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 language | English (US) |
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Pages (from-to) | 862-872 |
Number of pages | 11 |
Journal | Multiple Sclerosis |
Volume | 22 |
Issue number | 7 |
DOIs | |
State | Published - Jun 1 2016 |
Keywords
- MEDI-551
- Neuromyelitis optica
- anti-CD19 monoclonal antibody
- ethics
- trial design
ASJC Scopus subject areas
- Neurology
- Clinical Neurology