injection Site Reactions | 311.5 Events per 100 patient years |
| Cohort I Somapacitan 0.16 mg/kg Week 0-52 | Cohort I: Adverse Events Rate, Including Injection Site Reactions | 364.8 Events per 100 patient years |
| Cohort I Somapacitan 0.16 mg/kg (Previous Dose 0.04 mg/kg) Week 52-156 | Cohort I: Adverse Events Rate, Including Injection Site Reactions | 163.1 Events per 100 patient years |
| Cohort I Somapacitan 0.16 mg/kg (Previous Dose 0.08 mg/kg) Week 52-156 | Cohort I: Adverse Events Rate, Including Injection Site Reactions | 281.2 Events per 100 patient years |
| Cohort I Somapacitan 0.16 mg/kg (Previous Dose 0.16 mg/kg) Week 52-156 | Cohort I: Adverse Events Rate, Including Injection Site Reactions | 264.9 Events per 100 patient years |
| Cohort I Somapacitan 0.16 mg/kg Week 156 - 364 | Cohort I: Adverse Events Rate, Including Injection Site Reactions | 186.3 Events per 100 patient years |
Secondary
Cohort I: Change in Height Standard Deviation Score (HSDS)
Change in height standard deviation score is presented from baseline (week 0) to end of the main trial period week 26 and end of extension trial period week 52. The formula to calculate HSDS is: HSDS = ((Height / M)\*\*L-1) / (L\*S). L: The gender and age-specific power in the Box-Cox transformation, M: The gender and age-specific median, S: The gender and age-specific generalized coefficient of variation. The range for HSDS was -10 to +10. Negative scores indicated a height below the mean height for a child with the same age and gender, whereas positive scores indicated a height above the mean height for a child with the same age and gender.
Time frame: Baseline (Week 0), week 26, week 52
Population: This outcome measure was assessed only for Cohort 1 and FAS was used to analyse this outcome measure. FAS is defined as all randomised participants that received at least one dose of randomised treatment. Here, Overall number of participants analysed = participants with available data for this outcome measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Cohort I - Norditropin 0.034 mg/kg | Cohort I: Change in Height Standard Deviation Score (HSDS) | Change at week 26 | 0.66 Score on scale | Standard Deviation 0.38 |
| Cohort I - Norditropin 0.034 mg/kg | Cohort I: Change in Height Standard Deviation Score (HSDS) | Change at week 52 | 0.98 Score on scale | Standard Deviation 0.5 |
| Cohort I - Somapacitan 0.04 mg/kg | Cohort I: Change in Height Standard Deviation Score (HSDS) | Change at week 52 | 0.59 Score on scale | Standard Deviation 0.46 |
| Cohort I - Somapacitan 0.04 mg/kg | Cohort I: Change in Height Standard Deviation Score (HSDS) | Change at week 26 | 0.31 Score on scale | Standard Deviation 0.29 |
| Cohort I - Somapacitan 0.08 mg/kg | Cohort I: Change in Height Standard Deviation Score (HSDS) | Change at week 26 | 0.63 Score on scale | Standard Deviation 0.29 |
| Cohort I - Somapacitan 0.08 mg/kg | Cohort I: Change in Height Standard Deviation Score (HSDS) | Change at week 52 | 0.95 Score on scale | Standard Deviation 0.48 |
| Cohort I - Somapcitan 0.16 mg/kg | Cohort I: Change in Height Standard Deviation Score (HSDS) | Change at week 26 | 0.89 Score on scale | Standard Deviation 0.51 |
| Cohort I - Somapcitan 0.16 mg/kg | Cohort I: Change in Height Standard Deviation Score (HSDS) | Change at week 52 | 1.45 Score on scale | Standard Deviation 0.86 |
Secondary
Cohort I: Change in Height Velocity Standard Deviation Score (HVSDS)
Change in height velocity standard deviation score is presented from baseline (week 0) to end of main trial period week 26 and end of extension trial period week 52. HVSDS was calculated using the formula: HVSDS = (height velocity - mean)/standard deviation (SD), where height velocity was the height velocity variable measured, mean and SD of height velocity by gender and age for the reference population. The range for HVSDS was -10 to +10. Negative scores indicated a height velocity below the mean height velocity for a child with the same age and gender, whereas positive scores indicated a height velocity above the mean height velocity for a child with the same age and gender.
