Growth Hormone Deficiency in Children
Conditions
Brief summary
The study compares 2 medicines for children who do not have enough hormone to grow: somapacitan given once a week (a new medicine) and Norditropin® given once a day (the medicine doctors can already prescribe). Researchers will test to see how well somapacitan works. The study will also test if somapacitan is safe. Participants will either get somapacitan or Norditropin® - which treatment participants get, is decided by chance. Both participants and the study doctor will know which treatment participants get. The study will last for 4 years. Participants will attend 19 clinic visits and have 1 phone call with the study doctor.
Interventions
DRUGSomapacitan
Somapacitan will be administered subcutaneously (s.c.; under the skin) once weekly by PDS290 pen-injector. Somapacitan can be injected any time during the once weekly dosing day. The dose will be calculated based on the subject's current body weight.
DRUGNorditropin®
Norditropin® will be administered s.c. once daily by FlexPro® pen-injector. Norditropin® should be injected daily in the evening. The dose will be calculated based on the subject's current body weight.
Sponsors
Novo Nordisk A/S
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Participants will receive either somapacitan once weekly or Norditropin® once daily for 52 weeks (main trial period). All participants completing the main trial period will receive somapacitan weekly for 3 years (extension trial period).
Eligibility
Sex/Gender
ALL
Age
No minimum to 11 Years
Healthy volunteers
No
Inclusion criteria
* Prepubertal children: a) Boys: Age more than or equal to 2 years and 26 weeks and less than 11.0 years at screening. Testis volume less than 4 ml. b) Girls: Age more than or equal to 2 years and 26 weeks and less than 10.0 years at screening. Tanner stage 1 for breast development (no palpable glandular breast tissue) * Confirmed diagnosis of growth hormone deficiency determined by two different growth hormone stimulation tests performed within 12 months prior to randomisation, defined as a peak growth hormone level of less than or equal to 10.0 ng/ml using the World Health Organisation (WHO) International Somatropin 98/574 standard * Impaired height defined as at least 2.0 standard deviations below the mean height for chronological age and gender at screening according to the standards of Center for Disease Control and Prevention * Impaired height velocity, defined as annualised height velocity below the 25th percentile for chronological age and gender according to the standards of Prader calculated over a time span of minimum 6 months and maximum 18 months prior to screening * Insulin-like Growth Factor-I (IGF-I) less than -1.0 SDS at screening, compared to age and gender normalized range measured at central laboratory * No prior exposure to growth hormone therapy or IGF-I treatment
Exclusion criteria
* Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with standing height measurements * Current inflammatory diseases requiring systemic corticosteroid treatment for longer than 2 consecutive weeks within the last 3 months prior to screening * Children requiring inhaled glucocorticoid therapy at a dose of greater than 400 μg/day of inhaled budesonide or equivalents for longer than 4 consecutive weeks within the last 12 months prior to screening * Diagnosis of attention deficit hyperactivity disorder * Concomitant administration of other treatments that may have an effect on growth, e.g. but not limited to methylphenidate for treatment of attention deficit hyperactivity disorder * Prior history or presence of malignancy including intracranial tumours
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Height Velocity: In-trial Observation Period | From baseline (week 0) to visit 7 (week 52) | Height velocity (HV) was derived from height measurements taken at baseline and Week 52 visit as: HV = (height at 52 weeks visit - height at baseline)/(time from baseline to 52 weeks visit in years). Data is reported for 'in-trial' observation period. In-trial observation period: from first administration and up until visit 7 or last trial contact, whichever comes first. |
