Trial Outcomes & Findings for A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9 (NCT NCT05723835)

Participants were screened and assigned to treatment across 14 sites in 5 countries.

A total of 47 participants with short stature either born small for gestational age(SGA), Noonan syndrome(NS), Turner syndrome(TS), or idiopathic short stature(ISS) were either treatment naïve or previously treated with growth hormone(GH) were enrolled \& exposed to once weekly dosing of somapacitan 0.24 milligrams per kilogram per week(mg/kg/week). 26-week main phase is followed by 130-week extension phase I. Study was ongoing at data cut-off date(24Nov2024).

A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9

This outcome measure reported number of AEs in children with short stature for indication SGA. Children with SGA are born small for gestational age with insufficient catch-up growth by 2 years of age or older. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment.

This outcome measure reported number of AEs in participants with short stature for indication TS. TS is a chromosomal disorder which leads to short stature. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment.

This outcome measure reported number of AEs in participants with short stature for indication NS which is a genetically heterogeneous developmental disorder characterized by postnatally reduced growth and other major disorders. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment.

This outcome measure reported number of AEs in participants with short stature for indication ISS. ISS describes short children with normal GH secretion. ISS is a condition in which the height of the individual is more than 2 standard deviations below the corresponding mean height for a given age, sex and population, without evidence of systemic, endocrine, nutritional or chromosomal abnormalities. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment.

This outcome measure reported number of AEs possibly or probably related to somapacitan reported in children with short stature for indication SGA. Children with SGA are born small for gestational age with insufficient catch-up growth by 2 years of age or older. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment.

This outcome measure reported number of AEs possibly or probably related to somapacitan reported in participants with short stature for indication TS. TS is a chromosomal disorder which leads to short stature. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment.

This outcome measure reported number of AEs possibly or probably related to somapacitan reported in participants with short stature for indication NS. An NS is a genetically heterogeneous developmental disorder characterized by postnatally reduced growth and other major disorders. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment.

This outcome measure reported number of AEs possibly or probably related to somapacitan in participants with short stature for indication ISS. ISS describes short children with normal GH secretion and it is a condition in which the height of the individual is more than 2 standard deviations below the corresponding mean height for a given age, sex and population, without evidence of systemic, endocrine, nutritional or chromosomal abnormalities. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment.

This outcome measure reported long-term safety in terms of number of AEs in children with short stature for indication SGA. Children with SGA are born small for gestational age with insufficient catch-up growth by 2 years of age or older. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment.

This outcome measure reported long-term safety in terms of number of AEs in participants with short stature for indication TS. TS is a chromosomal disorder which leads to short stature. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment.

This outcome measure reported long-term safety in terms of number of AEs in participants with short stature for indication NS which is a genetically heterogeneous developmental disorder characterized by postnatally reduced growth and other major disorders. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment.

This outcome measure reported long-term safety in terms of number of AEs in participants with short stature for indication ISS. ISS describes short children with normal GH secretion. ISS is a condition in which the height of the individual is more than 2 standard deviations below the corresponding mean height for a given age, sex and population, without evidence of systemic, endocrine, nutritional or chromosomal abnormalities. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment.

This outcome measure reported height velocity in children with short stature for indication SGA. Height velocity at week 26 was derived as: (height at 26 weeks visit - height at baseline)/ (time from baseline to 26 weeks visit in years).

This outcome measure reported height velocity in children with short stature for indication TS. Height velocity at week 26 was derived as: (height at 26 weeks visit - height at baseline)/ (time from baseline to 26 weeks visit in years).

This outcome measure reported height velocity in children with short stature for indication NS. Height velocity at week 26 was derived as: (height at 26 weeks visit - height at baseline)/(time from baseline to 26 weeks visit in years).

This outcome measure reported height velocity in children with short stature for indication ISS. Height velocity at week 26 was derived as: (height at 26 weeks visit - height at baseline)/ (time from baseline to 26 weeks visit in years).

