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| Item Type: | Article |
|---|---|
| Title: | Placebo-controlled study in neuromyelitis optica : ethical and design considerations |
| Creators Name: | 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. 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 |
| 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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