Recommendations for myasthenia gravis clinical trials

Michael Benatar; Donald B. Sanders; Ted M. Burns; Gary R. Cutter; Jeffrey T. Guptill; Fulvio Baggi; Henry J. Kaminski; Renato Mantegazza; Matthew N. Meriggioli; Joanne Quan; Gil I. Wolfe; Richard J. Barohn; Ted M. Burns; Gary R. Cutter; Jeffrey T. Guptill; James F. Howard; Henry J. Kaminski; R. Antegazza; Matthew N. Merrigioli; Steven Piantadosi; Alexis Smirnow; Gil I. Wolfe

doi:10.1002/mus.23330

Recommendations for myasthenia gravis clinical trials

Michael Benatar, Donald B. Sanders, Ted M. Burns, Gary R. Cutter, Jeffrey T. Guptill, Fulvio Baggi, Henry J. Kaminski, Renato Mantegazza, Matthew N. Meriggioli, Joanne Quan, Gil I. Wolfe, Richard J. Barohn, Ted M. Burns, Gary R. Cutter, Jeffrey T. Guptill, James F. Howard, Henry J. Kaminski, R. Antegazza, Matthew N. Merrigioli, Steven PiantadosiAlexis Smirnow, Gil I. Wolfe

Research output: Contribution to journal › Article › peer-review

90 Scopus citations

Abstract

The recommendations for clinical research standards published in 2000 by a task force of the Medical Scientific Advisory Board (MSAB) of the Myasthenia Gravis Foundation of America (MGFA) were largely successful in introducing greater uniformity in the recording and reporting of MG clinical trials. Recognizing that changes in clinical trial design and implementation may increase the likelihood that new therapies are developed for MG, the MGFA MSAB Task Force here presents updated recommendations for the design and implementation of clinical trials in MG, including (a) the use of a quantitative measure, such as the MG-Composite, that is weighted for clinical significance and incorporates patient reported outcomes; (b) consideration of nontrial strategies; and (c) development of biomarkers that support mechanistic studies of pharmacotherapies. The hope is that these updated task force recommendations will expedite the development and acceptance of more effective and less noxious therapies for MG.

Original language	English (US)
Pages (from-to)	909-917
Number of pages	9
Journal	Muscle and Nerve
Volume	45
Issue number	6
DOIs	https://doi.org/10.1002/mus.23330
State	Published - Jun 2012
Externally published	Yes

Keywords

All clinical trials
Clinical trials methodology/study design
Myasthenia
Outcome measures
Quality of life

ASJC Scopus subject areas

Physiology
Clinical Neurology
Cellular and Molecular Neuroscience
Physiology (medical)

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10.1002/mus.23330

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Benatar, M., Sanders, D. B., Burns, T. M., Cutter, G. R., Guptill, J. T., Baggi, F., Kaminski, H. J., Mantegazza, R., Meriggioli, M. N., Quan, J., Wolfe, G. I., Barohn, R. J., Burns, T. M., Cutter, G. R., Guptill, J. T., Howard, J. F., Kaminski, H. J., Antegazza, R., Merrigioli, M. N., ... Wolfe, G. I. (2012). Recommendations for myasthenia gravis clinical trials. Muscle and Nerve, 45(6), 909-917. https://doi.org/10.1002/mus.23330

Benatar, M, Sanders, DB, Burns, TM, Cutter, GR, Guptill, JT, Baggi, F, Kaminski, HJ, Mantegazza, R, Meriggioli, MN, Quan, J, Wolfe, GI, Barohn, RJ, Burns, TM, Cutter, GR, Guptill, JT, Howard, JF, Kaminski, HJ, Antegazza, R, Merrigioli, MN, Piantadosi, S, Smirnow, A & Wolfe, GI 2012, 'Recommendations for myasthenia gravis clinical trials', Muscle and Nerve, vol. 45, no. 6, pp. 909-917. https://doi.org/10.1002/mus.23330

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abstract = "The recommendations for clinical research standards published in 2000 by a task force of the Medical Scientific Advisory Board (MSAB) of the Myasthenia Gravis Foundation of America (MGFA) were largely successful in introducing greater uniformity in the recording and reporting of MG clinical trials. Recognizing that changes in clinical trial design and implementation may increase the likelihood that new therapies are developed for MG, the MGFA MSAB Task Force here presents updated recommendations for the design and implementation of clinical trials in MG, including (a) the use of a quantitative measure, such as the MG-Composite, that is weighted for clinical significance and incorporates patient reported outcomes; (b) consideration of nontrial strategies; and (c) development of biomarkers that support mechanistic studies of pharmacotherapies. The hope is that these updated task force recommendations will expedite the development and acceptance of more effective and less noxious therapies for MG.",

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author = "Michael Benatar and Sanders, {Donald B.} and Burns, {Ted M.} and Cutter, {Gary R.} and Guptill, {Jeffrey T.} and Fulvio Baggi and Kaminski, {Henry J.} and Renato Mantegazza and Meriggioli, {Matthew N.} and Joanne Quan and Wolfe, {Gil I.} and Barohn, {Richard J.} and Burns, {Ted M.} and Cutter, {Gary R.} and Guptill, {Jeffrey T.} and Howard, {James F.} and Kaminski, {Henry J.} and R. Antegazza and Merrigioli, {Matthew N.} and Steven Piantadosi and Alexis Smirnow and Wolfe, {Gil I.}",

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Recommendations for myasthenia gravis clinical trials

Abstract

Keywords

ASJC Scopus subject areas

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