Short Stature Children Born Small for Gestational Age (SGA) Clinical Trial
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
| Verified date | March 2024 |
| Source | Novo Nordisk A/S |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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.
| Status | Active, not recruiting |
| Enrollment | 62 |
| Est. completion date | December 23, 2026 |
| Est. primary completion date | May 5, 2021 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 2 Years to 11 Years |
| Eligibility | 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. |
| Country | Name | City | State |
|---|---|---|---|
| Algeria | CHU Bab El Oued Pediatrics Dept | Algiers | |
| Algeria | Endo and Diab Dept El Oued | Algiers | |
| Algeria | endocrino-diabetology department, Hospital IBN BADIS. | Constantine | |
| Austria | Med. Univ. Graz -Klinische Abteilung f. Allgemeine Pädiatrie | Graz | |
| Austria | Kepler Universitätsklinikum GmbH - Med Campus IV (vorm.LFKK) | Linz | Upper Austria |
| Austria | LKH Salzburg- Univ. Klinik f. Kinder- und Jugendheilkunde | Salzburg | |
| Austria | LKH St. Poelten, Kinder-und Jugendheilkunde | St. Poelten | |
| Estonia | Tallinn Children's Hospital | Tallinn | |
| France | Ap-Hp-Hopital de Bicetre-2 | Le Kremlin-Bicetre | |
| France | Hopital de La Timone | MARSEILLE Cédex 05 | |
| France | Ap-Hp-Hopital Necker-2 | Paris | |
| France | Hôpital Necker | Paris | |
| Hungary | Szegedi Tudományegyetem Gyermekgyógyászati Klinika | Szeged | |
| India | Amrita Institute Of Medical Sciences & Research Centre | Kochi | Kerala |
| India | All India Institute of Medical Sciences | New Dehli | New Delhi |
| India | Jehangir Clinical Development Centre | Pune | Maharashtra |
| Ireland | Children's Health Ireland, Crumlin | Dublin | |
| Israel | Rambam Medical Center Children A Dept. | Haifa | |
| Italy | AOU Meyer | Firenze | |
| Italy | Bambin Gesù | Roma | |
| Japan | Kurume University Hospital, Pediatrics | Fukuoka | |
| Japan | Fukuoka Children's Hospital | Fukuoka-shi, Fukuoka | |
| Japan | Hyogo Prefectual Kobe Children's Hospital Dept. Metab & endo | Kobe-shi, Hyogo | |
| Japan | Nara Prefecture General Medical Center_ Nara-shi, Nara | Nara-shi, Nara | |
| Japan | Niigata University Medical & Dental Hospital, Pediatrics | Niigata-shi, Niigata | |
| Japan | Osaka City General Hospital, Pediatric Endocrinology and Me | Osaka | |
| Japan | Osaka Women's and Children's Hospital | Osaka | |
| Japan | Saitama Children's Medical Center, Endocrinorogy&Metabolism | Saitama-shi, Saitama | |
| Japan | National Center for Child Health and Dev, Endo and Metabo | Tokyo | |
| Japan | Tokyo Medical and Dental University Hospital | Tokyo | |
| Latvia | Children's Clinical University Hospital | Riga | |
| Poland | SPSK nr 1 im Prof. T Sokolowskiego | Szczecin | |
| Poland | Instytut Pamieci Centrum Zdrowia Dziecka | Warszawa | |
| Russian Federation | Republic Children's Hospital of Ministry of Health of Udmurt | Izhevsk | |
| Russian Federation | Setchenov First Moscow State Medical University | Moscow | |
| Russian Federation | Children's clinical city hospital #1 | Novosibirsk | |
| Russian Federation | SPSBHI City Children out-patient clinic #44 | Saint-Petersburg | |
| Russian Federation | Samara Regional Children Clinical Hospital n.a. N.N. Ivanova | Samara | |
| Russian Federation | Siberian State Medical University | Tomsk | |
| Serbia | Institute for Mother and Child Health Care of Serbia | Belgrade | |
| Serbia | University Children's Hospital Tirsova | Belgrade | |
| Serbia | University Clinical Centre Kragujevac | Kragujevac | |
| Serbia | Clinical Center in Nis | Nis | |
| Serbia | Institute for Health Care of Children and Adolescents | Novi Sad | |
| Spain | Hospital Sant Joan de Déu | Esplugues Llobregat(Barcelona) | |
| Spain | Hospital Clínico de Santiago de Compostela | Santiago de Compostela | |
| Thailand | King Chulalongkorn Memorial hospital-Ped-Endocrinology | Bangkok | |
| Ukraine | Kharkiv Regional Children Clincial Hospital_Lubyanka | Kharkiv | |
| Ukraine | Institute of Endocrinology and Metabolism of AMSU | Kyiv | |
| United States | Univ of AL at Birmingham_BRM | Birmingham | Alabama |
| United States | CCHMC_Cinc | Cincinnati | Ohio |
| United States | NYU Langone Hospital-LI | Mineola | New York |
| United States | Goryeb Children's Hospital | Morristown | New Jersey |
| United States | Univ Oklahoma Sci Ctr OK City | Oklahoma City | Oklahoma |
| Lead Sponsor | Collaborator |
|---|---|
| Novo Nordisk A/S |
United States, Algeria, Austria, Estonia, France, Hungary, India, Ireland, Israel, Italy, Japan, Latvia, Poland, Russian Federation, Serbia, Spain, Thailand, Ukraine,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Height velocity | cm/year | From baseline (week 0) to week 26 | |
| Secondary | Change in bone age | years | From baseline (week 0) to week 52 | |
| Secondary | Change in height standard deviation score (SDS) | -10 to +10 | From baseline (week 0) to week 26 | |
| Secondary | Change in height velocity SDS | -10 to +10 | From baseline (week 0) to week 26 | |
| Secondary | Change in fasting plasma glucose | mmol/l | From screening (visit 1) to week 26 | |
| Secondary | Change in homeostatic model assessment (HOMA) | Percent | From screening (visit 1) to week 26 | |
| Secondary | Change in glycated haemoglobin (HbA1c) | Percentage points | From screening (visit 1) to week 26 | |
| Secondary | Change in insulin-like growth factor I (IGF-I) SDS | -10 to +10 | From screening (visit 1) to week 26 | |
| Secondary | Change in insulin-like growth factor binding protein 3 (IGFBP-3) SDS | -10 to +10 | From screening (visit 1) to week 26 |