Toward more efficient clinical trials for amyotrophic lateral sclerosis

Merit E. Cudkowicz; Jon Katz; Dan H. Moore; Gilmore O'Neill; Jonathan D. Glass; Hiroshi Mitsumoto; Stanley Appel; Bernard Ravina; Karl Kieburtz; Ira Shoulson; Petra Kaufmann; Jaffar Khan; Ericka Simpson; Jeremy Shefner; Bruce Levin; Valerie Cwik; David Schoenfeld; Swati Aggarwal; Michael P. McDermott; Robert G. Miller

doi:10.3109/17482960903358865

Toward more efficient clinical trials for amyotrophic lateral sclerosis

Merit E. Cudkowicz, Jon Katz, Dan H. Moore, Gilmore O'Neill, Jonathan D. Glass, Hiroshi Mitsumoto, Stanley Appel, Bernard Ravina, Karl Kieburtz, Ira Shoulson, Petra Kaufmann, Jaffar Khan, Ericka Simpson, Jeremy Shefner, Bruce Levin, Valerie Cwik, David Schoenfeld, Swati Aggarwal, Michael P. McDermott, Robert G. Miller

Research output: Contribution to journal › Review article › peer-review

65 Scopus citations

Abstract

More than 30 phase II or III clinical trials have been carried out in amyotrophic lateral sclerosis (ALS). Only riluzole, however, has been shown to extend survival and/or time to tracheostomy. Many early ALS trials lacked solid pharmacodynamic and pharmacokinetic data for the treatment being tested, challenging the interpretation of the efficacy and pathway relevance. Understanding of the genetics and pathophysiology of ALS has improved considerably in the past decade, but biomarkers of disease activity remain lacking. A more efficient approach to early phase clinical trials is needed to accelerate the identification of useful agents for ALS. Here we summarize our current thinking about phase II design options and the potential benefits of a clinical trial network for phase II trials in ALS.

Original language	English (US)
Pages (from-to)	259-265
Number of pages	7
Journal	Amyotrophic Lateral Sclerosis
Volume	11
Issue number	3
DOIs	https://doi.org/10.3109/17482960903358865
State	Published - 2010

Keywords

Amyotrophic lateral sclerosis
Network
Phase II
Therapy development

ASJC Scopus subject areas

Clinical Neurology
Neurology

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Cudkowicz, M. E., Katz, J., Moore, D. H., O'Neill, G., Glass, J. D., Mitsumoto, H., Appel, S., Ravina, B., Kieburtz, K., Shoulson, I., Kaufmann, P., Khan, J., Simpson, E., Shefner, J., Levin, B., Cwik, V., Schoenfeld, D., Aggarwal, S., McDermott, M. P., & Miller, R. G. (2010). Toward more efficient clinical trials for amyotrophic lateral sclerosis. Amyotrophic Lateral Sclerosis, 11(3), 259-265. https://doi.org/10.3109/17482960903358865

Cudkowicz, ME, Katz, J, Moore, DH, O'Neill, G, Glass, JD, Mitsumoto, H, Appel, S, Ravina, B, Kieburtz, K, Shoulson, I, Kaufmann, P, Khan, J, Simpson, E, Shefner, J, Levin, B, Cwik, V, Schoenfeld, D, Aggarwal, S, McDermott, MP & Miller, RG 2010, 'Toward more efficient clinical trials for amyotrophic lateral sclerosis', Amyotrophic Lateral Sclerosis, vol. 11, no. 3, pp. 259-265. https://doi.org/10.3109/17482960903358865

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abstract = "More than 30 phase II or III clinical trials have been carried out in amyotrophic lateral sclerosis (ALS). Only riluzole, however, has been shown to extend survival and/or time to tracheostomy. Many early ALS trials lacked solid pharmacodynamic and pharmacokinetic data for the treatment being tested, challenging the interpretation of the efficacy and pathway relevance. Understanding of the genetics and pathophysiology of ALS has improved considerably in the past decade, but biomarkers of disease activity remain lacking. A more efficient approach to early phase clinical trials is needed to accelerate the identification of useful agents for ALS. Here we summarize our current thinking about phase II design options and the potential benefits of a clinical trial network for phase II trials in ALS.",

keywords = "Amyotrophic lateral sclerosis, Network, Phase II, Therapy development",

author = "Cudkowicz, {Merit E.} and Jon Katz and Moore, {Dan H.} and Gilmore O'Neill and Glass, {Jonathan D.} and Hiroshi Mitsumoto and Stanley Appel and Bernard Ravina and Karl Kieburtz and Ira Shoulson and Petra Kaufmann and Jaffar Khan and Ericka Simpson and Jeremy Shefner and Bruce Levin and Valerie Cwik and David Schoenfeld and Swati Aggarwal and McDermott, {Michael P.} and Miller, {Robert G.}",

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AU - Mitsumoto, Hiroshi

AU - Appel, Stanley

AU - Ravina, Bernard

AU - Kieburtz, Karl

AU - Shoulson, Ira

AU - Kaufmann, Petra

AU - Khan, Jaffar

AU - Simpson, Ericka

AU - Shefner, Jeremy

AU - Levin, Bruce

AU - Cwik, Valerie

AU - Schoenfeld, David

AU - Aggarwal, Swati

AU - McDermott, Michael P.

AU - Miller, Robert G.

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AB - More than 30 phase II or III clinical trials have been carried out in amyotrophic lateral sclerosis (ALS). Only riluzole, however, has been shown to extend survival and/or time to tracheostomy. Many early ALS trials lacked solid pharmacodynamic and pharmacokinetic data for the treatment being tested, challenging the interpretation of the efficacy and pathway relevance. Understanding of the genetics and pathophysiology of ALS has improved considerably in the past decade, but biomarkers of disease activity remain lacking. A more efficient approach to early phase clinical trials is needed to accelerate the identification of useful agents for ALS. Here we summarize our current thinking about phase II design options and the potential benefits of a clinical trial network for phase II trials in ALS.

KW - Amyotrophic lateral sclerosis

KW - Network

KW - Phase II

KW - Therapy development

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Toward more efficient clinical trials for amyotrophic lateral sclerosis

Abstract

Keywords

ASJC Scopus subject areas

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