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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT03878446
Other study ID # NN8640-4245
Secondary ID U1111-1207-97412
Status Active, not recruiting
Phase Phase 2
First received
Last updated
Start date July 4, 2019
Est. completion date December 23, 2026

Study information

Verified date June 2024
Source Novo Nordisk A/S
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

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.

Study Design


Related Conditions & MeSH terms

  • Dwarfism
  • Short Stature Children Born Small for Gestational Age (SGA)

Intervention

Drug:
Somapacitan
Somapacitan injected under the skin once a week.
Norditropin®
Norditropin® injected under the skin once a day.

Locations

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
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
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

Sponsors (1)

Lead Sponsor Collaborator
Novo Nordisk A/S

Countries where clinical trial is conducted

United States,  Algeria,  Austria,  France,  Hungary,  India,  Ireland,  Israel,  Italy,  Japan,  Latvia,  Russian Federation,  Serbia,  Spain,  Thailand,  Ukraine, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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 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
Secondary 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
Secondary 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
Secondary 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
Secondary 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
Secondary 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
Secondary 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
Secondary 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
Secondary 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