Placebo-controlled study in neuromyelitis optica - Ethical and design considerations

Bruce A.C. Cree, Jeffrey L. Bennett, Mark Sheehan, Jeffrey Cohen, Hans Peter Hartung, Orhan Aktas, Ho Jin Kim, Friedemann Paul, Sean Pittock, Brian Weinshenker, Dean Wingerchuk, Kazuo Fujihara, Gary Cutter, Kaushik Patra, Armando Flor, Gerard Barron, Soraya Madani, John N. Ratchford, Eliezer Katz

Research output: Contribution to journalArticle

29 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)862-872
Number of pages11
JournalMultiple Sclerosis
Volume22
Issue number7
DOIs
StatePublished - Jun 1 2016

Keywords

  • MEDI-551
  • Neuromyelitis optica
  • anti-CD19 monoclonal antibody
  • ethics
  • trial design

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology

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    Cree, B. A. C., Bennett, J. L., Sheehan, M., Cohen, J., Hartung, H. P., Aktas, O., Kim, H. J., Paul, F., Pittock, S., Weinshenker, B., Wingerchuk, D., Fujihara, K., Cutter, G., Patra, K., Flor, A., Barron, G., Madani, S., Ratchford, J. N., & Katz, E. (2016). Placebo-controlled study in neuromyelitis optica - Ethical and design considerations. Multiple Sclerosis, 22(7), 862-872. https://doi.org/10.1177/1352458515620934