A Research Study in Children Born Small and Who Stayed Small. Treatment is Somapacitan Once a Week Compared to Norditropin® Once a Day

A Dose-finding Trial Evaluating the Effect and Safety of Once-weekly Treatment of Somapacitan Compared to Daily Norditropin® in Children With Short Stature Born Small for Gestational Age With no Catch-up Growth by 2 Years of Age or Older

The study compares 2 medicines used for the treatment of children who are born small and who stayed small: somapacitan given once a week (a new medicine) and Norditropin® given once a day (the medicine doctors can already prescribe).

Participants will either get somapacitan or Norditropin® - which treatment is decided by chance. Both participants and the study doctor will know which treatment the participants get. The study will last for 5 years. Participants will take either an injection once every week or once every day.

Study Overview

• Status: Active, not recruiting
• Conditions: Short Stature Children Born Small for Gestational Age (SGA)
• Intervention / Treatment: Drug: Somapacitan; Drug: Norditropin®

• Study Type: Interventional
• Enrollment (Actual): 62
• Phase: Phase 2

Contacts and Locations

Study Locations

• Algeria
  • Algiers, Algeria, 16000
    • CHU Bab El Oued Pediatrics Dept
  • Algiers, Algeria, 16000
    • Endo and Diab Dept El Oued
  • Constantine, Algeria, 25000
    • endocrino-diabetology department, Hospital IBN BADIS.
• Austria
  • Graz, Austria, 8036
    • Med. Univ. Graz -Klinische Abteilung f. Allgemeine Pädiatrie
  • Salzburg, Austria, A 5020
    • LKH Salzburg- Univ. Klinik f. Kinder- und Jugendheilkunde
  • Sankt Pölten, Austria, 3100
    • LKH St. Poelten, Kinder-und Jugendheilkunde
  • Vöcklabruck, Austria, 4840
    • Salzkammergut-Klinikum Vöcklabruck
  • Upper Austria
    • Linz, Upper Austria, Austria, 4020
      • Kepler Universitätsklinikum GmbH - Med Campus IV (vorm.LFKK)
• Canada
  • Alberta
    • Edmonton, Alberta, Canada, T6G 2B7
      • Stollery Children's Hospital
• Denmark
  • Copenhagen Ø, Denmark, 2100
    • Rigshospitalet Klinik for Vækst og Reproduktion Afsnit 5064
• Estonia
  • Tallinn, Estonia, 13419
    • Tallinn Children's Hospital
  • Tartu, Estonia, 50406
    • Tartu University Hospital Children's Clinic
• France
  • Angers, France, 49100
    • Centre Hospitalier Universitaire D'Angers-2
  • Le Kremlin-Bicêtre, France, 94270
    • Ap-Hp-Hopital de Bicetre-2
  • Marseille, France, 13005
    • Assistance Publique Hopitaux de Marseille-Hopital de La Timone-2
  • Marseille Cédex 05, France, 13385
    • Hopital de la Timone
  • Paris, France, 75015
    • Hôpital Necker
  • Paris, France, 75015
    • Ap-Hp-Hopital Necker-2
  • Toulouse, France, 31059
    • Hopital Des Enfants-2
• Hungary
  • Budapest, Hungary, 1023
    • Észak-Közép-budai Centrum, Szent János Kórház és Szakrendelő
  • Szeged, Hungary, 6720
    • Szegedi Tudományegyetem Gyermekgyógyászati Klinika
• India
  • Kerala
    • Kochi, Kerala, India, 682041
      • Amrita Institute Of Medical Sciences & Research Centre
  • Maharashtra
    • Pune, Maharashtra, India, 411001
      • Jehangir Clinical Development Centre
  • New Delhi
    • New Dehli, New Delhi, India, 110029
      • All India Institute of Medical Sciences
• Ireland
  • Dublin, Ireland, D12 N512
    • CHI Crumlin Dept of Endocrinology
• Israel
  • Haifa, Israel, 31096
    • Rambam MC - Department of Pediatrics A
  • Kfar Saba, Israel, 44281
    • Meir MC - Department of Paediatrics
  • Petah Tikva, Israel, 49202
    • Schneider MC - Endrocrinology and Diabetes
• Italy
  • Florence, Italy, 50139
    • IRCCS Meyer Firenze
  • Genova, Italy, 16147
    • Ospedale Pediatrico Gaslini
  • Milan, Italy, 20122
    • Ospedale Maggiore Policlinico UO Endocrinologia Diabetolgia
  • Roma, Italy, 00165
    • Ospedale Pediatrico Bambino Gesu
• Japan
  • Fukuoka, Japan, 830-0011
    • Kurume University Hospital, Pediatrics
  • Fukuoka-shi, Fukuoka, Japan, 813-0017
    • Fukuoka Children's Hospital
  • Fukuoka-shi, Fukuoka, Japan, 813-0017
    • Fukuoka Children's Hospital_Endocrinology and Metabolism
  • Fukuyama-shi, Hiroshima, Japan, 721-8511
    • Fukuyama City Hospital_Department of Pediatrics
  • Kobe-shi, Hyogo, Japan, 650-0047
    • Hyogo Prefectual Kobe Children's Hospital Dept. Metab & endo
  • Kyoto, Japan, 602-8566
    • Univ.HP, Kyoto Pref Univ of Medicine, Dept. of Pediatrics
  • Nara-shi, Nara, Japan, 630-8581
    • Nara Prefecture General Medical Center_ Nara-shi, Nara
  • Niigata-shi, Niigata, Japan, 951 8520
