CABOLIFE: A Prospective Non-Interventional Study on Effectiveness and Safety of Cabozantinib in Real-Life Setting for Previously Treated Patients with Neuroendocrine Tumours

Study type

• Study topic:
  Disease /health condition

  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:
  CABOMETYX
• Study drug International non-proprietary name (INN) or common name:
  CABOZANTINIB
• Anatomical Therapeutic Chemical (ATC) code:
  (L01EX07) cabozantinib

  cabozantinib
• Medical condition to be studied:
  Pancreatic neuroendocrine tumour metastatic
• Additional medical condition(s):
  Unresectable or metastatic, well-differentiated pancreatic and extra-pancreatic neuroendocrine tumours (pNET/epNET) after prior systemic therapy

Population studied

• Short description of the study population:
  150 adult participants in Austria and Germany who are prescribed cabozantinib tablets for unresectable or metastatic, well differentiated extra-pancreatic (epNET) and pancreatic (pNET) neuroendocrine tumours who have progressed following at least one prior systemic therapy other than somatostatin analogues prior to entry into the study will be included.
• 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)

Study design details

• Study design:
  Prospective, multicentre, non-interventional, single-arm study conducted in Germany and Austria with 150 adult participants with pNET or epNET. Observational period: up to 18 months wich includes 3-month follow-up if treatment discontinued.
• Main study objective:
  To describe the effectiveness of cabozantinib tablets in participantswith previously treated neuroendocrine tumours (NETs) in real-life in terms of disease control rate (DCR) at 6 months of cabozantinib treatment as assessed by the treating physician.
• Outcomes:
  Primary Outcome Measure:; - Disease Control Rate (DCR) at 6 Months; Secondary Outcome Measure:; - Objective Response Rate (ORR) up to 18 Months; - Change from baseline in global and subscale scores of the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire - Core 30 (EORTC QLQ-C30), at baseline and Every 3 Months up to 18 Months.; - Change from baseline in European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire - Gastrointestinal Neuroendocrine Tumors Module (EORTC QLQ-GINET21), baseline and Every 3 Months up to 18 Months; - Percentage of participants experiencing Adverse Events (AEs), non-serious, drug-related, or serious Adverse Events (SAEs), up to 18 Months; - Frequency of Dose Modifications Due to Adverse Events, up to 18 months; - Duration of treatment (DoT), up to 18 Months; - Proportion of participants switching to a new line of therapy and type of therapy received, up to 18 Months; - Disease Control Rate After Switch to Next Line of Therapy, 3 Months After Therapy Switch; - Chromogranin A Levels, baseline and Every 3 Months up to 18 Months; - Neuron-Specific Enolase blood levels, baseline and Every 3 Months up to 18 Months; - Time to next treatment (TTNT), up to 18 Months; - Progression-Free Survival (PFS), up to 18 Months; - Progression-Free Survival Rate at 12 and 18 Months
• Data analysis plan:
  In this non-interventional study, all the analyses will be primarily descriptive in nature. ; The primary endpoint will be DCR of cabozantinib-treated NET participants; the rate of disease control will be presented with the 95% confidence interval (CI). Secondary endpoints, i.e. ORR, QoL, safety, DCR of subsequent treatment, DoT, as well as TTNT will be presented descriptively. The Kaplan-Meier method will be used to obtain the estimates of median PFS (time between the start of treatment with cabozantinib until progression assessed clinically or via imaging as observed by the treating physician or death from any cause) and their associated two-sided 95% CIs or landmark 12-month and 18-month PFS.; Subgroup analyses will be only performed if data permit and subgroups may be merged into larger subgroups if deemed necessary. The subgroups will be based of NET localization (Lung NET, Gastro-Intestinal NET, pNET and other NET).