Title: Design strategies in multiple sclerosis clinical trials
Abstract: Annals of NeurologyVolume 36, Issue S1 p. S108-S112 Treatment Strategies Design strategies in multiple sclerosis clinical trials Dr George W. Ellison MD, Corresponding Author Dr George W. Ellison MD Department of Neurology School of Medicine, University of California, Los Angeles, CA10833 Le Conte Ave, Los Angeles, CA 90024-1916Search for more papers by this authorLawrence W. Myers MD, Lawrence W. Myers MD Department of Neurology School of Medicine, University of California, Los Angeles, CASearch for more papers by this authorBarbara D. Leake PhD, Barbara D. Leake PhD Department of Biomathematics, School of Medicine, University of California, Los Angeles, CASearch for more papers by this authorM. Ray Mickey PhD, M. Ray Mickey PhD Department of Biomathematics, School of Medicine, University of California, Los Angeles, CASearch for more papers by this authorDershin Ke MD, Dershin Ke MD Neurology Service, Wadsworth Veterans Administration Medical Center, Los Angeles, CASearch for more papers by this authorKarl Syndulko PhD, Karl Syndulko PhD Department of Neurology School of Medicine, University of California, Los Angeles, CA Neurology Service, Wadsworth Veterans Administration Medical Center, Los Angeles, CASearch for more papers by this authorWallace W. Tourtellotte MD, PhD, Wallace W. Tourtellotte MD, PhD Department of Neurology School of Medicine, University of California, Los Angeles, CA Neurology Service, Wadsworth Veterans Administration Medical Center, Los Angeles, CASearch for more papers by this author Dr George W. Ellison MD, Corresponding Author Dr George W. Ellison MD Department of Neurology School of Medicine, University of California, Los Angeles, CA10833 Le Conte Ave, Los Angeles, CA 90024-1916Search for more papers by this authorLawrence W. Myers MD, Lawrence W. Myers MD Department of Neurology School of Medicine, University of California, Los Angeles, CASearch for more papers by this authorBarbara D. Leake PhD, Barbara D. Leake PhD Department of Biomathematics, School of Medicine, University of California, Los Angeles, CASearch for more papers by this authorM. Ray Mickey PhD, M. Ray Mickey PhD Department of Biomathematics, School of Medicine, University of California, Los Angeles, CASearch for more papers by this authorDershin Ke MD, Dershin Ke MD Neurology Service, Wadsworth Veterans Administration Medical Center, Los Angeles, CASearch for more papers by this authorKarl Syndulko PhD, Karl Syndulko PhD Department of Neurology School of Medicine, University of California, Los Angeles, CA Neurology Service, Wadsworth Veterans Administration Medical Center, Los Angeles, CASearch for more papers by this authorWallace W. Tourtellotte MD, PhD, Wallace W. Tourtellotte MD, PhD Department of Neurology School of Medicine, University of California, Los Angeles, CA Neurology Service, Wadsworth Veterans Administration Medical Center, Los Angeles, CASearch for more papers by this author First published: 1994 https://doi.org/10.1002/ana.410360725Citations: 27AboutPDF ToolsRequest permissionExport citationAdd to favoritesTrack citation ShareShare Give accessShare full text accessShare full-text accessPlease review our Terms and Conditions of Use and check box below to share full-text version of article.I have read and accept the Wiley Online Library Terms and Conditions of UseShareable LinkUse the link below to share a full-text version of this article with your friends and colleagues. Learn more.Copy URL Abstract After analyzing our natural history data on the course of multiple sclerosis (MS) in more than 500 patients followed for 20 years and our experience in several therapeutic trials, we concluded that a phase III (full) trial for efficacy should have certain properties. For a power of 0.8, α of 0.05, and attrition rate of 10% per year, we think the trial should have a minimum sample size of 130 (65 in each arm; placebo versus active) if the design is based upon the proportion of subjects worsening by clinical measures. No stratification by entry Extended Disability Status Scale score is needed if worsening is defined as a change of 1.0 units (2 to 0.5 steps) maintained for 90 days for an entry score of 1 to 5.0 units; or 0.5 units (1 to 0.5 steps) if the entry score is 5.5 to 7 units. We need not stratify by course (relapsing–remitting versus relapsing–progressive) but are less certain about progression from the onset. No run-in period is required to define "activity." Minimum time for treatment is 3 years. We review the justification for our conclusions; modifications in sample size that are necessary if survival analysis is used; impact of the interferon-β trial (future trials will have an "active" control); and alternative strategies possible if magnetic resonance imaging serves as the primary outcome. Citing Literature Volume36, IssueS1Supplement: Multiple Sclerosis: Approaches to Management1994Pages S108-S112 RelatedInformation
Publication Year: 1994
Publication Date: 1994-01-01
Language: en
Type: article
Indexed In: ['crossref', 'pubmed']
Access and Citation
Cited By Count: 40
AI Researcher Chatbot
Get quick answers to your questions about the article from our AI researcher chatbot