    • Niigata University Medical & Dental Hospital_Pediatrics
  • Osaka, Japan, 534-0021
    • Osaka City General Hospital, Pediatric Endocrinology and Me
  • Osaka, Japan, 594-1101
    • Osaka Women's and Children's Hospital
  • Saitama-shi, Saitama, Japan, 330-8777
    • Saitama Children's Medical Center, Endocrinorogy&Metabolism
  • Tokyo, Japan, 157 8535
    • National Center for Child Health and Dev, Endo and Metabo
  • Tokyo, Japan, 113-8519
    • Institute of Science Tokyo Hospital
  • Tokyo, Japan, 113-8519
    • Institute of Science Tokyo Hospital_Pediatrics
  • Tokyo, Japan, 160-8582
    • Keio University Hospital, Pediatrics
• Latvia
  • Riga, Latvia, 1004
    • Children's Clinical University Hospital
• Norway
  • Bergen, Norway, 5021
    • Haukeland Universitetssykehus, Barneklinikken
• Poland
  • Szczecin, Poland, 71-252
    • SPSK nr 1 im Prof. T Sokolowskiego
  • Warsaw, Poland, 04-730
    • Instytut ''Pomnik - Centrum Zdrowia Dziecka''
• Russia
  • Izhevsk, Russia, 426009
    • Republic Children's Hospital of Ministry of Health of Udmurt
  • Moscow, Russia, 119435
    • Setchenov First Moscow State Medical University
  • Moscow, Russia, 125373
    • RMAPE
  • Novosibirsk, Russia, 630048
    • Children's clinical city hospital #1
  • Rostov-on-Don, Russia, 344013
    • FSBEI of Higher Education "Rostov State Medical University"
  • Saint Petersburg, Russia, 191144
    • SPSBHI City Children out-patient clinic #44
  • Samara, Russia, 443079
    • Samara Regional Children Clinical Hospital n.a. N.N. Ivanova
  • Tomsk, Russia, 634050
    • Siberian State Medical University
• Serbia
  • Belgrade, Serbia, 11000
    • University Children's Hospital Tirsova
  • Belgrade, Serbia, 11070
    • Institute for Mother and Child Health Care of Serbia
  • Kragujevac, Serbia, 34000
    • University Clinical Centre Kragujevac
  • Niš, Serbia, 18 000
    • Clinical Center in Nis
  • Novi Sad, Serbia, 21000
    • Institute for Health Care of Children and Adolescents
• Spain
  • Esplugues Llobregat(Barcelona), Spain, 08950
    • Hospital Sant Joan de Déu
  • Santiago de Compostela, Spain, 15706
    • Hospital Clínico de Santiago de Compostela
• Switzerland
  • Bern, Switzerland, 3010
    • Med. Kinder- und Poliklinik
  • Zurich, Switzerland, 8032
    • Kinderspital Endokrinologie, Zürich
• Thailand
  • Bangkok, Thailand, 10330
    • King Chulalongkorn Memorial hospital-Ped-Endocrinology
  • Bangkok, Thailand, 10330
    • King Chulalongkorn Memorial hospital_Ped-Endocrinology
  • Bangkok, Thailand, 10400
    • Phramongkutklao Hospital_Ped-Endo_Pediatrics
• Ukraine
  • Dnipro, Ukraine, 49100
    • Dnipropetrovsk Regional Children's Clinical Hospital - Endocrinology department
  • Kharkiv, Ukraine, 61093
    • Kharkiv Regional Children's Clinical Hospital - Endocrinological department
  • Kyiv, Ukraine, 04114
    • Komisarenko Institute of Endocrinology and Metabolism of NAMSU - Department of paediatric endocrine pathology
• United Kingdom
  • Hull, United Kingdom, HU3 2JZ
    • Hull Royal Infirmary_Hull
  • Liverpool, United Kingdom, L12 2AP
    • Alder Hey Children's Hospital
  • London, United Kingdom, E1 1BB
    • Royal London Hospital_London
  • Manchester, United Kingdom, M13 9WL
    • Royal Manchester Children's Hospital
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
      • Univ of AL at Birmingham_BRM
  • California
    • Orange, California, United States, 92868
      • Children's Hosp Of Orange
  • Idaho
    • Boise, Idaho, United States, 83712
      • St. Luke's Children's Endo
  • Minnesota
    • Minneapolis, Minnesota, United States, 55454
      • Univ of Minnesota M.C.H.
    • Saint Paul, Minnesota, United States, 55102
      • Children's Minnesota
  • New Jersey
    • Morristown, New Jersey, United States, 07962
      • Goryeb Children's Hospital
    • New Brunswick, New Jersey, United States, 08901
      • Rutgers-RWJMS
  • New York
    • Garden City, New York, United States, 11530
      • NYU Langone Hospital-LI
    • Mineola, New York, United States, 11501
      • NYU Langone Hospital-LI
    • New York, New York, United States, 10029
      • Icahn Sch of Med-Mt Sinai Hosp
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • CCHMC_Cinc
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73104
      • Univ Oklahoma Sci Ctr OK City
  • Texas
    • Fort Worth, Texas, United States, 76104
      • Cook Children's Hospital-Hematology-Oncology
  • Washington
    • Tacoma, Washington, United States, 98405
      • MultiCare Inst for Res & Innov

