A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to Grow (REAL 8)

A Study Comparing the Effect and Safety of Once Weekly Dosing of Somapacitan With Daily Norditropin® as Well as Evaluating Long-term Safety of Somapacitan in a Basket Study Design in Children With Short Stature Either Born Small for Gestational Age or With Turner Syndrome, Noonan Syndrome, or Idiopathic Short Stature

The study compares two medicines for treatment of children born small and who stay small, or with Turner Syndrome, Noonan Syndrome, or idiopathic short stature. The purpose of the study is to see how well treatment with somapacitan works compared to treatment with Norditropin®. Somapacitan is a new medicine, and Norditropin® is a medicine doctors can already prescribe in some countries. The study will last for upto 5.5 years. The participants will either get somapacitan once a week up to 5.5 years or Norditropin® once a day for 1 year followed by somapacitan once a week for up to 4.5 years. Which treatment the participants get is decided by chance.

Study Overview

• Status: Active, not recruiting
• Conditions: SGA, Turner Syndrome, Noonan Syndrome, ISS
• Intervention / Treatment: Drug: Somapacitan; Drug: Norditropin®

• Study Type: Interventional
• Enrollment (Actual): 412
• Phase: Phase 3

Contacts and Locations

Study Locations

• Austria
  • Dornbirn, Austria, 6850
    • Krankenhaus der Stadt Dornbirn
  • 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)
• Belgium
  • Brussels, Belgium, 1090
    • UZ Brussel
  • Brussels, Belgium, 1200
    • Cliniques Universitaires Saint-Luc - Serv. Pédiatrie
  • Edegem, Belgium, 2650
    • UZA - UZ Antwerpen - Kinderziekenhuis
  • Leuven, Belgium, 3000
    • UZ Leuven - Kindergeneeskunde
• Brazil
  • Paraná
    • Curitiba, Paraná, Brazil, 80810-040
      • Centro de Diabetes Curitiba
  • São Paulo
    • São Paulo, São Paulo, Brazil, 01228-000
      • Cpquali Pesquisa Clinica Ltda
    • São Paulo, São Paulo, Brazil, 01223-001
      • Santa Casa Sao Paulo
    • São Paulo, São Paulo, Brazil, 05403-000
      • Centro de Pesquisa Clínica do Hospital das Clínicas da Faculdade de Medicina da USP
• Bulgaria
  • Pleven, Bulgaria, 5800
    • UMHAT - Dr. Georgi Stranski EAD
  • Plovdiv, Bulgaria, 4002
    • UMHAT Sveti Georgi EAD, Plovdiv, Clinic of Pediatrics
  • Sofia, Bulgaria, 1606
    • SHATPD - Prof. Ivan Mitev EAD
  • Varna, Bulgaria, 9010
    • UMHAT Sveta Marina EAD, First Pediatric Clinic
• Canada
  • Ontario
    • Ottawa, Ontario, Canada, K1H 8L1
      • Children's Hosp-East Ontario
• China
  • Beijing Municipality
    • Beijing, Beijing Municipality, China, 100045
      • Beijing Children's Hospital, Capital Medical University
  • Guangdong
    • Guangzhou, Guangdong, China, 510080
      • The First Affiliated Hospital, Sun Yat-sen University
  • Guangxi
    • Liuchow, Guangxi, China, 545001
      • Liuzhou Maternity and Child Healthcare Hospital-Child Healthcare
  • Hainan
    • Sanya, Hainan, China, 572032
      • Sanya Central Hospital (Hainan Third People's Hospital)-Pediatric
  • Henan
    • Zhengzhou, Henan, China, 450018
      • Henan Children's Hospital Zhengzhou Children's Hospital-Endocrine Genetics and Metabolism
  • Hubei
    • Wuhan, Hubei, China, 430019
      • Wuhan Children Hospital-Endocrine Genetics and Metabolism
    • Wuhan, Hubei, China, 430019
      • Wuhan Children Hospital
    • Wuhan, Hubei, China, 430030
      • Tongji Hospital, Tongji Medical College of HUST-Pediatric
  • Hunan
    • Changsha, Hunan, China, 410007
      • Hunan Children's Hospital-Child Health Center
  • Jilin
