PrOspective Non-interventional study in patients with locally advanced or metastatic TRK fusion cancer treated with larotrectinib (ON-TRK)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Safety study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  VITRAKVI
• Medical condition to be studied:
  NTRK gene fusion overexpression

Population studied

• Age groups:
  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)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Estimated number of subjects:
  300

Study design details

• Main study objective:
  The primary objective of this study is to describe the safety of larotrectinib in adult and pediatric patients with locally advanced or metastatic TRK fusion cancer, including incidences of all treatment-emergent adverse events (TEAEs) in real-world practice conditions
• Outcomes:
  1. Number of participants with treatmentemergent adverse events (TEAEs), 2. Severity of TEAEs, 3. Seriousness of TEAEs, 4. Outcome of TEAEs, 5. Causality of TEAEs, 6. Action taken related to larotrectinib treatment. 1. ORR, 2. PFS, 3. OS, 4. (Pediatric cohort) Change in height and weight from baseline by visit, developmental milestones abnormalities and tanner stage abnormalities, 5. Neurological abnormalities, 6. Total dose, starting and ending dose, dose modification during treatment and duration of treatment, 7. DCR, 8. DOR, 9. TTR, 10. ORR,DCR,DOR,TTR,PFS,OS by patient subgroup(s).
• Data analysis plan:
  Statistical analyses will be of explorative and descriptive nature. The study is not designed to support any formal statistical testing. Unless otherwise stated, all CIs will be given at a 2-sided 95% level. All variables will be analyzed descriptively with appropriate statistical methods: categorical variables by frequency tables and continuous variables by sample statistics. Patients who took at least one dose of larotrectinib will be included in the safety analysis set. Patients who took at least one dose of larotrectinib, did not violate a major inclusion/exclusion criterion, and had at least one post baseline assessment after receiving larotrectinib will be included in the full analysis set. Safety data will be analyzed on the safety analysis set, effectiveness data on the full analysis set. Demographic and baseline data will be described for both full and safety analysis sets.