| Height Velocity: On-treatment Observation Period | From baseline (week 0) to visit 7 (week 52) | Height velocity was derived from height measurements taken at baseline and Week 52 visit as: HV = (height at 52 weeks visit - height at baseline)/(time from baseline to 52 weeks visit in years). Data is reported for 'on-treatment' observation period. On-treatment observation period: from first administration and up until last trial contact, visit 7 or 14 days after last administration, whichever comes first. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Bone Age | Baseline (week -2), week 52 | Change from baseline (week -2) in bone age at week 52 is presented. X-ray images of left hand and wrist for bone age assessment according to the Greulich and Pyle atlas were taken. In-trial observation period: from first administration and up until visit 7 or last trial contact, whichever comes first. |
| Change in Height Standard Deviation Score (HSDS) | Baseline (week 0), week 52 | Change from baseline (week 0) in HSDS at week 52 is presented. HSDS was derived using Centre for Disease Control and Prevention (CDC) standards. 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. Positive value in change from baseline in HSDS indicated that HSDS was better than baseline HSDS. In-trial observation period: from first administration and up until visit 7 or last trial contact, whichever comes first. |
| Change in Height Velocity Standard Deviation Score (HV SDS) | Baseline (week 0), week 52 | Change from baseline (week 0) in HV SDS at week 52 is presented. HV SDS was calculated using the formula: HV SDS = (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 HV SDS 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. Positive value in change from baseline in HV SDS indicated that HV SDS was better than baseline HV SDS. In-trial observation period: from first administration and up until visit 7 or last trial contact, whichever comes first. |
| Change in Fasting Plasma Glucose (FPG) at Week 52 | Baseline (week -2), week 52 | Change from baseline (week -2) in FPG at week 52 is presented. |
| Change in FPG at Week 104 | Baseline (week -2), week 104 | — |
| Change in FPG at Week 156 | Baseline (week -2), week 156 | — |
| Change in FPG at Week 208 | Baseline (week -2), week 208 | — |
| Change in Homeostatic Model Assessment Steady State Beta Cell Function (HOMA-B) at Week 52 | Baseline (week -2), week 52 | Change from baseline (week -2) in HOMA-B at week 52 is presented. HOMA-B is a measure of the beta cell function and was calculated as follows: HOMA-B = (20 \* fasting insulin (picomoles per liter \[pmol/L\]) \* 1/6(microunit per milliliter \[µU/mL\]))/ FPG(mmol/L)-3.5). Negative change from baseline in HOMA-B indicated a worse outcome. |
| Change in HOMA-B at Week 104 | Baseline (week -2), week 104 | — |
| Change in HOMA-B at Week 156 | Baseline (week -2), week 156 | — |
| Change in HOMA-B at Week 208 | Baseline (week -2), week 208 | — |
| Change in Homeostatic Model Assessment Insulin Resistance (HOMA-IR) at Week 52 | Baseline (week -2), week 52 | Change from baseline (week -2) in HOMA-IR at week 52 is presented. HOMA-IR is an evaluation of the insulin resistance and was calculated as HOMA-IR = fasting insulin (pmol/L) \* 1/6(µU/mL) \* FPG(mmol/L) / 22.5. Positive change from baseline in HOMA-IR indicated a worse outcome. |
| Change in HOMA-IR at Week 104 | Baseline (week -2), week 104 | — |
| Change in HOMA-IR at Week 156 | Baseline (week -2), week 156 | — |
| Change in HOMA-IR at Week 208 | Baseline (week -2), week 208 | — |
| Change in Glycated Haemoglobin (HbA1c) at Week 52 | Baseline (week -2), week 52 | Change from baseline (week -2) in HbA1c at week 52 is presented. |
| Change in HbA1c at Week 104 | Baseline (week -2), week 104 | — |
| Change in HbA1c at Week 156 | Baseline (week -2), week 156 | — |
| Change in HbA1c at Week 208 | Baseline (week -2), week 208 | — |
| Change in Insulin-like Growth Factor I (IGF-I) Standard Deviation Score (SDS) at Week 52 | Baseline (week 0), week 52 | Change from baseline (week 0) in IGF-I SDS at week 52 is presented. 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. For participants with low IGF-I SDS at baseline, a positive change from baseline in IGF-I SDS indicated a better outcome. Data is reported for 'in-trial' observation period. In-trial observation period: from first administration and up until visit 7 or last trial contact, whichever comes first. |
