Amyotrophic lateral sclerosis: a consensus viewpoint on designing and implementing a clinical trial

Amyotroph Lateral Scler Other Motor Neuron Disord. 2004 Jun;5(2):84-98. doi: 10.1080/14660820410020187.

Abstract

In November 2002, an advisory board meeting was convened by Novartis Pharma to provide recommendations and rationale for clinical trials designed to evaluate new treatments, such as TCH346, for amyotrophic lateral sclerosis (ALS). In terms of selecting appropriate outcome measures, the panel recommended the use of the ALS Functional Rating Scale (ALSFRS-R) to measure primary endpoints. A review of other key issues in this area including regional variations in the epidemiology, diagnosis and management of ALS, defining patient populations and doses of trial medication, and accommodating the likelihood of co-medication with pre-existing treatment in trial design, are discussed.

Publication types

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

MeSH terms

  • Amyotrophic Lateral Sclerosis* / diagnosis
  • Amyotrophic Lateral Sclerosis* / epidemiology
  • Amyotrophic Lateral Sclerosis* / physiopathology
  • Amyotrophic Lateral Sclerosis* / therapy
  • Animals
  • Clinical Trials as Topic / methods*
  • Clinical Trials as Topic / statistics & numerical data
  • Consensus Development Conferences as Topic*
  • Humans
  • Oxepins / therapeutic use
  • Riluzole / therapeutic use

Substances

  • Oxepins
  • dibenzo(b,f)oxepin-10-ylmethyl-methyl-prop-2-ynyl-amine
  • Riluzole