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

Phase 3Active Not RecruitingNCT05723835

Novo Nordisk A/S47 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

SGA Turner Syndrome Noonan Syndrome ISS

Interventions

SomapacitanDRUG

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

Eligibility

Sex: ALLMin age: 10 YearsMax age: 18 Years

Medical Language ↔ Plain English

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.

Locations (17)

Univ of AL at Birmingham_BRM

Birmingham, Alabama, United States

Sutter Valley Med Fdt Ped Endo

Sacramento, California, United States

Rocky Mt Ped and Endo

Centennial, Colorado, United States

Childrens National Medical Ctr

Washington D.C., District of Columbia, United States

Rocky Mt Clin Res, LLC

Idaho Falls, Idaho, United States

Children's Minnesota

Saint Paul, Minnesota, United States

University Malaya Medical Centre

Lembah Pantai, Kuala Lumpur, Malaysia

University Technology MARA (UiTM) - Sg Buloh

Bandar Puncak Alam, Selangor, Malaysia

Erasmus MC

Rotterdam, Netherlands

Kliniczny Szpital Wojewodzki nr 2 im. Sw. Jadwigi Krolowej w Rzeszowie

Rzeszów, Podkarpackie Voivodeship, Poland

...and 7 more locations

Outcomes

Primary Outcomes

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

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

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

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

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

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

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

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

Secondary Outcomes

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

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.

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

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

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.

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

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

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.

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

Data from ClinicalTrials.gov

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

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.

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

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

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

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

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

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

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

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

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

Height Velocity Reported Children Born Small for Gestational Age

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).

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

Height Velocity Reported for Turner Syndrome

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).

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

Height Velocity Reported for Noonan Syndrome

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).

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

Height Velocity Reported for Idiopathic Short Stature

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).

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

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

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.