Model-based decision making in early clinical development: minimizing the impact of a blood pressure adverse event

AAPS J. 2009 Mar;11(1):99-108. doi: 10.1208/s12248-009-9083-6. Epub 2009 Feb 6.

Abstract

We describe how modeling and simulation guided program decisions following a randomized placebo-controlled single-rising oral dose first-in-man trial of compound A where an undesired transient blood pressure (BP) elevation occurred in fasted healthy young adult males. We proposed a lumped-parameter pharmacokinetic-pharmacodynamic (PK/PD) model that captured important aspects of the BP homeostasis mechanism. Four conceptual units characterized the feedback PD model: a sinusoidal BP set point, an effect compartment, a linear effect model, and a system response. To explore approaches for minimizing the BP increase, we coupled the PD model to a modified PK model to guide oral controlled-release (CR) development. The proposed PK/PD model captured the central tendency of the observed data. The simulated BP response obtained with theoretical release rate profiles suggested some amelioration of the peak BP response with CR. This triggered subsequent CR formulation development; we used actual dissolution data from these candidate CR formulations in the PK/PD model to confirm a potential benefit in the peak BP response. Though this paradigm has yet to be tested in the clinic, our model-based approach provided a common rational framework to more fully utilize the limited available information for advancing the program.

MeSH terms

  • Administration, Oral
  • Animals
  • Biological Availability
  • Circadian Rhythm
  • Clinical Trials, Phase I as Topic / statistics & numerical data*
  • Colon
  • Computer Simulation*
  • Decision Making*
  • Delayed-Action Preparations
  • Dogs
  • Dose-Response Relationship, Drug
  • Drug Evaluation, Preclinical
  • Drugs, Investigational / administration & dosage
  • Drugs, Investigational / adverse effects
  • Drugs, Investigational / pharmacokinetics*
  • Gastrointestinal Contents / chemistry
  • Humans
  • Hypertension / chemically induced*
  • Hypertension / prevention & control
  • Ileum
  • Intestinal Absorption
  • Jejunum
  • Male
  • Models, Biological*
  • Randomized Controlled Trials as Topic / statistics & numerical data*
  • Tablets
  • Young Adult

Substances

  • Delayed-Action Preparations
  • Drugs, Investigational
  • Tablets