| Change in IGF-I SDS at Week 104 | Baseline (week 0), week 104 | — |
| Change in IGF-I SDS at Week 156 | Baseline (week 0), week 156 | — |
| Change in IGF-I SDS at Week 208 | Baseline (week 0), week 208 | — |
| Change in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) Standard Deviation Score (SDS) at Week 52 | Baseline (week 0), week 52 | Change from baseline (week 0) in IGFBP-3 SDS at week 52 is presented. 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. For participants with low IGFBP-3 SDS at baseline, a positive change from baseline in IGFBP-3 SDS indicated a better outcome. Data is reported for 'in-trial' observation period. In-trial observation period: from first administration and up until visit 7 or last trial contact, whichever comes first. |
| Change in IGFBP-3 SDS at Week 104 | Baseline (week 0), week 104 | — |
| Change in IGFBP-3 SDS at Week 156 | Baseline (week 0), week 156 | — |
| Change in IGFBP-3 SDS at Week 208 | Baseline (week 0), week 208 | — |
Countries
Algeria, Austria, Canada, Denmark, Estonia, France, Germany, Hungary, India, Ireland, Israel, Italy, Japan, Latvia, Norway, Poland, Russia, Serbia, Slovenia, South Korea, Spain, Switzerland, Thailand, Ukraine, United Kingdom, United States
Contacts
STUDY_DIRECTORClinical Transparency (dept. 2834)
Novo Nordisk A/S
Participant flow
Recruitment details
The trial was conducted at 86 sites in 20 countries as follows (number of sites that screened participants/ number of sites that randomized participants): Austria (2/1); Canada (1/1); Denmark (1/0); France (4/4); Germany (3/2); Hungary (1/0); India (3/3); Israel (4/4); Italy (3/2); Japan (17/14); Korea (8/6); Latvia (1/1); Poland (1/1); Russia (8/8); Serbia (5/3); Slovenia (1/1); Spain (2/2); Switzerland (1/1); Thailand (2/2); Ukraine (3/3); United Kingdom (5/5); United States (23/22).
Pre-assignment details
Data reported based on the main part of the trial i.e. up to week 52.
Participants by arm
| Arm | Count |
|---|---|
| Norditropin Participants received 0.034 milligrams/kilogram (mg/kg) norditropin subcutaneously (s.c.; under the skin) by FlexPro pen-injector once daily for 52 weeks (main trial period). Participants completing the main trial period will receive 0.16 mg/kg somapacitan once weekly for 3 years (extension period). | 68 |
| Somapacitan Participants received 0.16 mg/kg somapacitan s.c. once weekly by PDS290 pen-injector for 52 weeks (main trial period). Participants completing the main trial period will receive 0.16 mg/kg somapacitan once weekly for 3 years (extension period). | 132 |
| Total | 200 |
Baseline characteristics
| Characteristic | Norditropin | Somapacitan | Total |
|---|---|---|---|
| Age, Continuous | 6.43 years STANDARD_DEVIATION 2.42 | 6.38 years STANDARD_DEVIATION 2.23 | 6.40 years STANDARD_DEVIATION 2.29 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants | 4 Participants | 5 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 63 Participants | 119 Participants | 182 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 4 Participants | 9 Participants | 13 Participants |
| Race/Ethnicity, Customized Race Asian | 28 Participants | 46 Participants | 74 Participants |
| Race/Ethnicity, Customized Race Black or African American | 1 Participants | 0 Participants | 1 Participants |
| Race/Ethnicity, Customized Race Not reported | 3 Participants | 7 Participants | 10 Participants |
| Race/Ethnicity, Customized Race Other | 0 Participants | 1 Participants | 1 Participants |
| Race/Ethnicity, Customized Race White | 36 Participants | 78 Participants | 114 Participants |
| Sex: Female, Male Female | 18 Participants | 33 Participants | 51 Participants |
| Sex: Female, Male Male | 50 Participants | 99 Participants | 149 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 68 | 0 / 132 |
| other Total, other adverse events | 22 / 68 | 46 / 132 |
| serious Total, serious adverse events | 2 / 68 | 6 / 132 |
Outcome results
Primary
Height Velocity: In-trial Observation Period
Height velocity (HV) was derived from height measurements taken at baseline and Week 52 visit as: HV = (height at 52 weeks visit - height at baseline)/(time from baseline to 52 weeks visit in years). Data is reported for 'in-trial' observation period. In-trial observation period: from first administration and up until visit 7 or last trial contact, whichever comes first.
