Phase I study for ridaforolimus, an oral mTOR inhibitor, in Japanese patients with advanced solid tumors

Yoshitaka Seki, Noboru Yamamoto, Yosuke Tamura, Yasushi Goto, Takashi Shibata, Maki Tanioka, Hajime Asahina, Hiroshi Nokihara, Yasuhide Yamada, Takashi Shimamoto, Kazuo Noguchi, Tomohide Tamura

Research output: Contribution to journalArticlepeer-review

23 Citations (Scopus)


Purpose: Ridaforolimus is a non-prodrug mTOR inhibitor. The safety, pharmacokinetics (PK), and antitumor activity of oral ridaforolimus were assessed in Japanese patients with refractory solid tumors. Methods: Ridaforolimus (20 or 40 mg) was administered as a single dose on Day 1, followed by once daily dosing five times a week for a 3-week cycle beginning on Day 8. Full PK sampling was performed on Days 1 and 26. Results: Thirteen patients (7 at 20 mg and 6 at 40 mg) were enrolled. The median treatment duration was 82 days. The most common drug-related adverse events were stomatitis, hypertriglyceridemia, and proteinuria. Two patients had dose-limiting toxicities (grade 3 stomatitis at 20 mg, and grade 3 anorexia and vomiting at 40 mg). Four patients had grade 1 interstitial pneumonitis. Ridaforolimus in the whole blood was rapidly absorbed and slowly eliminated with a half-life of approximately 56-58 h after a single dose. Two patients (with non-small cell lung cancer and angiosarcoma, respectively) achieved a partial response, and five patients (one with thymic cancer and four with soft tissue sarcomas) had a stable disease for ≥16 weeks. Conclusions: Ridaforolimus was well tolerated up to a dose of 40 mg in Japanese patients. Preliminary evidence of antitumor activity was observed for patients with solid tumors. Further investigation at this dose is warranted.

Original languageEnglish
Pages (from-to)1099-1105
Number of pages7
JournalCancer Chemotherapy and Pharmacology
Issue number4
Publication statusPublished - Apr 2012
Externally publishedYes


  • Interstitial pneumonitis
  • mTOR inhibitor
  • Phase I study
  • Ridaforolimus
  • Stomatitis

ASJC Scopus subject areas

  • Oncology
  • Toxicology
  • Pharmacology
  • Cancer Research
  • Pharmacology (medical)


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