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 Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
399
Somapacitan will be administered subcutaneously (s.c.) once weekly using PDS290 pen-injector.
Norditropin® will be administered s.c. once daily using FlexPro® pen-injector.
Univ of AL at Birmingham_BRM
Birmingham, Alabama, United States
Children's Hospital Los Angeles - Endocrinology
Los Angeles, California, United States
Sutter Valley Med Fdt Ped Endo
Sacramento, California, United States
Rady Childrens Hosp San Diego
San Diego, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
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)
Time frame: From baseline (week 0) to visit 7 (week 52)
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.
Time frame: 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.
Time frame: From baseline (week 0) to visit 7 (week 52)
Change in bone age reported separately for SGA, TS and NS
Measured in ratio
Time frame: From baseline (week 0) to visit 7 (week 52)
Change in bone age for ISS
Measured in ratio
Time frame: 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.
Time frame: 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.
Time frame: 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)
Time frame: 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 (%)
Time frame: 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 %
Time frame: 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
Time frame: 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)
Time frame: 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
Time frame: 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 %
Time frame: 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 %
Time frame: 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
Time frame: From screening (visit 1) to visit 15 (week 156)
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Rocky Mt Ped and Endo
Centennial, Colorado, United States
Ped Endo Assoc PC-G.V
Greenwood Village, Colorado, United States
Nemours/AI duPont Hosp-Chld
Wilmington, Delaware, United States
Childrens National Medical Ctr
Washington D.C., District of Columbia, United States
Nemours Chld Clnc Jacksonville
Jacksonville, Florida, United States
...and 189 more locations