Time frame: From baseline (week 0) to visit 7 (week 52)
Population: Full analysis set included all randomized participants.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Norditropin | Height Velocity: In-trial Observation Period | 11.8 centimeters per year (cm/year) | Standard Deviation 2.9 |
| Somapacitan | Height Velocity: In-trial Observation Period | 11.2 centimeters per year (cm/year) | Standard Deviation 2.5 |
Comparison: Height velocity at week 52 was analyzed using an analysis of covariance model with treatment, gender, age group, region, growth hormone (GH) peak group and gender by age group by region interaction term as factors, and baseline height as covariate.95% CI: [-1.1, 0.2]ANCOVA
Primary
Height Velocity: On-treatment Observation Period
Height velocity was derived from height measurements taken at baseline and Week 52 visit as: HV = (height at 52 weeks visit - height at baseline)/(time from baseline to 52 weeks visit in years). Data is reported for 'on-treatment' observation period. On-treatment observation period: from first administration and up until last trial contact, visit 7 or 14 days after last administration, whichever comes first.
Time frame: From baseline (week 0) to visit 7 (week 52)
Population: Full analysis set included all randomized participants. Overall Number of Participants Analyzed = participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Norditropin | Height Velocity: On-treatment Observation Period | 11.8 cm/year | Standard Deviation 2.9 |
| Somapacitan | Height Velocity: On-treatment Observation Period | 11.2 cm/year | Standard Deviation 2.5 |
Comparison: Height velocity at 52 weeks was analyzed using a mixed model for repeated measurements, with treatment, gender, age group, region, GH peak group and gender by age group by region interaction terms as factors and baseline height as a covariate, all nested within week as a factor.95% CI: [-1.1, 0.2]Mixed Models Analysis
Secondary
Change in Bone Age
Change from baseline (week -2) in bone age at week 52 is presented. X-ray images of left hand and wrist for bone age assessment according to the Greulich and Pyle atlas were taken. In-trial observation period: from first administration and up until visit 7 or last trial contact, whichever comes first.
Time frame: Baseline (week -2), week 52
Population: Full analysis set included all randomized participants. Overall Number of Participants Analyzed = participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Norditropin | Change in Bone Age | 1.28 Years | Standard Deviation 0.76 |
| Somapacitan | Change in Bone Age | 1.13 Years | Standard Deviation 0.78 |
Secondary
Change in Fasting Plasma Glucose (FPG) at Week 52
Change from baseline (week -2) in FPG at week 52 is presented.
Time frame: Baseline (week -2), week 52
Population: Safety analysis set included all randomized participants exposed to at least one dose of randomized treatment. Overall Number of Participants Analyzed = participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Norditropin | Change in Fasting Plasma Glucose (FPG) at Week 52 | 0.255 Millimoles per liter (mmol/L) | Standard Deviation 0.79 |
| Somapacitan | Change in Fasting Plasma Glucose (FPG) at Week 52 | 0.083 Millimoles per liter (mmol/L) | Standard Deviation 0.567 |
Secondary
Change in FPG at Week 104
Time frame: Baseline (week -2), week 104
Secondary
Change in FPG at Week 156
Time frame: Baseline (week -2), week 156
Secondary
Change in FPG at Week 208
Time frame: Baseline (week -2), week 208
Secondary
Change in Glycated Haemoglobin (HbA1c) at Week 52
Change from baseline (week -2) in HbA1c at week 52 is presented.
