A phase I trial of the mTOR inhibitor temsirolimus in combination with capecitabine in patients with advanced malignancies

Cancer Med. 2021 Mar;10(6):1944-1954. doi: 10.1002/cam4.3672. Epub 2021 Feb 27.

Abstract

Background: Temsirolimus is an mTOR antagonist with proven anticancer efficacy. Preclinical data suggest greater anticancer effect when mTOR inhibitors are combined with cytotoxic chemotherapy. We performed a Phase I assessment of the combination of temsirolimus and capecitabine in patients with advanced solid tumors.

Methods: Patients were enrolled in an alternating dose escalation of temsirolimus (at 15 or 25 mg IV weekly) and capecitabine (at 750, 1000, and 1250 mg/m2 twice daily) in separate Q2-week and Q3-week cohorts. At the recommended Phase II doses (RP2Ds) of temsirolimus and capecitabine (Q2), seven patients were also treated with oxaliplatin (85 mg/m2 , day 1) to determine triplet combination safety and efficacy.

Results: Forty-five patients were enrolled and 41 were evaluable for dose-limiting toxicities (DLTs). The most common adverse events (AEs) were mucositis, fatigue, and thrombocytopenia. The most common grade 3/4 AEs were hypophosphatemia and anemia. Five patients had DLTs, including hypophosphatemia, mucositis, and thrombocytopenia. The RP2Ds were temsirolimus 25 mg +capecitabine 1000 mg/m2 (Q2); and temsirolimus 25 mg +capecitabine 750 mg/m2 (Q3). Of the 38 patients evaluable for response, one had a partial response (PR) and 19 had stable disease (SD). The overall disease control rate was 52%. Five of the 20 patients with SD/PR maintained disease control for >6 months.

Conclusions: The combination of temsirolimus and capecitabine is safe on both a Q2-week and a Q3-week schedule. The combination demonstrated promising evidence of disease control in this highly refractory population and could be considered for testing in disease-specific phase II trials.

Keywords: 5-fluorouracil; capecitabine; colorectal cancer; mTOR; temsirolimus.

Publication types

  • Clinical Trial, Phase I
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Anemia / chemically induced
  • Antineoplastic Agents / administration & dosage*
  • Antineoplastic Agents / adverse effects
  • Antineoplastic Combined Chemotherapy Protocols / administration & dosage*
  • Antineoplastic Combined Chemotherapy Protocols / adverse effects
  • Capecitabine / administration & dosage*
  • Drug Administration Schedule
  • Fatigue / chemically induced
  • Female
  • Fever / chemically induced
  • Humans
  • Hypophosphatemia / chemically induced
  • Male
  • Middle Aged
  • Mucositis / chemically induced
  • Neoplasms / drug therapy*
  • Neoplasms / pathology
  • Oxaliplatin / administration & dosage
  • Oxaliplatin / adverse effects
  • Sirolimus / administration & dosage
  • Sirolimus / adverse effects
  • Sirolimus / analogs & derivatives*
  • TOR Serine-Threonine Kinases / antagonists & inhibitors*
  • Thrombocytopenia / chemically induced
  • Treatment Outcome

Substances

  • Antineoplastic Agents
  • Oxaliplatin
  • temsirolimus
  • Capecitabine
  • MTOR protein, human
  • TOR Serine-Threonine Kinases
  • Sirolimus