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BackgroundTo date, no treatment for neuromyelitis optica (NMO) has been granted regulatory approval, and no controlled clinical studies have been reported.ObjectiveTo design a placebo-controlled study in NMO that appropriately balances patient safety and clinical-scientific integrity.MethodsWe 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.ResultsThe 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.ConclusionThe 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 publication

DOI

10.1177/1352458515620934

Type

Journal article

Journal

Multiple sclerosis (Houndmills, Basingstoke, England)

Publication Date

06/2016

Volume

22

Pages

862 - 872

Addresses

Multiple Sclerosis Center, Department of Neurology, University of California San Francisco, San Francisco, CA, USA bruce.cree@ucsf.edu.

Keywords

Humans, Neuromyelitis Optica, Immunologic Factors, Treatment Outcome, Research Design, Time Factors, Ethics, Research, Antibodies, Monoclonal, Humanized