    • Changchun, Jilin, China, 130021
      • The First Bethune Hospital of Jilin University-Pediatric
  • Shandong
    • Jinan, Shandong, China, 250098
      • Shandong Provincial Hospital-Pediatric
  • Shanghai Municipality
    • Shanghai, Shanghai Municipality, China, 200127
      • Shanghai Children's Medical Center-Endocrine Genetics and Metabolism
    • Shanghai, Shanghai Municipality, China, 200062
      • Shanghai Children's Hospital -Endocrine Genetics and Metabolism
  • Sichuan
    • Chengdu, Sichuan, China, 610000
      • Chengdu Women's and Children's Central Hospital
• Croatia
  • Zagreb, Croatia, 10 000
    • KBC "Sestre Milosrdnice"
  • Zagreb, Croatia, 10000
    • KBC Zagreb, Zavod za dječju endokrinologiju i dijabetes
• Finland
  • Helsinki, Finland, 00290
    • HUS Pediatric Unit
• France
  • Angers, France, 49100
    • Centre Hospitalier Universitaire D'Angers-2
  • Angers, France, 49033
    • Centre Hospitalier Régional Universitaire d' Angers
  • Bordeaux, France, 33076
    • CHU Pellegrin
  • Bordeaux, France, 33000
    • Centre Hospitalier Universitaire de Bordeaux-Hopital Pellegrin
  • Le Kremlin-Bicêtre, France, 94275
    • Hopital de Bicetre_Le Kremlin-Bicetre
  • Le Kremlin-Bicêtre, France, 94270
    • Ap-Hp-Hopital de Bicetre
  • Marseille, France, 13005
    • Assistance Publique Hopitaux de Marseille-Hopital de La Timone-2
  • Marseille Cédex 05, France, 13385
    • Hôpital de la Timone
  • Paris, France, 75015
    • Hopital Necker
  • Paris, France, 75015
    • Ap-Hp-Hopital Necker
  • Toulouse, France, 31059
    • CHU de Toulouse
  • Toulouse, France, 31059
    • Hopital des Enfants
• Germany
  • Bonn, Germany, 53127
    • Universitätsklinikum Bonn - Kinderklinik
  • Erlangen, Germany, 91054
    • Universitätsklinikum Erlangen - Kinder- und Jugendklinik
  • Frankfurt am Main, Germany, 60596
    • Endokrinologikum Frankfurt
  • Homburg, Germany, 66421
    • Klinik für Allgemeine Pädiatrie und Neonatologie
  • Homburg, Germany, 66424
    • Universität des Saarlandes - Pädiatrie und Neonatologie
• Greece
  • Athens, Greece, 12462
    • U.G.H. "Attikon", Pediatric Endocrinology Outpatient Clinic
  • Goudi/Athens, Greece, 115-27
    • Childrens' Hospital of Athens "Agia Sofia"
  • Goudi/Athens, Greece, 115-27
    • P & A Kyriakou Childrens' Hospital - Department of Endocrinology Growth and Development
  • Pátrai, Greece, 26504
    • General University of Patra - Paediatric Department
  • Thessaloniki, Greece, 54636
    • "AHEPA" University General Hospital of Thessaloniki
  • Thessaloniki, Greece, GR 54642
    • Ippokrateio Gen Hosp of Thessaloniki, 1st Pediatric Clinic
  • Thessaloniki, Greece, GR-54636
    • "AHEPA" University General Hospital of Thessaloniki
  • Thessaloniki, Greece, 546-42
    • 'Ippokrateio' General Hospital of Thessaloniki
  • Thessaloniki, Greece, 54642
    • Ippokrateio Gen Hosp of Thessaloniki, 1st Pediatric Clinic
• 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
  • Cork, Ireland
    • Cork University Hospital
  • Dublin, Ireland, D01 XD99
    • CHI at Temple Street
  • Dublin, Ireland, D12 N512
    • CHI Crumlin Dept of Endocrinology
  • Dublin, Ireland, D24NR0A
    • CHI Tallaght University Hospital
• Israel
  • Beersheba, Israel, 84101
    • Soroka MC - Pediatric Endocrinology
  • Haifa, Israel, 31096
    • Rambam MC - Department of Pediatrics A
  • Petah Tikva, Israel, 49202
    • Schneider MC - Endrocrinology and Diabetes
  • Tel Litwinsky, Israel, 52621
    • Sheba MC - Pediatric endocrinology and adolescent diabetes clinics
  • Ẕerifin, Israel, 7033001