Time frame: Baseline (Week 0), week 26, week 52
Population: This outcome measure was assessed only for Cohort 1 and FAS was used to analyse this outcome measure. FAS is defined as all randomised participants that received at least one dose of randomised treatment. Here, Overall number of participants analysed = participants with available data for this outcome measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Cohort I - Norditropin 0.034 mg/kg | Cohort I: Change in Height Velocity Standard Deviation Score (HVSDS) | Change at week 26 | 9.02 Score on scale | Standard Deviation 5.03 |
| Cohort I - Norditropin 0.034 mg/kg | Cohort I: Change in Height Velocity Standard Deviation Score (HVSDS) | Change at week 52 | 7.41 Score on scale | Standard Deviation 4.08 |
| Cohort I - Somapacitan 0.04 mg/kg | Cohort I: Change in Height Velocity Standard Deviation Score (HVSDS) | Change at week 52 | 4.72 Score on scale | Standard Deviation 2.79 |
| Cohort I - Somapacitan 0.04 mg/kg | Cohort I: Change in Height Velocity Standard Deviation Score (HVSDS) | Change at week 26 | 4.93 Score on scale | Standard Deviation 3.25 |
| Cohort I - Somapacitan 0.08 mg/kg | Cohort I: Change in Height Velocity Standard Deviation Score (HVSDS) | Change at week 26 | 7.27 Score on scale | Standard Deviation 3.76 |
| Cohort I - Somapacitan 0.08 mg/kg | Cohort I: Change in Height Velocity Standard Deviation Score (HVSDS) | Change at week 52 | 6.14 Score on scale | Standard Deviation 3.36 |
| Cohort I - Somapcitan 0.16 mg/kg | Cohort I: Change in Height Velocity Standard Deviation Score (HVSDS) | Change at week 26 | 10.01 Score on scale | Standard Deviation 4.67 |
| Cohort I - Somapcitan 0.16 mg/kg | Cohort I: Change in Height Velocity Standard Deviation Score (HVSDS) | Change at week 52 | 8.60 Score on scale | Standard Deviation 3.15 |
Secondary
Cohort I: Occurrence of Anti-NNC0195-0092 and Anti-hGH Antibodies
Participants who developed anti-NNC0195-0092 and anti-hGH antibodies are reported in this outcome measure.
Time frame: From week 0 Up to week 364
Population: This outcome measure was assessed only for Cohort 1 and SAS was used to analyse this outcome measure. SAS is defined as all randomized participants that received at least one dose of randomized treatment.~Participants who were randomised to somapacitan 0.04/0.08/0.16 mg/kg/week) received respective doses from week 0 to week 52. From week 52 to week 364, all randomised participants received open labelled somapacitan 0.16 mg/kg/week.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Cohort I - Norditropin 0.034 mg/kg | Cohort I: Occurrence of Anti-NNC0195-0092 and Anti-hGH Antibodies | Anti-NNC0195-0092 antibody | 0 Participants |
| Cohort I - Norditropin 0.034 mg/kg | Cohort I: Occurrence of Anti-NNC0195-0092 and Anti-hGH Antibodies | Anti-hGH antibody | 1 Participants |
| Cohort I - Somapacitan 0.04 mg/kg | Cohort I: Occurrence of Anti-NNC0195-0092 and Anti-hGH Antibodies | Anti-NNC0195-0092 antibody | 2 Participants |
| Cohort I - Somapacitan 0.04 mg/kg | Cohort I: Occurrence of Anti-NNC0195-0092 and Anti-hGH Antibodies | Anti-hGH antibody | 0 Participants |
| Cohort I - Somapacitan 0.08 mg/kg | Cohort I: Occurrence of Anti-NNC0195-0092 and Anti-hGH Antibodies | Anti-hGH antibody | 0 Participants |
