Inter-study variability of preclinical in vivo safety studies and translational exposure-QTc relationships--a PKPD meta-analysis

Br J Pharmacol. 2015 Sep;172(17):4364-79. doi: 10.1111/bph.13218. Epub 2015 Jul 21.

Abstract

Background and purpose: Preclinical cardiovascular safety studies (CVS) have been compared between facilities with respect to their sensitivity to detect drug-induced QTc prolongation (ΔQTc). Little is known about the consistency of quantitative ΔQTc predictions that are relevant for translation to humans.

Experimental approach: We derived typical ΔQTc predictions at therapeutic exposure (ΔQTcTHER ) with 95% confidence intervals (95%CI) for 3 Kv 11.1 (hERG) channel blockers (moxifloxacin, dofetilide and sotalol) from a total of 14 CVS with variable designs in the conscious dog. Population pharmacokinetic-pharmacodynamic (PKPD) analysis of each study was followed by a meta-analysis (pooling 2-6 studies including 10-32 dogs per compound) to derive meta-predictions of typical ΔQTcTHER . Meta-predictions were used as a reference to evaluate the consistency of study predictions and to relate results to those found in the clinical literature.

Key results: The 95%CIs of study-predicted ΔQTcTHER comprised in 13 out of 14 cases the meta-prediction. Overall inter-study variability (mean deviation from meta-prediction at upper level of therapeutic exposure) was 30% (range: 1-69%). Meta-ΔQTcTHER predictions for moxifloxacin, dofetilide and sotalol overlapped with reported clinical QTc prolongation when expressed as %-prolongation from baseline.

Conclusions and implications: Consistent exposure-ΔQTc predictions were obtained from single preclinical dog studies of highly variable designs by systematic PKPD analysis, which is suitable for translational purposes. The good preclinical-clinical pharmacodynamic correlations obtained suggest that such an analysis should be more routinely applied to increase the informative and predictive value of results obtained from animal experiments.

Publication types

  • Meta-Analysis
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Arrhythmias, Cardiac / chemically induced
  • Arrhythmias, Cardiac / physiopathology
  • Brugada Syndrome
  • Cardiac Conduction System Disease
  • Dogs
  • Dose-Response Relationship, Drug
  • Drug Evaluation, Preclinical / methods
  • Drug Evaluation, Preclinical / standards
  • Female
  • Fluoroquinolones / adverse effects
  • Fluoroquinolones / pharmacology
  • Heart Conduction System / abnormalities
  • Heart Conduction System / physiopathology
  • Heart Rate / drug effects
  • Heart Rate / physiology
  • Humans
  • Long QT Syndrome / chemically induced*
  • Long QT Syndrome / physiopathology
  • Male
  • Moxifloxacin
  • Phenethylamines / adverse effects
  • Phenethylamines / pharmacology
  • Potassium Channel Blockers / adverse effects*
  • Potassium Channel Blockers / pharmacology
  • Reproducibility of Results
  • Sotalol / adverse effects
  • Sotalol / pharmacology
  • Sulfonamides / adverse effects
  • Sulfonamides / pharmacology
  • Telemetry / methods
  • Telemetry / standards*
  • Translational Research, Biomedical / methods
  • Translational Research, Biomedical / standards*

Substances

  • Fluoroquinolones
  • Phenethylamines
  • Potassium Channel Blockers
  • Sulfonamides
  • Sotalol
  • dofetilide
  • Moxifloxacin