    • Shamir MC - Pediatric and Adolescents Endocrinology unit
• Italy
  • Florence, Italy, 50139
    • IRCCS Meyer Firenze
  • Genova, Italy, 16147
    • Ospedale Pediatrico Gaslini
  • Milan, Italy, 20132
    • Ospedale San Raffaele UO di Pediatria e UO di Neonatologia
  • Naples, Italy, 80138
    • Università degli Studi della Campania "L. Vanvitelli"
  • Roma, Italy, 00165
    • Ospedale Pediatrico Bambino Gesù
  • Roma, Italy, 00165
    • Bambin Gesù
  • Rome, Italy, 00168
    • Fondazione Policlinico Universitario Agostino Gemelli IRCCS
  • Rome, Italy, 00168
    • Fondazione Policlinico Universitario Agostino Gemelli IRCS
  • Lombardy
    • Milan, Lombardy, Italy, 20132
      • Ospedale San Raffaele S.r.l. - Unità Clinica Pediatria e neonatologia
• Japan
  • Fukuoka-shi, Fukuoka, Japan, 813-0017
    • Fukuoka Children's Hospital
  • Fukuoka-shi, Fukuoka, Japan, 813-0017
    • Fukuoka Children's Hospital_Endocrinology and Metabolism
  • Kobe-shi, Hyogo, Japan, 650-0047
    • Hyogo Prefectual Kobe Children's Hospital Dept. Metab & endo
  • Kobe-shi, Hyogo, Japan, 650-0047
    • Hyogo Prefectual Kobe Children's Hospital_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, Japan, 630-8581
    • Nara Prefecture General Medical Center_Nephrology
  • Niigata-shi, Niigata, Japan, 951 8520
    • Niigata University Medical & Dental Hospital_Pediatrics
  • Okayama-shi, Okayama, Japan, 701-1192
    • Okayama Medical Center_Cardiology
  • 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
  • Sapporo, Hokkaido, Japan, 060-8648
    • Hokkaido University Hospital, Pediatrics
  • Sapporo, Hokkaido, Japan, 060-8648
    • Hokkaido University Hospital_Pediatrics
  • Shizuoka-shi, Shizuoka, Japan, 420-8660
    • Shizuoka Children's Hospital, Department of Diabetes and metabolism
  • Tokyo, Japan, 113-8519
    • Institute of Science Tokyo Hospital
  • Tokyo, Japan, 113-8519
    • Institute of Science Tokyo Hospital_Pediatrics
  • Tokyo, Japan, 157 8535
    • National Center for Child Health and Development_Endo and Metabo
  • Tokyo, Japan, 160-8582
    • Keio University Hospital_Pediatrics
  • Toyoake, Aichi, Japan, 470-1192
    • Fujita Health University Hospital, Pediatrics
• Latvia
  • Riga, Latvia, 1004
    • Children's Clinical University Hospital
• Lithuania
  • Kaunas, Lithuania, 50161
    • Hospital of Lithuanian University of Health Sciences Kauno Klinikos
• Malaysia
  • Bandar Puncak Alam Selangor Darul Ehsan, Malaysia, 42300
    • University Technology MARA (UiTM) - Puncak Alam
  • Kelantan
    • Kota Bharu, Kelantan, Malaysia, 16150
      • Hospital Universiti Sains Malaysia_Kota Bharu, Kelantan
  • Kuala Lumpur
    • Kuala Lumpur, Kuala Lumpur, Malaysia, 59100
      • University Malaya Medical Centre
    • Lembah Pantai, Kuala Lumpur, Malaysia, 59100
      • University Malaya Medical Centre
  • Selangor
    • Bandar Puncak Alam, Selangor, Malaysia, 42300
      • Hospital Al-Sultan Abdullah UiTM
• Mexico
  • Puebla City, Mexico, 72190
    • Consultorio de Endocrinología y Pediatría
• Netherlands
  • Rotterdam, Netherlands, 3015 GD
    • Erasmus MC
• Poland
  • Cracow, Poland, 30-663
    • Uniwer. Szpital Dzieciecy Klinika Endokry. Dzieci i Mlodzie
  • Cracow, Poland, 30-663
    • Uniwersytecki Szptal Dziecięcy w Krakowie
  • Gdansk, Poland, 80-952
    • UCK, Klinika Pediatrii, Diabetologii i Endokrynologii,
  • Katowice, Poland, 40-752
    • Górnośląskie Centrum Zdrowia Dziecka Szpital Kliniczny Nr 6
  • Lodz, Poland, 93-338
    • Klinika Endokrynologii i Chorób Metabolicznych
  • Szczecin, Poland, 71-252
    • SPSK nr 1 im Prof. T Sokolowskiego