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 11 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Pre-pubertal children, boys:

  1. age between 2.5 and 11.0 years at screening.
  2. testes volume below 4 ml.

• Pre-pubertal children, girls:

  1. age between 2.5 and 10.0 years at screening.
  2. Tanner stage 1 for breast development (no palpable glandular breast tissue).
• Born small for gestational age (birth length and/or weight below -2 standard deviation scores) (according to national standards).
• Impaired height defined as at least 2.5 standard deviations below the mean height for chronological age and gender at screening according to the standards of Centers for Disease Control and Prevention at screening.
• Impaired height velocity defined as annualized height velocity below the 50th percentile for chronological age and gender according to the standards of Prader calculated over a time span of minimum 6 months and maximum 18 months prior to screening.
• No prior exposure to growth hormone therapy or Insulin-like Growth Factor-I (IGF-I) treatment.

Exclusion Criteria:

• Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with standing height measurements.
• Children with hormonal deficiencies including suspected or confirmed growth hormone deficiency according to local practise.
• 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 of greater than 400 μg/day of inhaled budesonide or equivalents for longer than 4 consecutive weeks within the last 12 months prior to screening.
• Concomitant administration of other treatments that may have an effect on growth, e.g but not limited to methylphenidate for treatment of attention deficit hyperactivity disorder.
• Diagnosis of attention deficit hyperactivity disorder.
• Prior history or presence of malignancy including intracranial tumours.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

