Challenges and opportunities in designing clinical trials for neuromyelitis optica

Brian G. Weinshenker, Gerard Barron, Jacinta M. Behne, Jeffery L. Bennett, Peter S. Chin, Bruce A C Cree, Jerome De Seze, Armando Flor, Kazuo Fujihara, Benjamin Greenberg, Sayumi Higashi, William Holt, Omar Khan, Volker Knappertz, Michael Levy, Angela T. Melia, Jacqueline Palace, Terry J. Smith, Maria Pia Sormani, Katja Van Herle & 6 others Susan Vanmeter, Pablo Villoslada, Marc K. Walton, Warren Wasiewski, Dean M. Wingerchuk, Michael R. Yeaman

Research output: Contribution to journalArticle

19 Citations (Scopus)

Abstract

Current management of neuromyelitis optica (NMO) is noncurative and only partially effective. Immunosuppressive or immunomodulatory agents are the mainstays of maintenance treatment. Safer, better-tolerated, and proven effective treatments are needed. The perceived rarity of NMO has impeded clinical trials for this disease. However, a diagnostic biomarker and recognition of a wider spectrum of NMO presentations has expanded the patient population from which study candidates might be recruited. Emerging insights into the pathogenesis of NMO have provided rationale for exploring new therapeutic targets. Academic, pharmaceutical, and regulatory communities are increasingly interested in meeting the unmet needs of patients with NMO. Clinical trials powered to yield unambiguous outcomes and designed to facilitate rapid evaluation of an expanding pipeline of experimental agents are needed. NMO-related disability occurs incrementally as a result of attacks; thus, limiting attack frequency and severity are critical treatment goals. Yet, the severity of NMO and perception that currently available agents are effective pose challenges to study design. We propose strategies for NMO clinical trials to evaluate agents targeting recovery from acute attacks and prevention of relapses, the 2 primary goals of NMO treatment. Aligning the interests of all stakeholders is an essential step to this end.

Original languageEnglish (US)
Pages (from-to)1805-1815
Number of pages11
JournalNeurology
Volume84
Issue number17
DOIs
StatePublished - Apr 28 2015

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Neuromyelitis Optica
Clinical Trials
Therapeutics
Immunosuppressive Agents
Secondary Prevention
Biomarkers

ASJC Scopus subject areas

  • Clinical Neurology

Cite this

Weinshenker, B. G., Barron, G., Behne, J. M., Bennett, J. L., Chin, P. S., Cree, B. A. C., ... Yeaman, M. R. (2015). Challenges and opportunities in designing clinical trials for neuromyelitis optica. Neurology, 84(17), 1805-1815. https://doi.org/10.1212/WNL.0000000000001520

Challenges and opportunities in designing clinical trials for neuromyelitis optica. / Weinshenker, Brian G.; Barron, Gerard; Behne, Jacinta M.; Bennett, Jeffery L.; Chin, Peter S.; Cree, Bruce A C; De Seze, Jerome; Flor, Armando; Fujihara, Kazuo; Greenberg, Benjamin; Higashi, Sayumi; Holt, William; Khan, Omar; Knappertz, Volker; Levy, Michael; Melia, Angela T.; Palace, Jacqueline; Smith, Terry J.; Sormani, Maria Pia; Van Herle, Katja; Vanmeter, Susan; Villoslada, Pablo; Walton, Marc K.; Wasiewski, Warren; Wingerchuk, Dean M.; Yeaman, Michael R.

In: Neurology, Vol. 84, No. 17, 28.04.2015, p. 1805-1815.

Research output: Contribution to journalArticle

Weinshenker, BG, Barron, G, Behne, JM, Bennett, JL, Chin, PS, Cree, BAC, De Seze, J, Flor, A, Fujihara, K, Greenberg, B, Higashi, S, Holt, W, Khan, O, Knappertz, V, Levy, M, Melia, AT, Palace, J, Smith, TJ, Sormani, MP, Van Herle, K, Vanmeter, S, Villoslada, P, Walton, MK, Wasiewski, W, Wingerchuk, DM & Yeaman, MR 2015, 'Challenges and opportunities in designing clinical trials for neuromyelitis optica', Neurology, vol. 84, no. 17, pp. 1805-1815. https://doi.org/10.1212/WNL.0000000000001520
Weinshenker BG, Barron G, Behne JM, Bennett JL, Chin PS, Cree BAC et al. Challenges and opportunities in designing clinical trials for neuromyelitis optica. Neurology. 2015 Apr 28;84(17):1805-1815. https://doi.org/10.1212/WNL.0000000000001520
Weinshenker, Brian G. ; Barron, Gerard ; Behne, Jacinta M. ; Bennett, Jeffery L. ; Chin, Peter S. ; Cree, Bruce A C ; De Seze, Jerome ; Flor, Armando ; Fujihara, Kazuo ; Greenberg, Benjamin ; Higashi, Sayumi ; Holt, William ; Khan, Omar ; Knappertz, Volker ; Levy, Michael ; Melia, Angela T. ; Palace, Jacqueline ; Smith, Terry J. ; Sormani, Maria Pia ; Van Herle, Katja ; Vanmeter, Susan ; Villoslada, Pablo ; Walton, Marc K. ; Wasiewski, Warren ; Wingerchuk, Dean M. ; Yeaman, Michael R. / Challenges and opportunities in designing clinical trials for neuromyelitis optica. In: Neurology. 2015 ; Vol. 84, No. 17. pp. 1805-1815.
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