DRUG
Somapacitan
Somapacitan will be administered subcutaneously (s.c.) once weekly using PDS290 pen-injector.
Status
Active, not recruiting
Phase
Phase 3
Enrollment
412
Locations
199
Results
Not posted
Publications
2
Study summary
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.
Interventions
DRUG
Somapacitan will be administered subcutaneously (s.c.) once weekly using PDS290 pen-injector.
DRUG
Norditropin® will be administered s.c. once daily using FlexPro® pen-injector.
Timeline
First posted
Apr 15, 2022
Study start
Aug 10, 2022
Primary completion
Aug 5, 2024
Study completion
Oct 29, 2027
Results posted
Not reported
Registry updated
Jun 12, 2026
Outcomes
Height velocity reported separately for small for gestational age (SGA), Turner syndrome (TS), Noonan syndrome (NS) and idiopathic short stature (ISS)
Time frame · From baseline (week 0) to visit 7 (week 52)
Measured in centimeter per year (cm/year)
Change in Height standard deviation scores (SDS) reported separately for SGA, TS, NS and ISS
Time frame · From baseline (week 0) to visit 7 (week 52)
Measured in score. Positive score indicates that the value is closer to or above the reference population compared to baseline.
Change in Height Velocity SDS reported separately for SGA, TS, NS and ISS
Time frame · From baseline (week 0) to visit 7 (week 52)
Measured in score. Positive score indicates that the value is closer to or above the reference population compared to baseline.
Change in bone age reported separately for SGA, TS and NS
Time frame · From baseline (week 0) to visit 7 (week 52)
Measured in ratio
Change in bone age for ISS
Time frame · From screening (visit 1) to visit 7 (week 52)
Measured in ratio
Change in insulin-like growth factor 1 (IGF-1) SDS reported separately for SGA, TS, NA and ISS
Time frame · From baseline (week 0) to visit 7 (week 52).
Measured in score. Positive score indicates that the value is closer to or above the reference population compared to baseline.
Change in insulin-like growth factor binding protein-3 (IGFBP-3) SDS reported separately for SGA, TS, NA and ISS
Time frame · From baseline (week 0) to visit 7 (week 52).
Measured in score. Positive score indicates that the value is closer to or above the reference population compared to baseline.
Change in fasting plasma glucose reported separately for SGA, TS, NS and ISS
Time frame · From screening (visit 1) to visit 7 (week 52)
Measured in millimoles per litre (mmol/L)
Change in homeostatic model assessment-B (HOMA-B) reported separately for SGA, TS, NS and ISS
Time frame · From screening (visit 1) to visit 7 (week 52)
Measured in percentage (%)
Change in homeostatic model assessment of insulin resistance (HOMAIR) reported separately for SGA, TS, NS and ISS
Time frame · From screening (visit 1) to visit 7 (week 52)
Measured in %
Change in glycated haemoglobin (HbA1c) reported separately for SGA, TS, NS and ISS
Time frame · From screening (visit 1) to visit 7 (week 52)
Measured in percentage of HbA1c
Eligibility
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 locations
Austria · Belgium · Brazil · Bulgaria · Canada · China · Croatia · Finland · France · Germany · Greece · India · Ireland · Israel · Italy · Japan · Latvia · Lithuania · Malaysia · Mexico · Netherlands · Poland · Portugal · Saudi Arabia · Serbia · Slovenia · South Africa · South Korea · Spain · Switzerland · Thailand · United Kingdom · United States. Showing up to 24 locations stored in the fast local snapshot.
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
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
Atlanta Diabetes Associates
Atlanta, Georgia, United States
St. Luke's Children's Endo
Boise, Idaho, United States
Rocky Mt Clin Res, LLC
Idaho Falls, Idaho, United States
Riley Hosp for Child-Indiana U
Indianapolis, Indiana, United States
University OF Iowa
Iowa City, Iowa, United States
UMass Medical School
Worcester, Massachusetts, United States
Univ of Minnesota M.C.H.
Minneapolis, Minnesota, United States
Children's Minnesota
Saint Paul, Minnesota, United States
Univ of Mississippi Med Ctr
Jackson, Mississippi, United States
The Children's Mercy Hospital
Kansas City, Missouri, United States
Goryeb Children's Hospital
Morristown, New Jersey, United States
UBMD Peds-Div of Endo/Diabetes
Buffalo, New York, United States
NYU Langone Hospital-LI
Garden City, New York, United States
NYU Langone Hospital-LI
Mineola, New York, United States
Publications
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