Regorafenib in advanced colorectal cancer: a phase I study

British Journal of Cancer, 05/09/2012

Strumberg D et al. – Regorafenib showed tolerability and antitumour activity in patients with metastatic colorectal cancer (CRC). This expanded–cohort phase I study provided the foundation for further clinical trials of regorafenib in this patient population.


  • Patients received oral regorafenib 60–220mg daily (160mg daily in the extension cohort) in cycles of 21days on, 7days off treatment.
  • Assessments included toxicity, response, pharmacokinetics and pharmacodynamics.


  • Thirty-eight patients with heavily pretreated CRC (median 4 prior lines of therapy, range 0–7) were enrolled in the dose-escalation and extension phases; 26 patients received regorafenib 160mg daily.
  • Median treatment duration was 53days (range 7–280days).
  • The most common treatment-related toxicities included hand–foot skin reaction, fatigue, voice change and rash.
  • Twenty-seven patients were evaluable for response: 1 achieved partial response and 19 had stable disease.
  • Median progression-free survival was 107days (95% CI, 66–161).
  • At steady state, regorafenib and its active metabolites had similar systemic exposure.
  • Pharmacodynamic assessment indicated decreased tumour perfusion in most patients.

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