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Placebo-controlled study in neuromyelitis optica : ethical and design considerations

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Item Type:Article
Title:Placebo-controlled study in neuromyelitis optica : ethical and design considerations
Creators Name:Cree, B.A.C. and Bennett, J.L. and Sheehan, M. and Cohen, J. and Hartung, H.P. and Aktas, O. and Kim, H.J. and Paul, F. and Pittock, S. and Weinshenker, B. and Wingerchuk, D. and Fujihara, K. and Cutter, G. and Patra, K. and Flor, A. and Barron, G. and Madani, S. and Ratchford, J.N. and Katz, E.
Abstract:BACKGROUND: To date, no treatment for neuromyelitis optica (NMO) has been granted regulatory approval, and no controlled clinical studies have been reported. OBJECTIVE: To design a placebo-controlled study in NMO that appropriately balances patient safety and clinical-scientific integrity. METHODS: We assessed the "standard of care" for NMO to establish the ethical framework for a placebo-controlled trial. We implemented measures that balance the need for scientific robustness while mitigating the risks associated with a placebo-controlled study. The medical or scientific community, patient organizations, and regulatory authorities were engaged early in discussions on this placebo-controlled study, and their input contributed to the final study design. RESULTS: The N-MOmentum study (NCT02200770) is a clinical trial that randomizes NMO patients to receive MEDI-551, a monoclonal antibody that depletes CD19+ B-cells, or placebo. The study design has received regulatory, ethical, clinical, and patient approval in over 100 clinical sites in more than 20 countries worldwide. CONCLUSION: The approach we took in the design of the N-MOmentum trial might serve as a roadmap for other rare severe diseases when there is no proven therapy and no established clinical development path.
Keywords:Neuromyelitis Optica, Trial Design, Ethics, MEDI-551, Anti-CD19 Monoclonal Antibody
Source:Multiple Sclerosis Journal
ISSN:1352-4585
Publisher:Sage Publications (U.K.)
Volume:22
Number:7
Page Range:862-872
Date:June 2016
Official Publication:https://doi.org/10.1177/1352458515620934
PubMed:View item in PubMed

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