  • Zabrze, Poland, 41-800
    • SPSK nr 1 im. prof.S.Szyszko w Zabrzu
  • Zabrze, Poland, 41-800
    • Klinika Pediatrii SUM, SPSK nr 1 im. prof.S.Szyszko w Zabrzu_LOC#1
  • Podkarpackie Voivodeship
    • Rzeszów, Podkarpackie Voivodeship, Poland, 35-301
      • Kliniczny Szpital Wojewodzki nr 2 im. Sw. Jadwigi Krolowej w Rzeszowie
• Portugal
  • Lisbon, Portugal, 1649-035
    • Centro Hospitalar Lisboa Norte
  • Lisbon, Portugal, 1649-035
    • Unidade Local De Saúde De Santa Maria E.P.E.
  • Lisbon, Portugal, 1169-045
    • Unidade Local De Saúde De São José, E.P.E.- Hospital D. Estefânia
  • Porto, Portugal, 4050-651
    • ULS De Santo António, E.P.E. - Hospital de Santo António
  • Vila Nova de Gaia, Portugal, 4400-129
    • ULS De Gaia/Espinho_H.Santos Silva_Pediatria
• Saudi Arabia
  • Riyadh, Saudi Arabia, 12713
    • King Faisal Specialist Hospital & Research Centre, Riyadh
  • Riyadh, Saudi Arabia, 14812
    • National Guard Hospital
• 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
    • University Clinical Centre Nis
  • Novi Sad, Serbia, 21000
    • Institute for Health Care of Children and Adolescents
  • RS
    • Niš, RS, Serbia, 18 000
      • University Clinical Centre Nis
• Slovenia
  • Ljubljana, Slovenia, 1525
    • PeK - Dept. of Paediatric Endocrinology, Diabetes and Metabolism
  • Ljubljana, Slovenia, 1525
    • University Children's Hospital
• South Africa
  • Gauteng
    • Johannesburg, Gauteng, South Africa, 1864
      • Department of Peadiatrics CHBAH
    • Pretoria, Gauteng, South Africa, 0181
      • Dr JC van Dyk's Rooms
  • KwaZulu-Natal
    • Mayville, KwaZulu-Natal, South Africa, 4058
      • Dr Y Ganie's site
    • Westville, KwaZulu-Natal, South Africa, 3600
      • Dr K Pillay's site
• South Korea
  • Sejong-si, South Korea, 30099
    • Chungnam National University Sejong Hospital
  • Seoul, South Korea, 05505
    • Asan Medical Center
  • Seoul, South Korea, 03722
    • Severance Hospital, Yonsei University Health System
  • Gyeongsangnam-do
    • Yangsan, Gyeongsangnam-do, South Korea, 50612
      • Pusan National University Yangsan Hospital
• Spain
  • Esplugues de Llobregat, Spain, 08950
    • Hospital Sant Joan de Déu
  • A Coruña
    • Santiago de Compostela, A Coruña, Spain, 15706
      • Complejo Hospitalario Universitario de Santiago
  • Madrid
    • Boadilla del Monte, Madrid, Spain, 28660
      • Hospital Madrid Montepríncipe
    • Boadilla del Monte, Madrid, Spain, 28660
      • H.Madrid Montepríncipe
• Switzerland
  • Bern, Switzerland, 3010
    • Med. Kinder- und Poliklinik
• Thailand
  • Bangkok, Thailand, 10330
    • King Chulalongkorn Memorial hospital-Ped-Endocrinology
  • Bangkok, Thailand, 10400
    • PMK - Paediatrics Endocrinology Centre
  • Bangkok, Thailand, 10330
    • King Chulalongkorn Memorial hospital_Ped-Endocrinology
  • Bangkok, Thailand, 10400
    • Phramongkutklao Hospital_Ped-Endo_Pediatrics
  • Bangkok, Thailand, 10700
    • Siriraj Hospital_Dept of Paediatrics
  • Chiang Mai, Thailand, 50200
    • Maharaj Nakorn Chiang Mai Hospital_Pediatric Endocrinology
  • Khon Kaen, Thailand, 40002
    • Srinagarind Hospital
  • Bangkok
    • Ratchathewi, Bangkok, Thailand, 10400
      • Ramathibodi Hospital - Ped-Endo and Metabolism
  • Ratchathewi
    • Bangkok, Ratchathewi, Thailand, 10400
      • Ramathibodi Hospital - Ped-Endo and Metabolism
• United Kingdom
  • Cambridge, United Kingdom, CB2 0QQ
    • Addenbrooke's Hospital_Cambridge
  • Hull, United Kingdom, HU3 2JZ
    • Hull Royal Infirmary
  • Liverpool, United Kingdom, L12 2AP
    • Alder Hey Children's Hospital
