Amyotrophic lateral sclerosis: a consensus viewpoint on designing and implementing a clinical trial.
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.
Leigh
P Nigel
PN
Department of Neurology, Institute of Psychiatry, London, UK. spgtpni@iop.kcl.ac.uk
Swash
Michael
M
Iwasaki
Yasuo
Y
Ludolph
Albert
A
Meininger
Vincent
V
Miller
Robert G
RG
Mitsumoto
Hiroshi
H
Shaw
Pamela
P
Tashiro
Kunio
K
Van Den Berg
Leonard
L
D000690:Amyotrophic Lateral Sclerosis / Q000175:diagnosis / Q000453:epidemiology / Q000503:physiopathology / Q000628:therapy; D000818:Animals; D002986:Clinical Trials as Topic / Q000379:methods* / Q000706:statistics & numerical data; D003245:Consensus Statements as Topic; D006801:Humans; D010082:Oxepins / Q000627:therapeutic use; D019782:Riluzole / Q000627:therapeutic use
D010082:Oxepins; C111178:dibenzo(b,f)oxepin-10-ylmethyl-methyl-prop-2-ynyl-amine; D019782:Riluzole