This outcome measure reported height standard deviation scores in children with short stature for indication SGA. Height SDS at Week 26 was derived as the Height SDS value at baseline (Week 0) subtracted from the Height SDS value at Week 26. Height SDS is derived as: Height SDSi = ({\[Heighti/population median\]\^Skewness}-1)/(Skewness∗population SD); where i indicates the visit and SD indicates the standard deviation (SD). The population median and standard deviation are the ones corresponding to the age at visit i. The population median and standard deviation and skewness are based on reference data. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater height. The positive score indicates that the value is closer to or above the reference population compared to baseline.

This outcome measure reported height standard deviation scores in children with short stature for indication TS. Height SDS at Week 26 was derived as the Height SDS value at baseline (Week 0) subtracted from the Height SDS value at Week 26. Height SDS is derived as: Height SDSi = ({\[Heighti/population median\]\^Skewness}-1)/(Skewness∗population SD); where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The population median and standard deviation and skewness are based on reference data. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater height. The positive score indicates that the value is closer to or above the reference population compared to baseline.

This outcome measure reported height standard deviation scores in children with short stature for indication NS. Height SDS at Week 26 was derived as the Height SDS value at baseline (Week 0) subtracted from the Height SDS value at Week 26. Height SDS is derived as: Height SDSi = ({\[Heighti/population median\]\^Skewness}-1)/(Skewness∗population SD); where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The population median and standard deviation and skewness are based on reference data. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater height. The positive score indicates that the value is closer to or above the reference population compared to baseline.

This outcome measure reported height standard deviation scores in children with short stature for indication ISS. Height SDS at Week 26 was derived as the Height SDS value at baseline (Week 0) subtracted from the Height SDS value at Week 26. Height SDS is derived as: Height SDSi = ({\[Heighti/population median\]\^Skewness}-1)/(Skewness∗population SD); where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The population median and standard deviation and skewness are based on reference data. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater height. The positive score indicates that the value is closer to or above the reference population compared to baseline.

This outcome measure reported change in height velocity SDS in children with short stature for indication SGA. Change in height velocity SDS at week 26 was calculated as the height velocity SDS value at baseline Week 0 subtracted from the height velocity SDS value at Week 26. Height Velocity SDS is derived as: HV SDSi = (HVi - population mean HV)/population SD; where i indicates the visit. The population mean and standard deviation corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater height velocity. The positive score indicated that the value is closer to or above the reference population compared to baseline.

This outcome measure reported change in height velocity SDS in children with short stature for indication TS. Change in height velocity SDS at week 26 was calculated as the height velocity SDS value at baseline (Week 0) subtracted from the height velocity SDS value at Week 26. Height Velocity SDS is derived as: HV SDSi = (HVi - population mean HV)/population SD; where i indicates the visit. The population mean and standard deviation corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater height velocity. The positive score indicated that the value is closer to or above the reference population compared to baseline.

This outcome measure reported change in height velocity SDS in children with short stature for indication NS. Change in height velocity SDS at Week 26 was the Height Velocity SDS value at baseline (Week 0) subtracted from the Height Velocity SDS value at Week 26. Height Velocity SDS is derived as: HV SDSi = (HVi - population mean HV)/population SD; where i indicates the visit. The population mean and standard deviation corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater height velocity. The positive score indicated that the value is closer to or above the reference population compared to baseline.

This outcome measure reported change in height velocity SDS in children with short stature for indication ISS. Change in height velocity SDS at Week 26 was the Height Velocity SDS value at baseline (Week 0) subtracted from the Height Velocity SDS value at Week 26. Height Velocity SDS is derived as: HV SDSi = (HVi - population mean HV)/population SD; where i indicates the visit. The population mean and standard deviation corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater height velocity. The positive score indicated that the value is closer to or above the reference population compared to baseline.

This outcome measure reported change in IGF-1 SDS in children with short stature for indication SGA. Change in IGF-I SDS was derived as IGF-1 SDS value at baseline Week 0 subtracted from the IGF-I SDS value at Week 26. IGF-I SDS is derived as: IGF - I SDSi = ({\[IGF - I i\]/population median}\^Skewness - 1)/ Skewness ∗ population SD; where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater IGF-1. The positive score indicated that the value is closer to or above the reference population compared to baseline.