5

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Somapacitan 0.24 mg/kg/week; Same treatment in main period (26 weeks) and extension period I (26 weeks). Dosage of Somapacitan during extension period II will be determined based on the data from main phase.	Drug: Somapacitan; Somapacitan injected under the skin once a week.
Experimental: Somapacitan 0.20 mg/kg/week; Same treatment in main period (26 weeks) and extension period I (26 weeks). Dosage of Somapacitan during extension period II will be determined based on the data from main phase.	Drug: Somapacitan; Somapacitan injected under the skin once a week.
Experimental: Somapacitan 0.16 mg/kg/week; Same treatment in main period (26 weeks) and extension period I (26 weeks). Dosage of Somapacitan during extension period II will be determined based on the data from main phase.	Drug: Somapacitan; Somapacitan injected under the skin once a week.
Active Comparator: Norditropin® 0.035 mg/kg/day; Same treatment in main period (26 weeks) and extension period I (26 weeks). Participants will switch to Somapacitan during extension period II. Dosage of Somapacitan during extension period II will be determined based on the data from main phase.	Drug: Norditropin®; Norditropin® injected under the skin once a day.
Active Comparator: Norditropin® 0.067 mg/kg/day; Same treatment in main period (26 weeks) and extension period I (26 weeks). Participants will switch to Somapacitan during extension period II. Dosage of Somapacitan during extension period II will be determined based on the data from main phase.	Drug: Norditropin®; Norditropin® injected under the skin once a day.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Height Velocity	Height velocity (HV) was derived from height measurements taken at baseline (week 0) and week 26 visit as: HV = (height at 26 weeks visit - height at baseline)/(time from baseline to 26 weeks visit in years). The outcome measure was evaluated based on the data from in-trial observation period. In-trial observation period: from first administration and up until week 26 or last trial contact, whichever came first.	Baseline (week 0); week 26