  • London, United Kingdom, SE1 7EH
    • Evelina London Children's Hospital
  • London, United Kingdom, SE1 7EH
    • Evelina Children's Hospital - St Thomas'
  • Manchester, United Kingdom, M13 9WL
    • Royal Manchester Children's Hospital
  • Tooting, United Kingdom, SW17 0RE
    • St Georges Hospital
  • Tooting, United Kingdom, SW17 0RE
    • St George's Hospital
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
      • Univ of AL at Birmingham_BRM
  • California
    • Los Angeles, California, United States, 90027
      • Children's Hospital Los Angeles - Endocrinology
    • Sacramento, California, United States, 95821
      • Sutter Valley Med Fdt Ped Endo
    • San Diego, California, United States, 92123
      • Rady Childrens Hosp San Diego
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Children's Hospital Colorado
    • Centennial, Colorado, United States, 80112
      • Rocky Mt Ped and Endo
    • Greenwood Village, Colorado, United States, 80111-2803
      • Ped Endo Assoc PC-G.V
  • Delaware
    • Wilmington, Delaware, United States, 19803
      • Nemours/AI duPont Hosp-Chld
  • District of Columbia
    • Washington D.C., District of Columbia, United States, 20010-2978
      • Childrens National Medical Ctr
  • Florida
    • Jacksonville, Florida, United States, 32207
      • Nemours Chld Clnc Jacksonville
  • Georgia
    • Atlanta, Georgia, United States, 30318
      • Atlanta Diabetes Associates
  • Idaho
    • Boise, Idaho, United States, 83712
      • St. Luke's Children's Endo
    • Idaho Falls, Idaho, United States, 83404-7596
      • Rocky Mt Clin Res, LLC
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Riley Hosp for Child-Indiana U
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • University of Iowa
  • Massachusetts
    • Worcester, Massachusetts, United States, 01655
      • UMass Medical School
  • Minnesota
    • Minneapolis, Minnesota, United States, 55454
      • Univ of Minnesota M.C.H.
    • Saint Paul, Minnesota, United States, 55102
      • Children's Minnesota
  • Mississippi
    • Jackson, Mississippi, United States, 39216
      • Univ of Mississippi Med Ctr
  • Missouri
    • Kansas City, Missouri, United States, 64108
      • The Children's Mercy Hospital
  • New Jersey
    • Morristown, New Jersey, United States, 07962
      • Goryeb Children's Hospital
  • New York
    • Buffalo, New York, United States, 14203
      • UBMD Peds-Div of Endo/Diabetes
    • Garden City, New York, United States, 11530
      • NYU Langone Hospital-LI
    • Mineola, New York, United States, 11501
      • NYU Langone Hospital-LI
  • North Carolina
    • Raleigh, North Carolina, United States, 27610
      • WakeMed Childn Endo-Dbt_Raleig
  • Ohio
    • Akron, Ohio, United States, 44308
      • Akron Children's Hospital
    • Cincinnati, Ohio, United States, 45229
      • CCHMC_Cinc
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73104
      • Univ Oklahoma Sci Ctr OK City
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15224
      • UPMC Child Hosp-Pittsburgh
  • Texas
    • Dallas, Texas, United States, 75235
      • UT Southwestern Med Cntr
    • Fort Worth, Texas, United States, 76104
      • Cook Children's Medical Center
    • Shavano Park, Texas, United States, 78231
      • Consano Clinical Research, LLC
  • Virginia
    • Charlottesville, Virginia, United States, 22903
      • University Of Virginia Hospital
  • Washington
    • Tacoma, Washington, United States, 98405
      • MultiCare Inst for Res & Innov
  • Wisconsin
    • Marshfield, Wisconsin, United States, 54449
      • Marshfield Clinic