This outcome measure reported change in IGF-1 SDS in children with short stature for indication TS. Change in IGF-I SDS was derived as IGF-1 SDS value at baseline Week 0 subtracted from the IGF-I SDS value at Week 26. IGF-I SDS is derived as: IGF - I SDSi = ({\[IGF - I i\]/population median}\^Skewness - 1)/ Skewness ∗ population SD; where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater IGF-1. The positive score indicated that the value is closer to or above the reference population compared to baseline.

This outcome measure reported change in IGF-1 SDS in children with short stature for indication NS. Change in IGF-I SDS was derived as IGF-1 SDS value at baseline Week 0 subtracted from the IGF-I SDS value at Week 26. IGF-I SDS is derived as: IGF - I SDSi = ({\[IGF - I i\]/population median}\^Skewness - 1)/ Skewness ∗ population SD; where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater IGF-1. The positive score indicated that the value is closer to or above the reference population compared to baseline.

This outcome measure reported change in IGF-1 SDS in children with short stature for indication ISS. Change in IGF-I SDS was derived as IGF-1 SDS value at baseline Week 0 subtracted from the IGF-I SDS value at Week 26. IGF-I SDS is derived as: IGF - I SDSi = ({\[IGF - I i\]/population median}\^Skewness - 1)/ Skewness ∗ population SD; where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater IGF-1. The positive score indicated that the value is closer to or above the reference population compared to baseline.

This outcome measure reported change in IGFBP-3 scores in children with short stature for indication SGA. Change in IGFBP-3 SCS at Week 26 was derived as the IGFBP-3 SDS value at baseline Week 0 subtracted from IGFBP-3 SDS value at Week 26. IGFBP-3 SDS is derived as: IGFBP - 3 SDSi = ({\[IGFBP - 3 i/population median\]\^Skewness} - 1)/Skewness ∗ population SD; where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater IGFBP-3. The positive score indicated that the value is closer to or above the reference population compared to baseline.

This outcome measure reported change in IGFBP-3 scores in children with short stature for indication TS. Change in IGFBP-3 SCS at Week 26 was derived as the IGFBP-3 SDS value at baseline Week 0 subtracted from IGFBP-3 SDS value at Week 26. IGFBP-3 SDS is derived as: IGFBP - 3 SDSi = ({\[IGFBP - 3 i/population median\]\^Skewness} - 1)/Skewness ∗ population SD; where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater IGFBP-3. The positive score indicated that the value is closer to or above the reference population compared to baseline.

This outcome measure reported change in IGFBP-3 scores in children with short stature for indication NS. Change in IGFBP-3 SCS at Week 26 was derived as the IGFBP-3 SDS value at baseline Week 0 subtracted from IGFBP-3 SDS value at Week 26. IGFBP-3 SDS is derived as: IGFBP - 3 SDSi = ({\[IGFBP - 3 i/population median\]\^Skewness} - 1)/Skewness ∗ population SD; where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater IGFBP-3. The positive score indicated that the value is closer to or above the reference population compared to baseline.

This outcome measure reported change in IGFBP-3 scores in children with short stature for indication ISS. Change in IGFBP-3 SCS at Week 26 was derived as the IGFBP-3 SDS value at baseline Week 0 subtracted from IGFBP-3 SDS value at Week 26. IGFBP-3 SDS is derived as: IGFBP - 3 SDSi = ({\[IGFBP - 3 i/population median\]\^Skewness} - 1)/Skewness ∗ population SD; where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater IGFBP-3. The positive score indicated that the value is closer to or above the reference population compared to baseline.

The steady state pharmacokinetics in terms of Cavg was evaluated for once-weekly somapacitan in children born small for gestational age who were either naïve or non-naïve to GH treatment.

The steady state pharmacokinetics in terms of Cavg was evaluated for once-weekly somapacitan in children with Turner Syndrome who were either naïve or non-naïve to GH treatment.

The steady state pharmacokinetics in terms of Cavg was evaluated for once-weekly somapacitan in children with Noonan Syndrome who were either naïve or non-naïve to GH treatment.

The steady state pharmacokinetics in terms of Cavg was evaluated for once-weekly somapacitan in children with idiopathic short stature who were either naïve or non-naïve to GH treatment.