Observational Study on the Safety and Effectiveness of Tepezza (teprotumumab) in South Korea

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Effectiveness study (incl. comparative)

  Safety study (incl. comparative)
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medicinal product name:
  TEPEZZA
• Study drug International non-proprietary name (INN) or common name:
  TEPROTUMUMAB
• Additional medical condition(s):
  Thyroid Eye Disease (TED)

Population studied

• Short description of the study population:
  Adult patients (≥19 years) prescribed TEPEZZA according to the approved indication in South Korea who provide informed consent. Patients with contraindications or participating in another interventional study are excluded.;
• Age groups:
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 65 years)

      • Adults (65 to < 75 years)
      • Adults (75 to < 85 years)
      • Adults (85 years and over)
• Estimated number of subjects:
  41

Study design details

• Study design:
  This study is an observational, prospective, cohort, multicenter study based on primary data collection in patients treated with TEPEZZA in routine clinical practice in South Korea.
• Main study objective:
  The main objective of the study is to describe the safety of TEPEZZA in routine clinical practice and evaluate effectiveness in patients with thyroid eye disease.
• Setting:
  Approximately 20 participating sites in South Korea enrolling patients prescribed TEPEZZA according to routine clinical practice. Patients are followed from first dose until end of study, up to 8 weeks after the last dose or longer for unresolved hearing impairment.
• Comparators:
  None (single-cohort observational study)
• Outcomes:
  The primary outcomes of the study are:; ; • Incidence of adverse events; • Incidence of serious adverse events; • Incidence of adverse drug reactions; • Incidence of serious adverse drug reactions; • Incidence of unexpected adverse events; • Incidence of unexpected serious adverse events; • Incidence of unexpected adverse drug reactions; • Incidence of unexpected serious adverse drug reactions; • Incidence of adverse events leading to TEPEZZA discontinuation; • Incidence of fatal events; ; The secondary outcomes of the study are:; ; • Proptosis response at End of Study (EOS); • The change in proptosis measurement in the study eye from baseline at EOS; • Overall response at EOS (in case of acute thyroid eye disease [TED]); • Achieving Clinical Activity Score (CAS) value of 0 or 1 at EOS (in case of acute TED); • Clinical outcome assessment by the investigator at EOS
• Data analysis plan:
  Analyses will be descriptive. Categorical variables will be summarized using frequencies and percentages, and continuous variables using descriptive statistics. Safety analyses will include all subjects receiving at least one dose of TEPEZZA with one safety follow-up. Effectiveness analyses will include subjects with at least one follow-up assessment.