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NCT Number: NCT05723835

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

The purpose of this study is to find out if somapacitan is safe and how well somapacitan works in children either born small for gestational age or with Turner syndrome, Noonan syndrome or idiopathic short stature. Somapacitan is a new growth hormone medicine for treatment of low level of growth hormone. The study will last for about 3 years. During the study, the participants will be treated with somapacitan once a week. Somapacitan can be injected anytime during the day. The study doctor or nurse will show how to inject somapacitan, so that the participant knows how to do it at home.

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Key information

Conditions

SGA Cardiovascular Abnormalities Cardiovascular Diseases Chromosome Disorders Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Craniofacial Abnormalities Disorders of Sex Development Endocrine System Diseases Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Gonadal Disorders Gonadal Dysgenesis Heart Defects, Congenital Heart Diseases ISS Male Urogenital Diseases Musculoskeletal Abnormalities Musculoskeletal Diseases Noonan Syndrome Sex Chromosome Disorders Sex Chromosome Disorders of Sex Development Skin and Connective Tissue Diseases Turner Syndrome Urogenital Abnormalities Urogenital Diseases

Age range

10 year–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

University Malaya Medical Centre, Lembah Pantai, Kuala Lumpur, Malaysia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Applicable to children with SGA:

  • Born small for gestational age (birth length below -2 SDS OR birth weight below -2 SDS OR both) (according to national standards).
  • Age:
  • Male participants: Age equal to or above 11.0 years and below 18.0 years at screening.
  • Female participants: Age equal to or above 10.0 years and below 18.0 years at screening.
  • Open epiphyses; defined as bone age less than (<) 14 years for females and bone age < 16 years for males.
  • For Growth Hormone (GH) treatment naïve participants: Impaired height defined as at least 2.5 standard deviations below the mean height for chronological age and sex at screening according to the standards of Centers for Disease Control and Prevention.

Applicable to children with TS:

  • Diagnosis of TS according to local clinical practice.
  • Age:
  • Female participants: Age equal to or above 10.0 years and below 18.0 years at screening.
  • Open epiphyses; defined as bone age < 14 years for females and bone age < 16 years for males.
  • For GH treatment naïve participants: Impaired height defined as at least 2.0 standard deviation below the mean height for chronological age and sex at screening according to the standards of Centers for Disease Control and Prevention.
  • For GH treatment naïve participants: Confirmed diagnosis of TS by 30-cell (or more) lymphocyte chromosomal analysis or confirmation of TS and TS mosaicism using comparative genomic hybridization (CGH)-array.

Applicable to children with NS:

  • Diagnosis of NS according to local clinical practice.
  • Age:
  • Male participants: Age equal to or above 11.0 years and below 18.0 years at screening.
  • Female participants: Age equal to or above 10.0 years and below 18.0 years at screening.
  • Open epiphyses; defined as bone age < 14 years for females and bone age < 16 years for males.
  • For GH treatment naïve participants: Clinical diagnosis of NS according to van der Burgt score list and genetic test result or confirmed mutation in any of the genes associated with NS before allocation.

Applicable to children with ISS:

  • Age:
  • Male participants: Age equal to or above 11.0 years and below 18.0 years at screening.
  • Female participants: Age equal to or above 10.0 years and below 18.0 years at screening.
  • Open epiphyses; defined as bone age < 14 years for females and bone age < 16 years for males.
  • For GH treatment naïve participants: Impaired height defined as at least 2.5 standard deviations below the mean height for chronological age and sex at screening
  • For GH treatment naïve participants: Normal GH secretion (GH peak above 7 ng/mL) during GH stimulation test performed within 18 months prior to screening.
  • For GH treatment naïve participants: Bone age not delayed more than 2 years compared to chronological age at screening.

Exclusion criteria

  • Children with suspected or confirmed growth hormone deficiency according to local practice.
  • Children diagnosed with diabetes mellitus or screening values from the central laboratory.
  • Fasting plasma glucose above or equal to 126 milligrams per deciliter (mg/dL) [7.0 millimoles per litre (mmol/L)] or
  • Glycated hemoglobin (HbA1c) above or equal to 6.5%.
  • 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 greater than 400 micrograms per day (µg/day) of inhaled budesonide or equivalent (i.e., 250 µg/day for fluticasone propionate) for longer than 4 consecutive weeks within the last 12 months prior to screening.
  • History or known presence of malignancy including intracranial tumours.