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 10 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion criteria:

1. Informed consent of parent or legally acceptable representative of participant and child assent, as age appropriate must be obtained before any study-related activities. Study-related activities are any procedures that are carried out as part of the study, including activities to determine suitability for the study.

2. No prior exposure to growth promoting therapy, including but not limited to growth hormone, IGF-I and ghrelin analogues.

   Applicable to children with SGA:

3. Born small for gestational age (birth length below -2 SDS OR birth weight below -2 SDS OR both) (according to national standards).

4. Prepubertal children:

   1. Boys:

      • Age above or equal to 2 years and 26 weeks and below 11.0 years at screening.
      • Testis volume below 4 mL

   2. Girls:

      • Age above or equal to 2 years and 26 weeks and below 10.0 years at screening.
      • Tanner stage 1 for breast development: No palpable glandular breast tissue)
5. 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.
6. Impaired height velocity defined as annualized height velocity below the 50th percentile for chronological age and sex according to the standards of Prader calculated over a time span of minimum 6 months and maximum 18 months prior to screening.

7. Body Mass Index below the 95th percentile according to Centers for Disease Control and Prevention, Body Mass Index-for-age growth charts.

   Applicable to girls with TS:

8. Confirmed diagnosis of TS by 30-cell (or more) lymphocyte chromosomal analysis.*

9. Prepubertal girls:

   • Age above or equal to 2 years and 26 weeks and below 10.0 years at screening.
   • Tanner stage 1 for breast development: No palpable glandular breast tissue)
10. Impaired height defined as at least 2.0 standard deviations below the mean height for chronological age and sex at screening according to the standards of Centers for Disease Control and Prevention.
11. Historical height measured 6-18 months prior to screening.

12. Thyroid hormone replacement therapy should be adequate and stable for at least 90 days prior to randomization, if applicable.

    Applicable to children with NS:

13. Clinical diagnosis of NS according to van der Burgt score list

14. Prepubertal children:

    1. Boys:

       • Age above or equal to 2 years and 26 weeks and below 11.0 years at screening.
       • Testis volume below 4 mL

    2. Girls:

       • Age above or equal to 2 years and 26 weeks and below 10.0 years at screening.
       • Tanner stage 1 for breast development: No palpable glandular breast tissue)
15. Impaired height defined as at least 2.0 standard deviations below the mean height for chronological age and sex at screening according to the standards of Centers for Disease Control and Prevention.
16. Historical height measured 6-18 months prior to screening.

17. Thyroid hormone replacement therapy should be adequate and stable for at least 90 days prior to randomization, if applicable.

    Applicable to children with ISS:

18. Prepubertal children:

    1. Boys:

       • Age above or equal to 2 years and 26 weeks and below 11.0 years at screening.
       • Testis volume below 4 mL

    2. Girls:

       • Age above or equal to 2 years and 26 weeks and below 10.0 years at screening.
       • Tanner stage 1 for breast development: No palpable glandular breast tissue)

19. Bone age:

    1. Boys:

       • Bone age below or equal to 12 years.
       • Bone age not delayed or advanced more than 2 years compared to chronological age.

    2. Girls:

       • Bone age below or equal to 11 years.
       • Bone age not delayed or advanced more than 2 years compared to chronological age.
20. 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.
21. Historical height measured 6-18 months prior to screening.

22. One normal GH secretion (GH peak above 7 ng/mL) during GH stimulation test performed within 18 months prior to screening or if such a test is not available for children with ISS, a test should be performed as part of the screening assessments and the result must be available prior to randomization.

    • If a 30-cell count is not available for patients with TS, a test should be done, and results must be available prior to randomization.

