Clinical trial NCT03878446: A Research Study in Children Born Small and Who Stayed Small. Treatment is Somapacitan Once a Week Compared to Norditropin® Once a Day
Official title: 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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Short Stature Children Born Small for Gestational Age (SGA)
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2019-03-18
Enrollment: 62
Sex: ALL
Minimum age (years): 2
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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.

Primary outcomes:
measure: Height Velocity
timeFrame: Baseline (week 0); week 26
Secondary outcomes:
measure: Change in Ratio of Bone Age Versus Chronological Age
timeFrame: Baseline (week 0); week 52
measure: Change in Height Standard Deviation Score (HSDS)
timeFrame: Baseline (week 0); week 26
measure: Change in Height Velocity Standard Deviation Score (HVSDS)
timeFrame: Baseline (week 0); week 26
measure: Change in Fasting Plasma Glucose
timeFrame: Baseline (week 0); week 26
measure: Change in Homeostatic Model Assessment for Steady State Beta Cell Function (HOMA-B)
timeFrame: Baseline (week 0); week 26
measure: Change in Homeostatic Model Assessment for Insulin Resistance (HOMA-IR)
timeFrame: Baseline (week 0); week 26
measure: Change in Glycated Haemoglobin (HbA1c)
timeFrame: Baseline (week 0); week 26
measure: Change in Insulin-like Growth Factor I (IGF-I) Standard Deviation Score (SDS)
timeFrame: Baseline (week 0); week 26
measure: Change in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) SDS
timeFrame: Baseline (week 0); week 26
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Univ of AL at Birmingham_BRM
state: Alabama
city: Morristown
country: United States
facility: Goryeb Children's Hospital
state: New Jersey
city: Garden City
country: United States
facility: NYU Langone Hospital-LI
state: New York
city: Mineola
country: United States
facility: NYU Langone Hospital-LI
state: New York
city: Cincinnati
country: United States
facility: CCHMC_Cinc
state: Ohio
city: Oklahoma City
country: United States
facility: Univ Oklahoma Sci Ctr OK City
state: Oklahoma
city: Algiers
country: Algeria
facility: CHU Bab El Oued Pediatrics Dept
city: Algiers
country: Algeria
facility: Endo and Diab Dept El Oued
city: Constantine
country: Algeria
facility: endocrino-diabetology department, Hospital IBN BADIS.
city: Linz
country: Austria
facility: Kepler Universitätsklinikum GmbH - Med Campus IV (vorm.LFKK)
state: Upper Austria
city: Graz
country: Austria
facility: Med. Univ. Graz -Klinische Abteilung f. Allgemeine Pädiatrie
city: Salzburg
country: Austria
facility: LKH Salzburg- Univ. Klinik f. Kinder- und Jugendheilkunde
city: Le Kremlin-Bicetre
country: France
facility: Ap-Hp-Hopital de Bicetre-2
city: MARSEILLE Cédex 05
country: France
facility: Hopital de La Timone
city: Paris
country: France
facility: Ap-Hp-Hopital Necker-2
city: Paris
country: France
facility: Hôpital Necker
city: Szeged
country: Hungary
facility: Szegedi Tudományegyetem Gyermekgyógyászati Klinika
city: Kochi
country: India
facility: Amrita Institute Of Medical Sciences & Research Centre
state: Kerala
city: Pune
country: India
facility: Jehangir Clinical Development Centre
state: Maharashtra
city: New Dehli
country: India
facility: All India Institute of Medical Sciences
state: New Delhi
city: Dublin
country: Ireland
facility: Children's Health Ireland, Crumlin
city: Haifa
country: Israel
facility: Rambam Medical Center Children A Dept.
city: Firenze
country: Italy
facility: IRCCS Meyer Firenze
city: Roma
country: Italy
facility: Bambin Gesù
city: Fukuoka-shi, Fukuoka
country: Japan
facility: Fukuoka Children's Hospital
city: Fukuoka
country: Japan
facility: Kurume University Hospital, Pediatrics
city: Kobe-shi, Hyogo
country: Japan
facility: Hyogo Prefectual Kobe Children's Hospital Dept. Metab & endo
city: Nara-shi, Nara
country: Japan
facility: Nara Prefecture General Medical Center_ Nara-shi, Nara
city: Niigata-shi, Niigata
country: Japan
facility: Niigata University Medical & Dental Hospital, Pediatrics
city: Osaka
country: Japan
facility: Osaka City General Hospital, Pediatric Endocrinology and Me
city: Osaka
country: Japan
facility: Osaka Women's and Children's Hospital
city: Saitama-shi, Saitama
country: Japan
facility: Saitama Children's Medical Center, Endocrinorogy&Metabolism
city: Tokyo
country: Japan
facility: Institute of Science Tokyo Hospital
city: Tokyo
country: Japan
facility: National Center for Child Health and Dev, Endo and Metabo
city: Riga
country: Latvia
facility: Children's Clinical University Hospital
city: Izhevsk
country: Russian Federation
facility: Republic Children's Hospital of Ministry of Health of Udmurt
city: Moscow
country: Russian Federation
facility: Setchenov First Moscow State Medical University
city: Novosibirsk
country: Russian Federation
facility: Children's clinical city hospital #1
city: Saint-Petersburg
country: Russian Federation
facility: SPSBHI City Children out-patient clinic #44
city: Samara
country: Russian Federation
facility: Samara Regional Children Clinical Hospital n.a. N.N. Ivanova
city: Tomsk
country: Russian Federation
facility: Siberian State Medical University
city: Belgrade
country: Serbia
facility: University Children's Hospital Tirsova
city: Belgrade
country: Serbia
facility: Institute for Mother and Child Health Care of Serbia
city: Kragujevac
country: Serbia
facility: University Clinical Centre Kragujevac
city: Nis
country: Serbia
facility: Clinical Center in Nis
city: Novi Sad
country: Serbia
facility: Institute for Health Care of Children and Adolescents
city: Esplugues Llobregat(Barcelona)
country: Spain
facility: Hospital Sant Joan de Déu
city: Santiago de Compostela
country: Spain
facility: Hospital Clínico de Santiago de Compostela
city: Bangkok
country: Thailand
facility: King Chulalongkorn Memorial hospital-Ped-Endocrinology
city: Kharkiv
country: Ukraine
facility: Kharkiv Regional Children Clincial Hospital_Lubyanka
city: Kyiv
country: Ukraine
facility: Institute of Endocrinology and Metabolism of AMSU
Study officials:
affiliation: Novo Nordisk A/S
name: Clinical Reporting Anchor and Disclosure 1452
role: STUDY_DIRECTOR
References: