A Research Study in Children Born Small and Who Stayed Small. Treatment is Somapacitan Once a Week Compared to Norditropin® Once a Day

Study Overview
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Brief Summary
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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 4 years. Participants will take either an injection once every week or once every day. Participants will have 9 clinic visits and will be in the study for 1 year. The follow-up period is at least 30 days.

Official Title
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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

Conditions
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Short Stature Children Born Small for Gestational Age (SGA)

Intervention / Treatment
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* Drug: somapacitan
* Drug: Norditropin®


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Pre-pubertal children, boys: age between 2.5 and 11.0 years at screening. testes volume below 4 ml. Pre-pubertal children, girls: age between 2.5 and 10.0 years at screening. 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.

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 11 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 0.24 mg/kg somapacitan<br>Same treatment in main period (13 weeks) and extension period (39 weeks) | Drug: somapacitan<br>* Somapacitan injected under the skin once a week for a total of 52 weeks<br>|
| Experimental: 0.20 mg/kg somapacitan<br>Same treatment in main period (13 weeks) and extension period (39 weeks) | Drug: somapacitan<br>* Somapacitan injected under the skin once a week for a total of 52 weeks<br>|
| Experimental: 0.16 mg/kg somapacitan<br>Same treatment in main period (13 weeks) and extension period (39 weeks) | Drug: somapacitan<br>* Somapacitan injected under the skin once a week for a total of 52 weeks<br>|
| Active Comparator: 0.035 mg/kg Norditropin®<br>Same treatment in main period (13 weeks) and extension period (39 weeks) | Drug: Norditropin®<br>* Norditropin® injected under the skin once a day for a total of 52 weeks<br>|
| Active Comparator: 0.067 mg/kg Norditropin®<br>Same treatment in main period (13 weeks) and extension period (39 weeks) | Drug: Norditropin®<br>* Norditropin® injected under the skin once a day for a total of 52 weeks<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Height velocity | cm/year | From baseline (week 0) to week 26 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in bone age | years | From baseline (week 0) to week 52 | 
| Change in height standard deviation score (SDS) | -10 to +10 | From baseline (week 0) to week 26 | 
| Change in height velocity SDS | -10 to +10 | From baseline (week 0) to week 26 | 
| Change in fasting plasma glucose | mmol/l | From screening (visit 1) to week 26 | 
| Change in homeostatic model assessment (HOMA) | Percent | From screening (visit 1) to week 26 | 
| Change in glycated haemoglobin (HbA1c) | Percentage points | From screening (visit 1) to week 26 | 
| Change in insulin-like growth factor I (IGF-I) SDS | -10 to +10 | From screening (visit 1) to week 26 | 
| Change in insulin-like growth factor binding protein 3 (IGFBP-3) SDS | -10 to +10 | From screening (visit 1) to week 26 |