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

Running, not enrolling · Phase 3

Conditions studied: SGA, Turner Syndrome, Noonan Syndrome, ISS

In brief

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.

Key facts

Study ID

NCT05723835

Run by

Novo Nordisk A/S

People needed

47

Starts

2023-02-01

Expected to finish

2027-10-29

Last updated by the study team

2026-07-09

Who can join

Age: 10 and older, up to 18. Sex: any. Healthy volunteers: not accepted.

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:
• 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.

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.
• 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.

Where it is running

• 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
• UCK, Klinika Pediatrii, Diabetologii i Endokrynologii, — Gdansk, Poland
• Instytut Centrum Zdrowia Matki Polki — Lodz, Poland
• Kliniczny Szpital Wojewodzki nr 2 im. Sw. Jadwigi Krolowej w Rzeszowie — Rzeszów, Poland
• SPSK nr 1 im. prof.S.Szyszko w Zabrzu — Zabrze, Poland
• Pusan National University Yangsan Hospital — Yangsan, Gyeongsangnam-do, South Korea
• Asan Medical Center — Seoul, South Korea
• Pusan National University Yangsan Hospital — Yangsan, South Korea
• Hospital Vall d'Hebron — Barcelona, Spain

Full record on ClinicalTrials.gov

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