Time frame: Baseline (week -2), week 52
Population: Safety analysis set included all randomized participants exposed to at least one dose of randomized treatment. Overall Number of Participants Analyzed = participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Norditropin | Change in Glycated Haemoglobin (HbA1c) at Week 52 | 0.11 Percentage of HbA1c | Standard Deviation 0.26 |
| Somapacitan | Change in Glycated Haemoglobin (HbA1c) at Week 52 | 0.09 Percentage of HbA1c | Standard Deviation 0.22 |
Secondary
Change in HbA1c at Week 104
Time frame: Baseline (week -2), week 104
Secondary
Change in HbA1c at Week 156
Time frame: Baseline (week -2), week 156
Secondary
Change in HbA1c at Week 208
Time frame: Baseline (week -2), week 208
Secondary
Change in Height Standard Deviation Score (HSDS)
Change from baseline (week 0) in HSDS at week 52 is presented. HSDS was derived using Centre for Disease Control and Prevention (CDC) standards. 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. Positive value in change from baseline in HSDS indicated that HSDS was better than baseline HSDS. In-trial observation period: from first administration and up until visit 7 or last trial contact, whichever comes first.
Time frame: Baseline (week 0), week 52
Population: Full analysis set included all randomized participants.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Norditropin | Change in Height Standard Deviation Score (HSDS) | 1.37 Score on a scale | Standard Deviation 0.69 |
| Somapacitan | Change in Height Standard Deviation Score (HSDS) | 1.21 Score on a scale | Standard Deviation 0.54 |
Secondary
Change in Height Velocity Standard Deviation Score (HV SDS)
Change from baseline (week 0) in HV SDS at week 52 is presented. HV SDS was calculated using the formula: HV SDS = (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 HV SDS 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. Positive value in change from baseline in HV SDS indicated that HV SDS was better than baseline HV SDS. In-trial observation period: from first administration and up until visit 7 or last trial contact, whichever comes first.
Time frame: Baseline (week 0), week 52
Population: Full analysis set included all randomized participants.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Norditropin | Change in Height Velocity Standard Deviation Score (HV SDS) | 8.97 Score on a scale | Standard Deviation 4.38 |
| Somapacitan | Change in Height Velocity Standard Deviation Score (HV SDS) | 7.97 Score on a scale | Standard Deviation 3.36 |
Secondary
Change in HOMA-B at Week 104
Time frame: Baseline (week -2), week 104
Secondary
Change in HOMA-B at Week 156
Time frame: Baseline (week -2), week 156
Secondary
Change in HOMA-B at Week 208
Time frame: Baseline (week -2), week 208
Secondary
Change in HOMA-IR at Week 104
Time frame: Baseline (week -2), week 104
Secondary
Change in HOMA-IR at Week 156
Time frame: Baseline (week -2), week 156
Secondary
Change in HOMA-IR at Week 208
Time frame: Baseline (week -2), week 208
Secondary
Change in Homeostatic Model Assessment Insulin Resistance (HOMA-IR) at Week 52
Change from baseline (week -2) in HOMA-IR at week 52 is presented. HOMA-IR is an evaluation of the insulin resistance and was calculated as HOMA-IR = fasting insulin (pmol/L) \* 1/6(µU/mL) \* FPG(mmol/L) / 22.5. Positive change from baseline in HOMA-IR indicated a worse outcome.