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Ratio of Bone Age Versus Chronological Age	Change in ratio of bone age (years) versus chronological age (years) from baseline (week 0) to week 52 is presented. X-rays of left hand and wrist for bone age assessment according to the Greulich and Pyle atlas were taken. X-ray images were sent to a central imaging laboratory for evaluation. Chronological Age (years) was calculated as: (Date minus Date of Birth) divided by 365.25. The outcome measure was evaluated based on the data from in-trial observation period. In-trial observation period: from first administration and up until week 52 or last trial contact, whichever came first.	Baseline (week 0); week 52
Change in Height Standard Deviation Score (HSDS)	Change in HSDS from baseline (week 0) to week 26 is presented. HSDS was derived using Centre for Disease Control and Prevention (CDC) standards. HSDS = [(height/population median)^skewness - 1]/(skewness * population standard deviation) where skewness, median and standard deviation is given by a reference growth table for the corresponding chronological age. The range for HSDS was -10 to +10. Negative scores indicated a height below the mean height for a child with the same age and gender, whereas positive scores indicated a height above the mean height for a child with the same age and gender. Positive value in change from baseline in HSDS indicated that HSDS was better than baseline HSDS In-trial observation period: from first administration and up until week 26 or last trial contact, whichever came first.	Baseline (week 0); week 26
Change in Height Velocity Standard Deviation Score (HVSDS)	Change in HVSDS from baseline (week 0) to week 26 is presented. HVSDS was derived using Prader standards. HV SDS was calculated using the formula: HV SDS = (height velocity - mean)/standard deviation (SD), where height velocity was the height velocity variable measured, mean and SD of height velocity by gender and age for the reference population. The range for HVSDS was -10 to +10. Negative scores indicated a height velocity below the mean height velocity for a child with the same age and gender, whereas positive scores indicated a height velocity above the mean height velocity for a child with the same age and gender. Positive value in change from baseline in HVSDS indicated that HVSDS was better than baseline HVSDS. The outcome measure was evaluated based on the data from in-trial observation period. In-trial observation period: from first administration and up until week 26 or last trial contact, whichever came first.	Baseline (week 0); week 26
Change in Fasting Plasma Glucose	Change in fasting plasma glucose from baseline (week 0) to week 26 is presented. The outcome measure was evaluated based on the data from on-treatment observation period. On-treatment observation period: from first administration and up until last trial contact, week 26 or 14 days after last administration, whichever came first.	Baseline (week 0); week 26
Change in Homeostatic Model Assessment for Steady State Beta Cell Function (HOMA-B)	Change in HOMA-B from baseline (week 0) to week 26 is presented. HOMA-B is a measure of the beta cell function and was calculated as follows: HOMA-B = (20 * fasting insulin (picomoles per liter [pmol/L]) * 1/6(microunit per milliliter [µU/mL]))/ FPG(mmol/L)-3.5). HOMA-beta score ranges from minus infinity to infinity (no limits). Higher score means better beta-cell function for HOMA-beta. Negative change from baseline (week 0) in HOMA-B indicated a worse outcome. The outcome measure was evaluated based on the data from on-treatment observation period. On-treatment observation period: from first administration and up until last trial contact, week 26 or 14 days after last administration, whichever came first.	Baseline (week 0); week 26
Change in Homeostatic Model Assessment for Insulin Resistance (HOMA-IR)	Change in HOMA-IR from baseline (week 0) to week 26 is presented. Insulin resistance is a condition in which cells fail to respond to normal actions of hormone in body. HOMA-IR is calculated using a participant's fasting plasma insulin and glucose levels. HOMA-IR = fasting serum insulin (micro international units per milliliter (μU/ml)) × fasting plasma glucose (millimoles per liter (mmol/l)) / 22.5. HOMA-IR scores are classified as follows: less than 1.0: considered insulin sensitive, 0.5-1.4: considered healthy, above 1.8: considered early insulin resistance; above 2.7 is considered significant insulin resistance. HOMA-IR score ranges from 0-infinity (no upper limit). Higher the score, higher the level of insulin resistance. The outcome measure was evaluated based on the data from on-treatment observation period. On-treatment observation period: from first administration and up until last trial contact, week 26 or 14 days after last administration, whichever came first.	Baseline (week 0); week 26
Change in Glycated Haemoglobin (HbA1c)	Change in HbA1c from baseline (week 0) to week 26 is presented. The outcome measure was evaluated based on the data from on-treatment observation period. On-treatment observation period: from first administration and up until last trial contact, week 26 or 14 days after last administration, whichever came first.	Baseline (week 0); week 26
Change in Insulin-like Growth Factor I (IGF-I) Standard Deviation Score (SDS)	Change in IGF-I SDS from baseline (week 0) to week 26 is presented. IGF-I SDS was provided by the central laboratory; its calculation is based on the actual value of IGF-1 minus mean reference value of IGF-1 divided by reference standard deviation of IGF-1. The range for IGF-I SDS was from -10 to +10. Negative scores indicated a IGF-I below the mean IGF-I for a child with the same age and gender, whereas positive scores indicated a IGF-I above the mean IGF-I for a child with the same age and gender. Positive value in change from baseline in IGF-I SDS indicated that IGF-I SDS was higher than baseline IGF-I SDS. The outcome measure was evaluated based on the data from on-treatment observation period. On-treatment observation period: from first administration and up until last trial contact, week 26 or 14 days after last administration, whichever came first.	Baseline (week 0); week 26
Change in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) SDS	Change in IGFBP-3 SDS from baseline (week 0) to week 26 is presented. The range for IGFBP-3 SDS was from -10 to +10. Negative scores indicated a IGFBP-3 below the mean IGFBP-3 for a child with the same age and gender, whereas positive scores indicated a IGFBP-3 above the mean IGFBP-3 for a child with the same age and gender. Positive value in change from baseline in IGFBP-3 SDS indicated that IGFBP-3 SDS was higher than baseline IGFBP-3 SDS. The outcome measure was evaluated based on the data from on-treatment observation period. On-treatment observation period: from first administration and up until last trial contact, week 26 or 14 days after last administration, whichever came first.	Baseline (week 0); week 26

Collaborators and Investigators

• Sponsor: Novo Nordisk A/S
• Investigators: Study Director: Clinical Reporting Anchor and Disclosure 1452, Novo Nordisk A/S

Study record dates

Study Major Dates

• Study Start (Actual): July 4, 2019
• Primary Completion (Actual): May 5, 2021
• Study Completion (Estimated): December 23, 2026

Study Registration Dates

• First Submitted: March 15, 2019
• First Submitted That Met QC Criteria: March 15, 2019
• First Posted (Actual): March 18, 2019

Study Record Updates

• Last Update Posted (Estimated): December 23, 2025
• Last Update Submitted That Met QC Criteria: December 5, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Endocrine System Diseases; Bone Diseases; Musculoskeletal Diseases; Genetic Diseases, Inborn; Bone Diseases, Developmental; Dwarfism; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptide Hormones; Peptides; Amino Acids, Peptides, and Proteins; Pituitary Hormones; Growth Hormone; Pituitary Hormones, Anterior; somapacitan; Human Growth Hormone
• Other Study ID Numbers: NN8640-4245; U1111-1207-9741 (Other Identifier: World Health Organization (WHO)); 2018-000232-10 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No