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Mult Scler. 2016 Jun;22(7):862-72. doi: 10.1177/1352458515620934. Epub 2015 Dec 14.

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

Author information

1
Multiple Sclerosis Center, Department of Neurology, University of California San Francisco, San Francisco, CA, USA bruce.cree@ucsf.edu.
2
Program in Neuroscience, Departments of Neurology and Ophthalmology, University of Colorado, Denver, CO, USA.
3
The Ethox Centre, Nuffield Department of Population Health, University of Oxford, Oxford, UK.
4
Cleveland Clinic, Cleveland, OH, USA.
5
Department of Neurology, Medical Faculty, Heinrich Heine University Düsseldorf, Berlin, Germany.
6
Research Institute and Hospital of National Cancer Center, Goyang, South Korea.
7
Charite-University Medicine Berlin, Berlin, Germany.
8
Mayo Clinic, Rochester, MN, USA.
9
Mayo Clinic, Scottsdale, AZ, USA.
10
Tohoku University, Sendai, Japan.
11
The University of Alabama at Birmingham, Birmingham, AL, USA.
12
MedImmune, Gaithersburg, MD, USA.
13
MedImmune, Cambridge, UK.

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:

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

PMID:
26666258
PMCID:
PMC4904355
DOI:
10.1177/1352458515620934
[Indexed for MEDLINE]
Free PMC Article

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