| Cohort I - Somapacitan 0.08 mg/kg | Cohort I: Occurrence of Anti-NNC0195-0092 and Anti-hGH Antibodies | Anti-NNC0195-0092 antibody | 2 Participants |
| Cohort I - Somapcitan 0.16 mg/kg | Cohort I: Occurrence of Anti-NNC0195-0092 and Anti-hGH Antibodies | Anti-NNC0195-0092 antibody | 2 Participants |
| Cohort I - Somapcitan 0.16 mg/kg | Cohort I: Occurrence of Anti-NNC0195-0092 and Anti-hGH Antibodies | Anti-hGH antibody | 0 Participants |
| Cohort I Somapacitan 0.16 mg/kg Week 0-52 | Cohort I: Occurrence of Anti-NNC0195-0092 and Anti-hGH Antibodies | Anti-NNC0195-0092 antibody | 6 Participants |
| Cohort I Somapacitan 0.16 mg/kg Week 0-52 | Cohort I: Occurrence of Anti-NNC0195-0092 and Anti-hGH Antibodies | Anti-hGH antibody | 0 Participants |
Secondary
Height Velocity (HV) (cm/Year) at Weeks 52 (Derived From Standing Height)
HV was derived from height measurements taken at baseline (week 0) and the week 52 as: HV = (height at 52 weeks visit-height at baseline) / (time from baseline to 52 weeks visit in years)
Time frame: Baseline (week 0); week 52
Population: This outcome measure was assessed only for Cohort 1 and FAS was used to analyse this outcome measure. FAS is defined as all randomized participants that received at least one dose of randomized treatment. Here, Overall number of participants analysed = participants with available data for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Cohort I - Norditropin 0.034 mg/kg | Height Velocity (HV) (cm/Year) at Weeks 52 (Derived From Standing Height) | 9.8 Cm/year | Standard Deviation 2.3 |
| Cohort I - Somapacitan 0.04 mg/kg | Height Velocity (HV) (cm/Year) at Weeks 52 (Derived From Standing Height) | 7.8 Cm/year | Standard Deviation 1.8 |
| Cohort I - Somapacitan 0.08 mg/kg | Height Velocity (HV) (cm/Year) at Weeks 52 (Derived From Standing Height) | 9.7 Cm/year | Standard Deviation 1.8 |
| Cohort I - Somapcitan 0.16 mg/kg | Height Velocity (HV) (cm/Year) at Weeks 52 (Derived From Standing Height) | 11.5 Cm/year | Standard Deviation 2.6 |
Secondary
Serum Somapacitan Concentrations
Serum somapacitan concentrations are presented at week 52.
Time frame: At week 52
Population: This outcome measure was assessed only for Cohort 1 and FAS was used to analyse this outcome measure. FAS is defined as all randomized participants that received at least one dose of randomized treatment. Here, Overall number of participants analysed (N) = participants with available data for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Cohort I - Norditropin 0.034 mg/kg | Serum Somapacitan Concentrations | 18.381 Ng/mL | Standard Deviation 20.911 |
| Cohort I - Somapacitan 0.04 mg/kg | Serum Somapacitan Concentrations | 44.563 Ng/mL | Standard Deviation 60.922 |
| Cohort I - Somapacitan 0.08 mg/kg | Serum Somapacitan Concentrations | 169.320 Ng/mL | Standard Deviation 212.233 |
Secondary
Total Score of TB-CGHD-O (The Treatment Burden Measure - Child Growth Hormone Deficiency - Observer)
Total score of Treatment Burden Measure (observer) was assessed at week 26 and at week 52 in children with growth hormone deficiency. This outcome measure was assessed using PRO questionnaires. The scale range for total score was from 0-100 and a lower score indicates a better health state.
