Phase I and pharmacokinetic study of 3'-C-ethynylcytidine (TAS-106), an inhibitor of RNA polymerase I, II and III,in patients with advanced solid malignancies

Invest New Drugs. 2012 Feb;30(1):316-26. doi: 10.1007/s10637-010-9535-y. Epub 2010 Sep 14.

Abstract

Background: TAS-106 is a novel nucleoside analog that inhibits RNA polymerases I, II and II and has demonstrated robust antitumor activity in a wide range of models of human cancer in preclinical studies. This study was performed to principally evaluate the feasibility of administering TAS-106 as a bolus intravenous (IV) infusion every 3 weeks.

Patients and methods: Patients with advanced solid malignancies were treated with escalating doses of TAS-106 as a single bolus IV infusion every 3 weeks. Plasma and urine sampling were performed during the first course to characterize the pharmacokinetic profile of TAS-106 and assess pharmacodynamic relationships.

Results: Thirty patients were treated with 66 courses of TAS-106 at eight dose levels ranging from 0.67-9.46 mg/m(2). A cumulative sensory peripheral neuropathy was the principal dose-limiting toxicity (DLT) of TAS-106 at the 6.31 mg/m(2) dose level, which was determined to be the maximum tolerated dose (MTD). Other mild-moderate drug-related toxicities include asthenia, anorexia, nausea, vomiting, myelosuppression, and dermatologic effects. Major objective antitumor responses were not observed. The pharmacokinetics of TAS-106 were dose-proportional. The terminal elimination half-life (t(1/2)) averaged 11.3 ± 3.3 h. Approximately 71% of TAS-106 was excreted in the urine as unchanged drug. Pharmacodynamic relationships were observed between neuropathy and: C(5min;) AUC(0-inf;) and dermatologic toxicity.

Conclusions: The recommended phase II dose of TAS-106 is 4.21 mg/m(2). However, due to a cumulative drug-related peripheral sensory neuropathy that proved to be dose-limiting, further evaluation of this bolus every 21 day infusion schedule will not be pursued and instead, an alternate dosing schedule of TAS-106 administered as a continuous 24-hour infusion will be explored to decrease C(max) in efforts to minimize peripheral neuropathy and maximize antitumor activity.

Publication types

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

MeSH terms

  • Adult
  • Aged
  • Antineoplastic Agents / administration & dosage*
  • Antineoplastic Agents / adverse effects
  • Antineoplastic Agents / pharmacokinetics
  • Area Under Curve
  • Biotransformation
  • Cytidine / administration & dosage
  • Cytidine / adverse effects
  • Cytidine / analogs & derivatives*
  • Cytidine / pharmacokinetics
  • Drug Administration Schedule
  • Enzyme Inhibitors / administration & dosage*
  • Enzyme Inhibitors / adverse effects
  • Enzyme Inhibitors / pharmacokinetics*
  • Feasibility Studies
  • Female
  • Half-Life
  • Humans
  • Infusions, Intravenous
  • Kaplan-Meier Estimate
  • Male
  • Maximum Tolerated Dose
  • Metabolic Clearance Rate
  • Middle Aged
  • Neoplasms / drug therapy*
  • Neoplasms / enzymology
  • Neoplasms / genetics
  • Neoplasms / pathology
  • RNA Polymerase I / antagonists & inhibitors*
  • RNA Polymerase I / metabolism
  • RNA Polymerase II / antagonists & inhibitors*
  • RNA Polymerase II / metabolism
  • RNA Polymerase III / antagonists & inhibitors*
  • RNA Polymerase III / metabolism
  • Texas
  • Treatment Outcome

Substances

  • 1-(3-C-ethynylribopentofuranosyl)cytosine
  • Antineoplastic Agents
  • Enzyme Inhibitors
  • Cytidine
  • RNA Polymerase II
  • RNA Polymerase I
  • RNA Polymerase III