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Proof of concept studies for tissue-protective agents in multiple sclerosis

University of Rochester Department of Neurology, Rochester, NY, USA lahar_mehta{at}urmc.rochester.edu

SR Schwid

University of Rochester Department of Neurology, Rochester, NY, USA

DL Arnold

Montreal Neurological Institute, Montreal, QC, USA

GR Cutter

University of Alabama-Birmingham, Department of Biostatistics, Birmingham, AL, USA

S Aradhye

Novartis Pharmaceuticals

LJ Balcer

University of Pennsylvania Department of Neurology and Epidemiology, Philadelphia, PA, USA

PA Calabresi

Johns Hopkins University Department of Neurology, Baltimore, MD, USA

JA Cohen

Cleveland Clinic, Cleveland, OH, USA

PE Cole

Eisai Medical Research, Inc

R Glanzman

Novartis Pharmaceuticals

S Goelz

Biogen Idec, Inc

M Inglese

New York University Department of Radiology and Neurology, New York, NY, USA

R Kapoor

National Hospital for Neurology and Neurosurgery, Queen Square, London, UK

L Kappos

University Hospital, Basel, Switzerland

R Kreitman

Teva Pharmaceutical Industries, Ltd

FD Lublin

Mount Sinai School of Medicine, New York, NY, USA

A Mann

Eisai Medical Research, Inc

RA Marrie

University of Manitoba, Winnipeg, MB, Canada

P O'Looney

16 National Multiple Sclerosis Society, New York, NY, USA

CH Polman

17 Vrije Universiteit Medical Center, Amsterdam, The Netherlands

BM Ravina

University of Rochester Department of Neurology, Rochester, NY, USA

SC Reingold

18 Scientific and Clinical Review Associates, LLC, New York, NY, USA

JR Richert

16 National Multiple Sclerosis Society, New York, NY, USA

AW Sandrock

Biogen Idec, Inc

E Waubant

19 University of California – San Francisco, Department of Neurology, San Francisco, CA, USA

Background

There is considerable interest in tissue-protective treatments for multiple sclerosis (MS).

Methods and Objectives

We convened a group of MS clinical trialists and related researchers to discuss designs for proof of concept studies utilizing currently available data and assessment methods.

Results

Our favored design was a randomized, double-blind, parallel-group study of active treatment versus placebo focusing on changes in brain volume from a post-baseline scan (3–6 months after starting treatment) to the final visit 1 year later. Study designs aimed at reducing residual deficits following acute exacerbations are less straightforward, depending greatly on the anticipated rapidity of treatment effect onset.

Conclusions

The next step would be to perform one or more studies of potential tissue-protective agents with these designs in mind, creating the longitudinal data necessary to refine endpoint selection, eligibility criteria, and sample size estimates for future trials.

Key Words: tissue protection • clinical trial designs • multiple sclerosis • outcome measurement

Multiple Sclerosis, Vol. 15, No. 5, 542-546 (2009)
DOI: 10.1177/1352458508101939


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