Exclusion criteria:

1. Known or suspected hypersensitivity to study intervention(s) or related products.
2. Previous randomization into same sub-study in this study.
3. Receipt of any investigational medicinal product within 3 months before screening or participation in another clinical study at the time of randomization.
4. Children with suspected or confirmed growth hormone deficiency according to local practice.

5. laboratory of

   1. fasting plasma glucose above or equal to 126 mg/dL (7.0 mmol/L) or
   2. HbA1c above or equal to 6.5%.
6. Current inflammatory diseases requiring systemic corticosteroid treatment for longer than 2 consecutive weeks within the last 3 months prior to screening.
7. Children requiring inhaled glucocorticoid therapy at a dose greater than 400 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.
8. 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 (ADHD).
9. Diagnosis of attention deficit hyperactivity disorder (ADHD).
10. History or known presence of any malignancy, intracranial tumour, or intracranial cyst.
11. History or known presence of active Hepatitis B or Hepatitis C (exceptions to this exclusion criterion is the presence of antibodies due to vaccination against Hepatitis B).
12. Any disorder, which in the investigator's opinion, might jeopardize participant's safety or compliance with the protocol.
13. The participant or the parent/legally acceptable representative is likely to be non-compliant in respect to study conduct, as judged by the investigator.
14. Current treatment with sex hormones or aromatase inhibitors.

15. Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with standing height measurements, such as, but not limited to:

    1. Known family history of skeletal dysplasia.
    2. Significant spinal abnormalities including but not limited to scoliosis, kyphosis and spina bifida variants.
    3. Any other disorder/condition that can cause short stature such as, but not limited to, psychosocial deprivation, nutritional disorders, chronic systemic illness and chronic renal disease.

Applicable to children with SGA:

1. TS (including mosaicism).
2. NS.
3. Hormonal deficiencies.
4. Children who are small due to malnutrition defined as -2 standard deviations according to standards. 0¬-5 years: weight for height on World Health Organization Multicentre Growth Reference Study 2006. Above 5 years: World Health Organization 2007 Body Mass Index.
5. 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.

Applicable to children with TS:

1. NS.
2. Mosaicism below 10%.
3. TS with Y-chromosome mosaicism where gonadectomy has not been performed.
4. NYHA class II or above or requiring medication for any heart condition.
5. Coeliac disease where participant is not stable on gluten free diet for the previous 12 months prior to screening.

Applicable to children with NS:

1. TS (including mosaicism).
2. Noonan-related disorders: 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. Molecular genetic panel testing results must be available prior to randomisation to exclude these.
3. Coeliac disease where participant is not stable on gluten free diet for the previous 12 months prior to screening.

Applicable to children with ISS:

1. TS (including mosaicism).
2. NS.
3. Hormonal deficiencies.
4. Born small for gestational age (defined as birth length below -2 SDS OR birth weight below -2 SDS OR both) (according to national standards).
5. 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.