Time frame: Baseline (week -2), week 52
Population: Safety analysis set included all randomized participants exposed to at least one dose of randomized treatment. Overall Number of Participants Analyzed = participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Norditropin | Change in Homeostatic Model Assessment Insulin Resistance (HOMA-IR) at Week 52 | 1.03 Percentage of HOMA-IR | Standard Deviation 1.12 |
| Somapacitan | Change in Homeostatic Model Assessment Insulin Resistance (HOMA-IR) at Week 52 | 0.60 Percentage of HOMA-IR | Standard Deviation 1.13 |
Secondary
Change in Homeostatic Model Assessment Steady State Beta Cell Function (HOMA-B) at Week 52
Change from baseline (week -2) in HOMA-B at week 52 is presented. HOMA-B is a measure of the beta cell function and was calculated as follows: HOMA-B = (20 \* fasting insulin (picomoles per liter \[pmol/L\]) \* 1/6(microunit per milliliter \[µU/mL\]))/ FPG(mmol/L)-3.5). Negative change from baseline in HOMA-B indicated a worse outcome.
Time frame: Baseline (week -2), week 52
Population: Safety analysis set included all randomized participants exposed to at least one dose of randomized treatment. Overall Number of Participants Analyzed = participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Norditropin | Change in Homeostatic Model Assessment Steady State Beta Cell Function (HOMA-B) at Week 52 | 69.24 Percentage of HOMA-B | Standard Deviation 81.59 |
| Somapacitan | Change in Homeostatic Model Assessment Steady State Beta Cell Function (HOMA-B) at Week 52 | 37.71 Percentage of HOMA-B | Standard Deviation 59.25 |
Secondary
Change in IGFBP-3 SDS at Week 104
Time frame: Baseline (week 0), week 104
Secondary
Change in IGFBP-3 SDS at Week 156
Time frame: Baseline (week 0), week 156
Secondary
Change in IGFBP-3 SDS at Week 208
Time frame: Baseline (week 0), week 208
Secondary
Change in IGF-I SDS at Week 104
Time frame: Baseline (week 0), week 104
Secondary
Change in IGF-I SDS at Week 156
Time frame: Baseline (week 0), week 156
Secondary
Change in IGF-I SDS at Week 208
Time frame: Baseline (week 0), week 208
Secondary
Change in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) Standard Deviation Score (SDS) at Week 52
Change from baseline (week 0) in IGFBP-3 SDS at week 52 is presented. 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. For participants with low IGFBP-3 SDS at baseline, a positive change from baseline in IGFBP-3 SDS indicated a better outcome. Data is reported for 'in-trial' observation period. In-trial observation period: from first administration and up until visit 7 or last trial contact, whichever comes first.
Time frame: Baseline (week 0), week 52
Population: Full analysis set included all randomized participants. Overall Number of Participants Analyzed = participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Norditropin | Change in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) Standard Deviation Score (SDS) at Week 52 | 1.68 Score on a scale | Standard Deviation 1.02 |
| Somapacitan | Change in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) Standard Deviation Score (SDS) at Week 52 | 1.58 Score on a scale | Standard Deviation 0.92 |
Secondary
Change in Insulin-like Growth Factor I (IGF-I) Standard Deviation Score (SDS) at Week 52
Change from baseline (week 0) in IGF-I SDS at week 52 is presented. 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. For participants with low IGF-I SDS at baseline, a positive change from baseline in IGF-I SDS indicated a better outcome. Data is reported for 'in-trial' observation period. In-trial observation period: from first administration and up until visit 7 or last trial contact, whichever comes first.
Time frame: Baseline (week 0), week 52
Population: Full analysis set included all randomized participants. Overall Number of Participants Analyzed = participants with available data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Norditropin | Change in Insulin-like Growth Factor I (IGF-I) Standard Deviation Score (SDS) at Week 52 | 2.41 Score on a scale | Standard Deviation 1.09 |
| Somapacitan | Change in Insulin-like Growth Factor I (IGF-I) Standard Deviation Score (SDS) at Week 52 | 2.32 Score on a scale | Standard Deviation 1.27 |