Observational study evaluating the long-term safety and efficacy of avapritinib in the first-line treatment of patients with platelet-derived growth factor receptor alpha (PDGFRA) D842V-mutated gastrointestinal stromal tumour (GIST)

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  AYVAKYT
• Study drug International non-proprietary name (INN) or common name:
  AVAPRITINIB
• Anatomical Therapeutic Chemical (ATC) code:
  (L01EX18) avapritinib

  avapritinib
• Medical condition to be studied:
  Gastrointestinal stromal tumour
• Additional medical condition(s):
  Platelet-derived growth factor receptor alpha (PDGFRA) D842V-mutated gastrointestinal stromal tumour (GIST)

Population studied

• 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:
  31

Study design details

• Main study objective:
  The overall objective is to collect long-term safety and efficacy data for avapritinib in first-line patients with PDGFRA D842V-mutated GIST, (or following ≤4 months of imatinib treatment).
• Outcomes:
  To describe types, severity and rates of AEs, SAEs, AEs leading to discontinuation or decreased dosing of avapritinib, AESIs, and deaths. ; This PASS has been implemented following a commitment to the EMA to address the Specific Obligation of the CMA in Europe to provide additional safety data for patients with PDGFRA D842V-mutated GIST on first-line avapritinib treatment, (or≤4 months of imatinib), ; To evaluate efficacy in terms of disease response to treatment, PFS and OS as well as duration of treatment and duration of response.
• Data analysis plan:
  Analyses will be performed on the safety population, i.e. enrolled patients who received at least one dose of avapritinib. ; For the primary endpoint, the number, proportions and incidence rate of patients experiencing an AE (overall and by SOC/PT terms), and the number, proportions of patients with avapritinib treatment changes due to an AE (overall and by comorbidity) will be determined. ; For the secondary endpoints, the following will be determined: 1) overall survival (OS), progression-free survival, treatment duration, tumor response and response duration by the Kaplan-Meier method, 2) OS rates at 12- and 24-months, and 3) overall response rates. ; Inviting consecutive patients at sites and retrospective patients who discontinued treatment will minimize selection bias. ; A statistical analysis plan will be developed before the first data lock point, which will detail handling of missing data, correction of inconsistencies or errors, and differences in outcome definitions.