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

NCT05723835 | Active Not Recruiting Phase 3 Results DMC FDA Drug

• 3 other identifiers: NN8640-4469U1111-1277-97652022-501055-87
• Study Type: interventional
• Target: 47
• Locations: 6 countries
• Sites: 17

Brief Summary

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.

Conditions

SGA; Turner Syndrome; Noonan Syndrome; ISS

Outcome Measures

Primary Outcomes (4)

• 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.

  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.

  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.

  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.

  From baseline (Week 0) to Week 26

Secondary Outcomes (32)

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

  From baseline (Week 0) to Week 26

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

  From baseline (Week 0) to Week 26

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

  From baseline (Week 0) to Week 26

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

  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

  From baseline (Week 0) to Week 156

Study Arms (1)

Somapacitan

EXPERIMENTAL

Participants will receive Somapacitan for 26-week main phase followed by 130-week extension phase.

Drug: Somapacitan

Interventions

Somapacitan DRUG

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

Somapacitan

Eligibility Criteria

• Age: 10 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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:

You may not qualify if:

• 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.

Sponsors & Collaborators

• Novo Nordisk A/S: lead

Study Sites (17)

Univ of AL at Birmingham_BRM

Birmingham, Alabama, 35233, United States

Location

Sutter Valley Med Fdt Ped Endo

Sacramento, California, 95821, United States

Location

Rocky Mt Ped and Endo

Centennial, Colorado, 80112, United States

Location

Childrens National Medical Ctr

Washington D.C., District of Columbia, 20010-2978, United States

Location

Rocky Mt Clin Res, LLC

Idaho Falls, Idaho, 83404-7596, United States

Location

Children's Minnesota

Saint Paul, Minnesota, 55102, United States

Location

University Malaya Medical Centre

Lembah Pantai, Kuala Lumpur, 59100, Malaysia

Location

University Technology MARA (UiTM) - Sg Buloh

Bandar Puncak Alam, Selangor, 42300, Malaysia

Location

Erasmus MC

Rotterdam, 3015 GD, Netherlands

Location

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

Rzeszów, Podkarpackie Voivodeship, 35-301, Poland

Location

UCK, Klinika Pediatrii, Diabetologii i Endokrynologii,

Gdansk, 80-952, Poland

Location

Instytut Centrum Zdrowia Matki Polki

Lodz, 93-338, Poland

Location

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

Rzeszów, 35-301, Poland

Location

SPSK nr 1 im. prof.S.Szyszko w Zabrzu

Zabrze, 41-800, Poland

Location

Asan Medical Center

Seoul, 05505, South Korea

Location

Pusan National University Yangsan Hospital

Yangsan, 50612, South Korea

Location

Hospital Vall d'Hebron

Barcelona, 08035, Spain

Location

MeSH Terms

Conditions

Turner Syndrome; Noonan Syndrome

Interventions

somapacitan

Condition Hierarchy (Ancestors)

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

Results Point of Contact

• Title: Clinical Reporting Office (2834)
• Organization: Novo Nordisk A/S

clinicaltrials@novonordisk.com

(+1) 866-867-7178

Study Officials

• Clinical Transparency (dept. 2834)

  Novo Nordisk A/S

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 29, 2023
• First Posted: February 13, 2023
• Study Start: February 1, 2023
• Primary Completion: June 7, 2024
• Study Completion (Estimated): October 29, 2027
• Last Updated: November 21, 2025
• Results First Posted: November 21, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will share

Locations

PL (5); KR (2); US (6); ES (1); MY (2); NL (1)