期刊
MULTIPLE SCLEROSIS JOURNAL
卷 22, 期 7, 页码 862-872出版社
SAGE PUBLICATIONS LTD
DOI: 10.1177/1352458515620934
关键词
Neuromyelitis optica; trial design; ethics; MEDI-551; anti-CD19 monoclonal antibody
资金
- MedImmune
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 fare severe diseases when there is no proven therapy and no established clinical development path.
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