Preliminary efficacy, safety, pharmacokinetics, pharmacodynamics and quality of life study of pegylated recombinant human arginase 1 in patients with advanced hepatocellular carcinoma

Invest New Drugs. 2015 Apr;33(2):496-504. doi: 10.1007/s10637-014-0200-8. Epub 2015 Feb 10.

Abstract

This study was designed to evaluate the efficacy, safety profile, pharmacokinetics, pharmacodynamics and quality of life of pegylated recombinant human arginase 1 (Peg-rhAgr1) in patients with advanced hepatocellular carcinoma (HCC). Patients were given weekly doses of Peg-rhAgr1 (1600 U/kg). Tumour response was assessed every 8 weeks using RECIST 1.1 and modified RECIST criteria. A total of 20 patients were recruited, of whom 15 were deemed evaluable for treatment efficacy. Eighteen patients (90%) were hepatitis B carriers. Median age was 61.5 (range 30-75). Overall disease control rate was 13%, with 2 of the 15 patients achieving stable disease for >8 weeks. The median progression-free survival (PFS) was 1.7 (95% CI: 1.67-1.73) months, with median overall survival (OS) of all 20 enrolled patients being 5.2 (95% CI: 3.3-12.0) months. PFS was significantly prolonged in patients with adequate arginine depletion (ADD) >2 months versus those who had ≤2 months of ADD (6.4 versus 1.7 months; p = 0.01). The majority of adverse events (AEs) were grade 1/2 non-hematological toxicities. Transient liver dysfunctions (25%) were the most commonly reported serious AEs and likely due to disease progression. Pharmacokinetic and pharmacodynamic data showed that Peg-rhAgr1 induced rapid and sustained arginine depletion. The overall quality of life of the enrolled patients was well preserved. Peg-rhAgr1 is well tolerated with a good toxicity profile in patients with advanced HCC. A weekly dose of 1600 U/kg is sufficient to induce ADD. Significantly longer PFS times were recorded for patients who had ADD for >2 months.

Publication types

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

MeSH terms

  • Adult
  • Aged
  • Antineoplastic Agents / administration & dosage
  • Antineoplastic Agents / adverse effects
  • Antineoplastic Agents / pharmacokinetics
  • Antineoplastic Agents / pharmacology*
  • Antineoplastic Combined Chemotherapy Protocols
  • Area Under Curve
  • Arginase / administration & dosage
  • Arginase / adverse effects
  • Arginase / pharmacokinetics
  • Arginase / pharmacology*
  • Carcinoma, Hepatocellular / drug therapy*
  • Chemistry, Pharmaceutical
  • Disease-Free Survival
  • Dose-Response Relationship, Drug
  • Female
  • Half-Life
  • Humans
  • Liver Neoplasms / drug therapy*
  • Male
  • Metabolic Clearance Rate
  • Middle Aged
  • Polyethylene Glycols / chemistry*
  • Prospective Studies
  • Quality of Life*
  • Recombinant Proteins
  • Response Evaluation Criteria in Solid Tumors

Substances

  • Antineoplastic Agents
  • Recombinant Proteins
  • Polyethylene Glycols
  • ARG1 protein, human
  • Arginase