Applicable to children with SGA:

  • Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with height, such as, but not limited to:
  • Poorly controlled or uncontrolled hormonal deficiencies.
  • Known chromosomal aneuploidy or significant gene mutations causing medical 'syndromes' with short stature, including but not limited to Laron syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, skeletal dysplasias, abnormal short stature homeobox (SHOX) gene analysis or absence of GH receptors.

Applicable to children with TS:

  • Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with height, such as, but not limited to:
  • Known family history of skeletal dysplasia.
  • Significant spinal abnormalities including but not limited to scoliosis, kyphosis and spina bifida variants.
  • Any other disorder that can cause short stature such as, but not limited to nutritional disorders, chronic systemic illness and chronic renal disease.
  • Mosaicism below 10%.
  • TS with Y-chromosome mosaicism where gonadectomy has not been performed.
  • New York Heart Association (NYHA) class II or above or requiring medication for any heart condition.

Applicable to children with NS:

  • Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with height, such as, but not limited to:
  • Known family history of skeletal dysplasia.
  • Significant spinal abnormalities including but not limited to scoliosis, kyphosis and spina bifida variants.
  • Any other disorder that can cause short stature such as, but not limited to nutritional disorders, chronic systemic illness and chronic renal disease.
  • Noonan-related disorders including but not limited to: Noonan syndrome with multiple lentigines (formerly called 'LEOPARD' syndrome), Noonan syndrome with loose anagen hair, cardiofaciocutaneous syndrome (CFC), Costello syndrome, neurofibromatosis type 1 (NF1) and Legius syndrome.

Applicable to children with ISS:

  • Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with height, such as, but not limited to:
  • Known family history of skeletal dysplasia.
  • Significant spinal abnormalities including but not limited to scoliosis, kyphosis and spina bifida variants.
  • Any other disorder that can cause short stature such as, but not limited to nutritional disorders, chronic systemic illness and chronic renal disease.
  • Poorly controlled or uncontrolled hormonal deficiencies.
  • Known chromosomal aneuploidy or significant gene mutations causing medical 'syndromes' with short stature, including but not limited to Laron syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, skeletal dysplasias, abnormal SHOX gene analysis or absence of GH receptors.

Treatment and study plan

Somapacitan

Drug

Somapacitan 0.24 milligrams per kilograms per week (mg/kg/week) will be administered subcutaneously (s.c.) using PDS290 pen-injector.

Primary outcomes

  1. Number of Adverse Events (AEs) Reported in Children Born Small for Gestational Age- Weeks 0 to 26

    Time frame: From baseline (Week 0) to Week 26

    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.

  2. Number of Adverse Events Reported for Turner Syndrome (TS)- Weeks 0 to 26

    Time frame: From baseline (Week 0) to Week 26

    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.

  3. Number of Adverse Events Reported for Noonan Syndrome- Weeks 0 to 26

    Time frame: From baseline (Week 0) to Week 26

    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.

  4. Number of Adverse Events Reported for Idiopathic Short Stature (ISS)- Weeks 0 to 26

    Time frame: From baseline (Week 0) to Week 26

    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.

Secondary outcomes

  1. Number of Adverse Events Possibly or Probably Related to Somapacitan Reported for Children Born Small for Gestational Age

    Time frame: From baseline (Week 0) to Week 26

    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.

  2. Number of Adverse Events Possibly or Probably Related to Somapacitan Reported for Turner Syndrome

    Time frame: From baseline (Week 0) to Week 26

    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.

  3. Number of Adverse Events Possibly or Probably Related to Somapacitan Reported for Noonan Syndrome

    Time frame: From baseline (Week 0) to Week 26

    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.

  4. Number of Adverse Events Possibly or Probably Related to Somapacitan Reported for Idiopathic Short Stature

    Time frame: From baseline (Week 0) to Week 26

    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.

  5. Number of Adverse Events Reported Long-term Safety for Children Born Small for Gestational Age- Weeks 0 to 156

    Time frame: From baseline (Week 0) to Week 156

    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.

  6. Number of Adverse Events Reported Long-term Safety for Turner Syndrome- Weeks 0 to 156

    Time frame: From baseline (Week 0) to Week 156

    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.

  7. Number of Adverse Events Reported Long-term Safety for Noonan Syndrome- Weeks 0 to 156

    Time frame: From baseline (Week 0) to Week 156

    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.

  8. Number of Adverse Events Reported Long-term Safety for Idiopathic Short Stature- Weeks 0 to 156

    Time frame: From baseline (Week 0) to Week 156

    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.

