A Phase II trial of aprinocarsen, an antisense oligonucleotide inhibitor of protein kinase C alpha, administered as a 21-day infusion to patients with advanced ovarian carcinoma

Cancer. 2004 Jan 15;100(2):321-6. doi: 10.1002/cncr.11909.

Abstract

Background: It has been postulated that protein kinase C alpha (PKC-alpha) plays a pivotal role in signal transduction in tumor cancer cells. Aprinocarsen, a 20-base antisense oligonucleotide, has shown ability to inhibit PKC-alpha protein expression and inhibit tumor growth in human xenograft models. In a previous Phase I trial, the authors demonstrated the safety and some evidence of activity in ovarian carcinoma of aprinocarsen administered as a 21-day, continuous, intravenous infusion.

Methods: In this Phase II trial, 36 patients with advanced ovarian carcinoma were treated with aprinocarsen at a dose of 2 mg/kg per day delivered as a 21-day, continuous, intravenous infusion. The primary objective was to determine the antitumor response, and the secondary objectives were to evaluate toxicity and to evaluate effects on quality of life (QOL).

Results: Between September 1997 and December 1999, 36 patients (median age, 58 years) were enrolled in this trial. Patients were stratified into 2 groups: a platinum-sensitive group (n = 12 patients) and a platinum-resistant group (n = 24 patients). All 36 patients were evaluable for toxicity, and 27 patients were fully assessable for antitumor response after 2 cycles of therapy. All patients had received prior treatments. No objective responses were noted in the platinum-sensitive group. In the platinum-resistant group, 1 patient had some evidence of antitumor activity indicated by a decrease in serum CA 125 and stable disease on imaging studies for 8 months. No changes were noted in overall patient ratings for any of the five QOL domains.

Conclusions: When it was administered as a single agent, aprinocarsen did not have significant clinical activity in patients with advanced ovarian carcinoma. Further study may be warranted in combination with platinum-based regimens.

Publication types

  • Clinical Trial
  • Clinical Trial, Phase II
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Aged
  • Antineoplastic Agents / administration & dosage
  • Antineoplastic Agents / adverse effects
  • Antineoplastic Agents / therapeutic use*
  • Drug Resistance, Neoplasm
  • Female
  • Humans
  • Infusions, Intravenous
  • Middle Aged
  • Oligodeoxyribonucleotides, Antisense / administration & dosage
  • Oligodeoxyribonucleotides, Antisense / adverse effects
  • Oligodeoxyribonucleotides, Antisense / therapeutic use*
  • Oligonucleotides, Antisense / administration & dosage
  • Oligonucleotides, Antisense / adverse effects
  • Oligonucleotides, Antisense / therapeutic use*
  • Ovarian Neoplasms / drug therapy*
  • Protein Kinase C / antagonists & inhibitors*
  • Protein Kinase C-alpha
  • Quality of Life
  • Self-Assessment
  • Thionucleotides / administration & dosage
  • Thionucleotides / adverse effects
  • Thionucleotides / therapeutic use*

Substances

  • Antineoplastic Agents
  • Oligodeoxyribonucleotides, Antisense
  • Oligonucleotides, Antisense
  • Thionucleotides
  • ISIS 3521
  • PRKCA protein, human
  • Protein Kinase C
  • Protein Kinase C-alpha