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

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Somapacitan; Participants will receive Somapacitan for 273 weeks	Drug: Somapacitan; Somapacitan will be administered subcutaneously (s.c.) once weekly using PDS290 pen-injector.
Active Comparator: Norditropin®; Participants will receive Norditropin® for 52 weeks (main treatment period) and Somapacitan for 221 weeks (extension period)	Drug: Norditropin®; Norditropin® will be administered s.c. once daily using FlexPro® pen-injector.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Height velocity reported separately for small for gestational age (SGA), Turner syndrome (TS), Noonan syndrome (NS) and idiopathic short stature (ISS)	Measured in centimeter per year (cm/year)	From baseline (week 0) to visit 7 (week 52)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Height standard deviation scores (SDS) reported separately for SGA, TS, NS and ISS	Measured in score. Positive score indicates that the value is closer to or above the reference population compared to baseline.	From baseline (week 0) to visit 7 (week 52)
Change in Height Velocity SDS reported separately for SGA, TS, NS and ISS	Measured in score. Positive score indicates that the value is closer to or above the reference population compared to baseline.	From baseline (week 0) to visit 7 (week 52)
Change in bone age reported separately for SGA, TS and NS	Measured in ratio	From baseline (week 0) to visit 7 (week 52)
Change in bone age for ISS	Measured in ratio	From screening (visit 1) to visit 7 (week 52)
Change in insulin-like growth factor 1 (IGF-1) SDS reported separately for SGA, TS, NA and ISS	Measured in score. Positive score indicates that the value is closer to or above the reference population compared to baseline.	From baseline (week 0) to visit 7 (week 52).
Change in insulin-like growth factor binding protein-3 (IGFBP-3) SDS reported separately for SGA, TS, NA and ISS	Measured in score. Positive score indicates that the value is closer to or above the reference population compared to baseline.	From baseline (week 0) to visit 7 (week 52).
Change in fasting plasma glucose reported separately for SGA, TS, NS and ISS	Measured in millimoles per litre (mmol/L)	From screening (visit 1) to visit 7 (week 52)
Change in homeostatic model assessment-B (HOMA-B) reported separately for SGA, TS, NS and ISS	Measured in percentage (%)	From screening (visit 1) to visit 7 (week 52)
Change in homeostatic model assessment of insulin resistance (HOMAIR) reported separately for SGA, TS, NS and ISS	Measured in %	From screening (visit 1) to visit 7 (week 52)
Change in glycated haemoglobin (HbA1c) reported separately for SGA, TS, NS and ISS	Measured in percentage of HbA1c	From screening (visit 1) to visit 7 (week 52)
Weekly average somapacitan concentration (Cavg) based on population PK analysis	Measured in nanograms per milliliter (ng/mL)	From visit 3 (week 4) to visit 7 (week 52)
Change in fasting plasma glucose reported separately for SGA, TS, NS and ISS	Measured in mmol/L	From screening (visit 1) to visit 15 (week 156)
Change in homeostatic model assessment-B (HOMA-B) reported separately for SGA, TS, NS and ISS	Measured in %	From screening (visit 1) to visit 15 (week 156)
Change in homeostatic model assessment of insulin resistance (HOMA-IR) reported separately for SGA, TS, NS and ISS	Measured in %	From screening (visit 1) to visit 15 (week 156)
Change in glycated haemoglobin (HbA1c) reported separately for SGA, TS, NS and ISS	Measured in percentage of HbA1c	From screening (visit 1) to visit 15 (week 156)

Collaborators and Investigators

• Sponsor: Novo Nordisk A/S
• Investigators: Study Director: Clinical Transparency dept. 2834, Novo Nordisk A/S

Publications and helpful links

General Publications

• Jorge AAL, Albanese A, Hojby M, Soltysik K, Edouard T, Grandone A, Tansey M, Mori J. Once-weekly somapacitan in children with Noonan syndrome: randomized controlled phase 3 trial. Eur J Endocrinol. 2026 Mar 4;194(3):393-403. doi: 10.1093/ejendo/lvag041.
• Abuzzahab J, Dauber A, Hojby M, Soltysik K, Kawai M, Murray PG, Phillip M, Battelino T. Somapacitan in children with idiopathic short stature: a randomized controlled phase 3 study. Eur J Endocrinol. 2026 Feb 4;194(2):242-253. doi: 10.1093/ejendo/lvag027.

Study record dates

Study Major Dates

• Study Start (Actual): August 10, 2022
• Primary Completion (Actual): August 5, 2024
• Study Completion (Estimated): October 29, 2027

Study Registration Dates

• First Submitted: April 8, 2022
• First Submitted That Met QC Criteria: April 8, 2022
• First Posted (Actual): April 15, 2022

Study Record Updates

• Last Update Posted (Actual): April 24, 2026
• Last Update Submitted That Met QC Criteria: April 23, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urogenital Diseases; Endocrine System Diseases; Musculoskeletal Diseases; Cardiovascular Diseases; Male Urogenital Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Heart Diseases; Genetic Diseases, Inborn; Connective Tissue Diseases; Gonadal Disorders; Craniofacial Abnormalities; Musculoskeletal Abnormalities; Congenital Abnormalities; Cardiovascular Abnormalities; Heart Defects, Congenital; Disorders of Sex Development; Urogenital Abnormalities; Sex Chromosome Disorders; Chromosome Disorders; Sex Chromosome Disorders of Sex Development; Gonadal Dysgenesis; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin and Connective Tissue Diseases; Noonan Syndrome; Turner Syndrome; 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: NN8640-4467; 2021-005607-13 (EudraCT Number); U1111-1270-0862 (Other Identifier: World Health Organization (WHO))

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