Ridaforolimus as a single agent in advanced endometrial cancer: Results of a single-arm, phase 2 trial

N. Colombo, D. S. McMeekin, P. E. Schwartz, C. Sessa, P. A. Gehrig, R. Holloway, P. Braly, D. Matei, A. Morosky, P. F. Dodion, M. H. Einstein, F. Haluska

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100 Scopus citations


Background: This open-label, multicentre, phase 2 trial evaluated the efficacy and tolerability of the mammalian target of rapamycin inhibitor ridaforolimus in women with advanced endometrial cancer. Methods: Women with measurable recurrent or persistent endometrial cancer and documented disease progression were treated with ridaforolimus 12.5 mg intravenously once daily for 5 consecutive days every 2 weeks in a 4-week cycle. The primary end point was clinical benefit response, defined as an objective response or prolonged stable disease of 16 weeks or more.Results:In all, 45 patients were treated with single-agent ridaforolimus. Clinical benefit was achieved by 13 patients (29%), including 5 (11%) with confirmed partial responses and 8 (18%) with prolonged stable disease. All patients with clinical benefit response received ridaforolimus for more than 4 months. In this heavily pretreated population, the 6-month progression-free survival was 18%. Ridaforolimus was generally well tolerated: adverse events were predictable and manageable, consistent with prior studies in other malignancies. Overall, the most common adverse events were diarrhoea (58%) and mouth sores (56%); most common grade 3 or higher adverse events were anaemia (27%) and hyperglycaemia (11%). Conclusion: Single-agent ridaforolimus has antitumor activity and acceptable tolerability in advanced endometrial cancer patients. Further clinical evaluation of ridaforolimus is warranted.

Original languageEnglish (US)
Pages (from-to)1021-1026
Number of pages6
JournalBritish Journal of Cancer
Issue number5
StatePublished - Mar 19 2013
Externally publishedYes

All Science Journal Classification (ASJC) codes

  • Oncology
  • Cancer Research


  • clinical benefit response
  • endometrial cancer
  • mammalian target of rapamycin inhibitor
  • ridaforolimus
  • stable disease


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