Time frame: At week 26, at week 52
Population: This outcome measure was assessed only for Cohort 1 and FAS was used to analyse this outcome measure. FAS is defined as all randomized participants that received at least one dose of randomized treatment. Here, Overall number of participants analysed = participants with available data for this outcome measure. Number analysed = participants analysed for specific category for this outcome measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Cohort I - Norditropin 0.034 mg/kg | Total Score of TB-CGHD-O (The Treatment Burden Measure - Child Growth Hormone Deficiency - Observer) | Total score at week 26 | 14.8 Score on scale | Standard Deviation 16.2 |
| Cohort I - Norditropin 0.034 mg/kg | Total Score of TB-CGHD-O (The Treatment Burden Measure - Child Growth Hormone Deficiency - Observer) | Total score at week 52 | 12.3 Score on scale | Standard Deviation 13.9 |
| Cohort I - Somapacitan 0.04 mg/kg | Total Score of TB-CGHD-O (The Treatment Burden Measure - Child Growth Hormone Deficiency - Observer) | Total score at week 52 | 13.7 Score on scale | Standard Deviation 13.2 |
| Cohort I - Somapacitan 0.04 mg/kg | Total Score of TB-CGHD-O (The Treatment Burden Measure - Child Growth Hormone Deficiency - Observer) | Total score at week 26 | 14.0 Score on scale | Standard Deviation 11.9 |
| Cohort I - Somapacitan 0.08 mg/kg | Total Score of TB-CGHD-O (The Treatment Burden Measure - Child Growth Hormone Deficiency - Observer) | Total score at week 26 | 10.2 Score on scale | Standard Deviation 11 |
| Cohort I - Somapacitan 0.08 mg/kg | Total Score of TB-CGHD-O (The Treatment Burden Measure - Child Growth Hormone Deficiency - Observer) | Total score at week 52 | 11.3 Score on scale | Standard Deviation 12.3 |
| Cohort I - Somapcitan 0.16 mg/kg | Total Score of TB-CGHD-O (The Treatment Burden Measure - Child Growth Hormone Deficiency - Observer) | Total score at week 26 | 6.8 Score on scale | Standard Deviation 8.9 |
| Cohort I - Somapcitan 0.16 mg/kg | Total Score of TB-CGHD-O (The Treatment Burden Measure - Child Growth Hormone Deficiency - Observer) | Total score at week 52 | 3.9 Score on scale | Standard Deviation 8.1 |
Secondary
Total Score of TB-CGHD-P (The Treatment Burden Measure - Child Growth Hormone Deficiency - Parent/Guardian)
Total score of Treatment Burden Measure (parent/guardian) is reported at week 26 and at week 52 in children with growth hormone deficiency. This outcome measure was assessed using PRO questionnaires. The scale range for total score was from 0-100 and a lower score indicates a better health state.
Time frame: At week 26, at week 52
Population: This outcome measure was assessed only for Cohort 1 and FAS was used to analyse this outcome measure. FAS is defined as all randomized participants that received at least one dose of randomized treatment. Here, Overall number of participants analysed = participants with available data for this outcome measure. Number analysed = participants analysed for specific category for this outcome measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Cohort I - Norditropin 0.034 mg/kg | Total Score of TB-CGHD-P (The Treatment Burden Measure - Child Growth Hormone Deficiency - Parent/Guardian) | Total score at week 26 | 26.9 Score on scale | Standard Deviation 24.7 |
| Cohort I - Norditropin 0.034 mg/kg | Total Score of TB-CGHD-P (The Treatment Burden Measure - Child Growth Hormone Deficiency - Parent/Guardian) | Total score at week 52 | 23.6 Score on scale | Standard Deviation 27.3 |
| Cohort I - Somapacitan 0.04 mg/kg | Total Score of TB-CGHD-P (The Treatment Burden Measure - Child Growth Hormone Deficiency - Parent/Guardian) | Total score at week 52 | 19.6 Score on scale | Standard Deviation 19 |
| Cohort I - Somapacitan 0.04 mg/kg | Total Score of TB-CGHD-P (The Treatment Burden Measure - Child Growth Hormone Deficiency - Parent/Guardian) | Total score at week 26 | 24.6 Score on scale | Standard Deviation 16.7 |
| Cohort I - Somapacitan 0.08 mg/kg | Total Score of TB-CGHD-P (The Treatment Burden Measure - Child Growth Hormone Deficiency - Parent/Guardian) | Total score at week 26 | 17.5 Score on scale | Standard Deviation 20.5 |
| Cohort I - Somapacitan 0.08 mg/kg | Total Score of TB-CGHD-P (The Treatment Burden Measure - Child Growth Hormone Deficiency - Parent/Guardian) | Total score at week 52 | 13.8 Score on scale | Standard Deviation 12.5 |
| Cohort I - Somapcitan 0.16 mg/kg | Total Score of TB-CGHD-P (The Treatment Burden Measure - Child Growth Hormone Deficiency - Parent/Guardian) | Total score at week 26 | 18.8 Score on scale | Standard Deviation 22.2 |
| Cohort I - Somapcitan 0.16 mg/kg | Total Score of TB-CGHD-P (The Treatment Burden Measure - Child Growth Hormone Deficiency - Parent/Guardian) | Total score at week 52 | 15.6 Score on scale | Standard Deviation 16.6 |