Individualized patient dosing in phase I clinical trials: the role of escalation with overdose control in PNU-214936

J Clin Oncol. 2004 Feb 15;22(4):602-9. doi: 10.1200/JCO.2004.12.034.

Abstract

Purpose: A patient-specific dose-escalation scheme using a Bayesian model of Escalation with Overdose Control (EWOC) was conducted to establish the maximum tolerated dose (MTD) of PNU-214936 in advanced non-small-cell lung cancer (NSCLC). PNU-214936 is a murine Fab fragment of the monoclonal antibody 5T4 fused to a mutated superantigen staphylococcal enterotoxin A (SEA).

Patients and methods: Seventy-eight patients with NSCLC were treated with an individualized dose of PNU-214936 calculated using EWOC, based on their anti-SEA antibody level, and given as a 3-hour infusion on 4 consecutive days.

Results: Fever (82%; grade 3 to 4, 2.6%) and hypotension (57%; grade 3 to 4, 9%) were the most common toxicities. Eight dose-limiting toxicities occurred, as defined as any grade 4 toxicity occurring within the first 5 days. The MTD was defined as a function of pretreatment anti-SEA antibody level. MTD ranged from 103 ng/kg for patients with anti-SEA concentrations < or = 10 pmol/mL, to 601 ng/kg for patients with anti-SEA concentrations of 91 to 150 pmol/mL. A minor tumor response was demonstrated in five of 66 assessable patients.

Conclusion: EWOC determined phase I doses of PNU-214936 that were adjusted for patient anti-SEA antibody level, while safeguarding against overdose. Furthermore, the method permitted the construction of a dosing algorithm that would allow patients in subsequent clinical investigations to be treated with a dose of PNU-214936 that is tailored to their specific tolerance for the agent, as reflected by their pretreatment anti-SEA.

Publication types

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

MeSH terms

  • Adult
  • Aged
  • Bayes Theorem
  • Carcinoma, Non-Small-Cell Lung / drug therapy*
  • Carcinoma, Non-Small-Cell Lung / immunology
  • Dose-Response Relationship, Drug
  • Enterotoxins / administration & dosage*
  • Enterotoxins / immunology
  • Female
  • Humans
  • Infusions, Intravenous
  • Interferon Inducers / administration & dosage*
  • Interferon Inducers / immunology
  • Lung Neoplasms / drug therapy*
  • Lung Neoplasms / immunology
  • Male
  • Maximum Tolerated Dose
  • Middle Aged
  • Models, Statistical
  • Superantigens / administration & dosage*
  • Superantigens / immunology

Substances

  • Enterotoxins
  • Interferon Inducers
  • Superantigens
  • enterotoxin A, Staphylococcal