A Research Study in Children With a Low Level of Hormone to Grow. Treatment is Somapacitan Once a Week Compared to Norditropin® Once a Day (REAL4)
Condition: Growth Hormone Deficiency in Children
MeSH terms: Dwarfism, Pituitary
Intervention: Somapacitan; Norditropin®
Intervention description: Somapacitan will be administered subcutaneously (s.c.; under the skin) once weekly by PDS290 pen-injector. Somapacitan can be injected any time during the once weekly dosing day. The dose will be calculated based on the subject's current body weight.; Norditropin® will be administered s.c. once daily by FlexPro® pen-injector. Norditropin® should be injected daily in the evening. The dose will be calculated based on the subject's current body weight.
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Intervention model description: Participants will receive either somapacitan once weekly or Norditropin® once daily for 52 weeks (main trial period). All participants completing the main trial period will receive somapacitan weekly for 3 years (extension trial period).
Masking: None (Open Label)
Enrollment: 200.0
Sex: All
Maximum age: 11 Years
Healthy volunteers: No

Summary:

      The study compares 2 medicines for children who do not have enough hormone to grow:
      somapacitan given once a week (a new medicine) and Norditropin® given once a day (the
      medicine doctors can already prescribe). Researchers will test to see how well somapacitan
      works. The study will also test if somapacitan is safe. Participants will either get
      somapacitan or Norditropin® - which treatment participants get, is decided by chance. Both
      participants and the study doctor will know which treatment participants get. The study will
      last for 4 years. Participants will attend 19 clinic visits and have 1 phone call with the
      study doctor.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Prepubertal children: a) Boys: Age more than or equal to 2 years and 26 weeks and less
             than 11.0 years at screening. Testis volume less than 4 ml. b) Girls: Age more than or
             equal to 2 years and 26 weeks and less than 10.0 years at screening. Tanner stage 1
             for breast development (no palpable glandular breast tissue)

          -  Confirmed diagnosis of growth hormone deficiency determined by two different growth
             hormone stimulation tests performed within 12 months prior to randomisation, defined
             as a peak growth hormone level of less than or equal to 10.0 ng/ml using the World
             Health Organisation (WHO) International Somatropin 98/574 standard

          -  Impaired height defined as at least 2.0 standard deviations below the mean height for
             chronological age and gender at screening according to the standards of Center for
             Disease Control and Prevention

          -  Impaired height velocity, defined as annualised height velocity below the 25th
             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

          -  Insulin-like Growth Factor-I (IGF-I) less than -1.0 SDS at screening, compared to age
             and gender normalized range measured at central laboratory

          -  No prior exposure to growth hormone therapy or 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

          -  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

          -  Diagnosis of attention deficit hyperactivity disorder

          -  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

          -  Prior history or presence of malignancy including intracranial tumours