Clinical trials in multiple sclerosis: Methodological issues

Research output: Contribution to journalArticle

Abstract

Purpose of review: The availability of partially effective immunomodulatory and immunosuppressive treatments for relapsing multiple sclerosis (MS) opens important ethical, methodological and practical issues in the design and conduct of new clinical trials in these patients. Recent findings: The recommendation of the National Health Authorities to prioritize phase III clinical trials using placebo arm raises ethical questions. In addition, patients are reluctant to be involved in such trials. Alternative clinical trial designs will be discussed. Relapses and active lesions are accepted measures of disease activity; new/enlarging T2 lesions and/or enhancing lesions are accepted surrogate markers of disease activity in phase II clinical trials. On the contrary, there are no accepted magnetic resonance imaging (MRI) surrogate markers of disease progression and also the clinical measures to monitor the degenerative aspects of the disease are not without important limitations. New scales of impairment, disability and quality of life will be reviewed extensively. We will also focus on the value of modern and quantitative MRI techniques, which hold substantial promise as tools to estimate the extent of MS-related irreversible tissue loss. Summary: The use of an active comparator in a superior clinical-trial design is becoming an attractive option for testing the efficacy of new drugs in relapsing MS. At present there are no fully reliable and sensitive clinical markers of the accumulation of irreversible tissue damage in MS. Although additional extensive application in longitudinal studies is needed, modern MRI techniques are promising tools to 1 monitor the neurodegenerative aspects of MS.

Original languageEnglish
Pages (from-to)245-252
Number of pages8
JournalCurrent Opinion in Neurology
Volume18
Issue number3
Publication statusPublished - Jun 2005

Fingerprint

Multiple Sclerosis
Clinical Trials
Biomarkers
Magnetic Resonance Imaging
Phase III Clinical Trials
Phase II Clinical Trials
Immunosuppressive Agents
Longitudinal Studies
Disease Progression
Placebos
Quality of Life
Recurrence
Health
Pharmaceutical Preparations
Therapeutics

Keywords

  • Clinical measures
  • Clinical trials
  • Disability
  • Magnetic resonance imaging
  • Multiple sclerosis

ASJC Scopus subject areas

  • Neuroscience(all)

Cite this

Clinical trials in multiple sclerosis : Methodological issues. / Comi, Giancarlo; Filippi, Massimo.

In: Current Opinion in Neurology, Vol. 18, No. 3, 06.2005, p. 245-252.

Research output: Contribution to journalArticle

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