A Prospective, Non-interventional (NIS), Long-term, Post-Authorisation Safety Study (PASS) of Patients Treated With Lonapegsomatropin (SkyPASS)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Other
• If ‘other’, further details on the scope of the study:
  The goal of this study is to further characterise the potential long-term safety risks of lonapegsomatropin in patients treated with lonapegsomatropin under real-world conditions in the post-marketing setting.

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  SKYTROFA
• Medical condition to be studied:
  Growth hormone deficiency

Population studied

• Age groups:
  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)
• Estimated number of subjects:
  500

Study design details

• Main study objective:
  To evaluate the occurrence of neoplasms (benign, malignant and unspecified) and type 2 diabetes mellitus in patients treated with lonapegsomatropin
• Outcomes:
  1. To evaluate the occurrence of neoplasms (benign, malignant and unspecified) Time Frame: 5 years 2. To evaluate the occurrence of type 2 diabetes mellitus Time Frame: 5 years, 3. Occurrence of renal, hepatic, immunologic and neurologic adverse events 4. Occurrence of medication errors in patients treated with lonapegsomatropin 5. Characterise Insulin-like Growth Factor-1 (IGF-1) response 6. Compare the occurrence of neoplasms in patients treated with lonapegsomatropin 7. Compare the occurrence of type 2 diabetes mellitus in patients treated with lonapegsomatropin
• Data analysis plan:
  The Analysis Population will be all patients enrolled. Unless otherwise specified, categorical endpoints will be summarised using counts and percent, continuous endpoints will be summarised using mean, standard deviation, median, minimum, and maximum values.