  9. Height Velocity Reported Children Born Small for Gestational Age

    Time frame: From Baseline (Week 0) to Week 26

    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).

  10. Height Velocity Reported for Turner Syndrome

    Time frame: From Baseline (Week 0) to Week 26

    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).

  11. Height Velocity Reported for Noonan Syndrome

    Time frame: From Baseline (Week 0) to Week 26

    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).

  12. Height Velocity Reported for Idiopathic Short Stature

    Time frame: From Baseline (Week 0) to Week 26

    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).

  13. Change in Height Standard Deviation Scores (SDS) Reported for Children Born Small for Gestational Age

    Time frame: Baseline (Week 0), Week 26

    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.

  14. Change in Height Standard Deviation Scores Reported for Turner Syndrome

    Time frame: Baseline (Week 0), Week 26

    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.

  15. Change in Height Standard Deviation Scores Reported for Noonan Syndrome

    Time frame: Baseline (Week 0), Week 26

    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.

  16. Change in Height Standard Deviation Scores Reported for Idiopathic Short Stature

    Time frame: Baseline (Week 0), Week 26

    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.

  17. Change in Height Velocity Standard Deviation Scores Reported Separately for Children Born Small for Gestational Age

    Time frame: Baseline (Week 0), Week 26

    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.

  18. Change in Height Velocity Standard Deviation Scores Reported Separately for Turner Syndrome

    Time frame: Baseline (Week 0), Week 26

    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.

  19. Change in Height Velocity Standard Deviation Scores Reported for Noonan Syndrome

    Time frame: Baseline (Week 0), Week 26

    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.

  20. Change in Height Velocity Standard Deviation Scores Reported Separately for Idiopathic Short Stature

    Time frame: Baseline (Week 0), Week 26

    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.

  21. Change in Insulin-like Growth Factor 1 (IGF-1) Standard Deviation Score Reported for Children Born Small for Gestational Age

    Time frame: Baseline (Week 0), Week 26

    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.

  22. Change in Insulin-like Growth Factor 1 Standard Deviation Score Reported for Turner Syndrome

    Time frame: Baseline (Week 0), Week 26

    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.

  23. Change in Insulin-like Growth Factor 1 Standard Deviation Score Reported for Noonan Syndrome

    Time frame: Baseline (Week 0), Week 26

    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.

  24. Change in Insulin-like Growth Factor 1 Standard Deviation Score Reported Separately for Idiopathic Short Stature

    Time frame: Baseline (Week 0), Week 26

    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.

  25. Change in Insulin-like Growth Factor Binding Protein-3 (IGFBP-3) SDS Reported for Children Born Small for Gestational Age

    Time frame: Baseline (week 0), Week 26

    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.

  26. Change in Insulin-like Growth Factor Binding Protein-3 SDS Reported for Turner Syndrome

    Time frame: Baseline (Week 0), Week 26

    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.

  27. Change in Insulin-like Growth Factor Binding Protein-3 SDS Reported for Noonan Syndrome

    Time frame: Baseline (Week 0), Week 26

    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.

  28. Change in Insulin-like Growth Factor Binding Protein-3 SDS Reported for Idiopathic Short Stature

    Time frame: Baseline (Week 0), Week 26

    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.

  29. Weekly Average Somapacitan Concentration (Cavg) Reported for Children Born Small for Gestational Age

    Time frame: Weeks 4, 8, 13, 20 and 26

    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.

  30. Weekly Average Somapacitan Concentration (Cavg) Reported for Turner Syndrome

    Time frame: Weeks 4, 8, 13, 20 and 26

    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.

  31. Weekly Average Somapacitan Concentration (Cavg) Reported for Noonan Syndrome

    Time frame: Weeks 4, 8, 13, 20 and 26

    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.

  32. Weekly Average Somapacitan Concentration (Cavg) Reported for Idiopathic Short Stature

    Time frame: Weeks 4, 8, 13, 20 and 26

    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.

Sponsors and collaborators

Lead sponsor

Novo Nordisk A/S

Industry

Registry information

Official study title

A Study Evaluating the Safety and Efficacy of Once-weekly Dosing of Somapacitan in a Basket Study Design in Paediatric Participants With Short Stature Either Born Small for Gestational Age or With Turner Syndrome, Noonan Syndrome or Idiopathic Short Stature

Acronym: REAL 9

Important dates

Study start
2023
Primary completion
2024
Study completion
2027
First posted
Feb 13, 2023
Registry last updated
Jul 9, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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