NN8640-4787 A Non-interventional, Observational, Registry-based Study to Investigate Long-term Safety and Clinical Parameters of Somapacitan Treatment in Paediatric Patients With Growth Hormone Deficiency During Routine Clinical Practice

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Safety study (incl. comparative)
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medicinal product name:
  SOGROYA
• Study drug International non-proprietary name (INN) or common name:
  SOMAPACITAN
• Anatomical Therapeutic Chemical (ATC) code:
  (H01AC07) somapacitan

  somapacitan
• Medical condition to be studied:
  Growth hormone deficiency

  Neoplasm

  Type 2 diabetes mellitus

Population studied

• Short description of the study population:
  Paediatric participants with growth hormone deficiency.; Inclusion Criteria:; - Treated with commercially available somapacitan according to local practice at the discretion of the physician.; - Primary confirmed diagnosis of growth hormone deficiency as per local practice.; - Male or female below 18 years of age at the time of signing informed consent in the GLoBE-Reg.; Exclusion Criteria:; - Participants with active malignancy or in treatment for active pre-existing malignancy.
• Age groups:
  • Paediatric Population (< 18 years)

    • Neonate

      • Preterm newborn infants (0 – 27 days)
      • Term newborn infants (0 – 27 days)
    • Infants and toddlers (28 days – 23 months)
    • Children (2 to < 12 years)
    • Adolescents (12 to < 18 years)
• Estimated number of subjects:
  400

Study design details

• Main study objective:
  To investigate long-term safety of somapacitan treatment in paediatric patients with growth hormone deficiency (GHD) in the setting of routine clinical practice with special focus on neoplasms and type 2 diabetes mellitus (T2DM).
• Outcomes:
  Number of adverse drug reactions, Number of medication errors (incorrect dose administration), Number of participants with incident neoplasm, Number of participants with incident diabetes mellitus type 2, Height velocity, Change in height velocity standard deviation score (HVSDS), Change in height standard deviation score (HSDS), Change in insulin-like growth factor I (IGF-I) standard deviation score (SDS), Change in bone age (measured as years), Change in bone age (measured as months), Participants reaching near adult height, Change in height SDS in participants reaching near adult height.
• Data analysis plan:
  No statistical comparison is planned due to limitations in the observational nature of the data collection.