Proof of concept studies for tissue-protective agents in multiple sclerosis

Lahar R. Mehta; S. R. Schwid; D. L. Arnold; G. R. Cutter; S. Aradhye; L. J. Balcer; P. A. Calabresi; J. A. Cohen; P. E. Cole; R. Glanzman; S. Goelz; M. Inglese; R. Kapoor; L. Kappos; R. Kreitman; F. D. Lublin; A. Mann; R. A. Marrie; P. O'Looney; C. H. Polman; B. M. Ravina; S. C. Reingold; J. R. Richert; A. W. Sandrock; E. Waubant

doi:10.1177/1352458508101939

Proof of concept studies for tissue-protective agents in multiple sclerosis

Lahar R. Mehta, S. R. Schwid, D. L. Arnold, G. R. Cutter, S. Aradhye, L. J. Balcer, P. A. Calabresi, J. A. Cohen, P. E. Cole, R. Glanzman, S. Goelz, M. Inglese, R. Kapoor, L. Kappos, R. Kreitman, F. D. Lublin, A. Mann, R. A. Marrie, P. O'Looney, C. H. PolmanB. M. Ravina, S. C. Reingold, J. R. Richert, A. W. Sandrock, E. Waubant

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

7 Scopus citations

Abstract

Background: There is considerable interest in tissue-protective treatments for multiple sclerosis (MS). Methods and Objectives: We convened a group of MS clinical trialists and related researchers to discuss designs for proof of concept studies utilizing currently available data and assessment methods. Results: Our favored design was a randomized, double-blind, parallel-group study of active treatment versus placebo focusing on changes in brain volume from a post-baseline scan (3-6 months after starting treatment) to the final visit 1 year later. Study designs aimed at reducing residual deficits following acute exacerbations are less straightforward, depending greatly on the anticipated rapidity of treatment effect onset. Conclusions: The next step would be to perform one or more studies of potential tissue-protective agents with these designs in mind, creating the longitudinal data necessary to refine endpoint selection, eligibility criteria, and sample size estimates for future trials.

Original language	English
Pages (from-to)	542-546
Number of pages	5
Journal	Multiple Sclerosis Journal
Volume	15
Issue number	5
DOIs	https://doi.org/10.1177/1352458508101939
State	Published - 2009

Keywords

Clinical trial designs
Multiple sclerosis
Outcome measurement
Tissue protection

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Mehta, L. R., Schwid, S. R., Arnold, D. L., Cutter, G. R., Aradhye, S., Balcer, L. J., Calabresi, P. A., Cohen, J. A., Cole, P. E., Glanzman, R., Goelz, S., Inglese, M., Kapoor, R., Kappos, L., Kreitman, R., Lublin, F. D., Mann, A., Marrie, R. A., O'Looney, P., ... Waubant, E. (2009). Proof of concept studies for tissue-protective agents in multiple sclerosis. Multiple Sclerosis Journal, 15(5), 542-546. https://doi.org/10.1177/1352458508101939

@article{509e2be12777440daa07e205f696017b,

title = "Proof of concept studies for tissue-protective agents in multiple sclerosis",

abstract = "Background: There is considerable interest in tissue-protective treatments for multiple sclerosis (MS). Methods and Objectives: We convened a group of MS clinical trialists and related researchers to discuss designs for proof of concept studies utilizing currently available data and assessment methods. Results: Our favored design was a randomized, double-blind, parallel-group study of active treatment versus placebo focusing on changes in brain volume from a post-baseline scan (3-6 months after starting treatment) to the final visit 1 year later. Study designs aimed at reducing residual deficits following acute exacerbations are less straightforward, depending greatly on the anticipated rapidity of treatment effect onset. Conclusions: The next step would be to perform one or more studies of potential tissue-protective agents with these designs in mind, creating the longitudinal data necessary to refine endpoint selection, eligibility criteria, and sample size estimates for future trials.",

keywords = "Clinical trial designs, Multiple sclerosis, Outcome measurement, Tissue protection",

author = "Mehta, {Lahar R.} and Schwid, {S. R.} and Arnold, {D. L.} and Cutter, {G. R.} and S. Aradhye and Balcer, {L. J.} and Calabresi, {P. A.} and Cohen, {J. A.} and Cole, {P. E.} and R. Glanzman and S. Goelz and M. Inglese and R. Kapoor and L. Kappos and R. Kreitman and Lublin, {F. D.} and A. Mann and Marrie, {R. A.} and P. O'Looney and Polman, {C. H.} and Ravina, {B. M.} and Reingold, {S. C.} and Richert, {J. R.} and Sandrock, {A. W.} and E. Waubant",

year = "2009",

doi = "10.1177/1352458508101939",

language = "English",

volume = "15",

pages = "542--546",

journal = "Multiple Sclerosis Journal",

issn = "1352-4585",

publisher = "SAGE Publications Ltd",

number = "5",

}

Mehta, LR, Schwid, SR, Arnold, DL, Cutter, GR, Aradhye, S, Balcer, LJ, Calabresi, PA, Cohen, JA, Cole, PE, Glanzman, R, Goelz, S, Inglese, M, Kapoor, R, Kappos, L, Kreitman, R, Lublin, FD, Mann, A, Marrie, RA, O'Looney, P, Polman, CH, Ravina, BM, Reingold, SC, Richert, JR, Sandrock, AW & Waubant, E 2009, 'Proof of concept studies for tissue-protective agents in multiple sclerosis', Multiple Sclerosis Journal, vol. 15, no. 5, pp. 542-546. https://doi.org/10.1177/1352458508101939

TY - JOUR

T1 - Proof of concept studies for tissue-protective agents in multiple sclerosis

AU - Mehta, Lahar R.

AU - Schwid, S. R.

AU - Arnold, D. L.

AU - Cutter, G. R.

AU - Aradhye, S.

AU - Balcer, L. J.

AU - Calabresi, P. A.

AU - Cohen, J. A.

AU - Cole, P. E.

AU - Glanzman, R.

AU - Goelz, S.

AU - Inglese, M.

AU - Kapoor, R.

AU - Kappos, L.

AU - Kreitman, R.

AU - Lublin, F. D.

AU - Mann, A.

AU - Marrie, R. A.

AU - O'Looney, P.

AU - Polman, C. H.

AU - Ravina, B. M.

AU - Reingold, S. C.

AU - Richert, J. R.

AU - Sandrock, A. W.

AU - Waubant, E.

PY - 2009

Y1 - 2009

N2 - Background: There is considerable interest in tissue-protective treatments for multiple sclerosis (MS). Methods and Objectives: We convened a group of MS clinical trialists and related researchers to discuss designs for proof of concept studies utilizing currently available data and assessment methods. Results: Our favored design was a randomized, double-blind, parallel-group study of active treatment versus placebo focusing on changes in brain volume from a post-baseline scan (3-6 months after starting treatment) to the final visit 1 year later. Study designs aimed at reducing residual deficits following acute exacerbations are less straightforward, depending greatly on the anticipated rapidity of treatment effect onset. Conclusions: The next step would be to perform one or more studies of potential tissue-protective agents with these designs in mind, creating the longitudinal data necessary to refine endpoint selection, eligibility criteria, and sample size estimates for future trials.

AB - Background: There is considerable interest in tissue-protective treatments for multiple sclerosis (MS). Methods and Objectives: We convened a group of MS clinical trialists and related researchers to discuss designs for proof of concept studies utilizing currently available data and assessment methods. Results: Our favored design was a randomized, double-blind, parallel-group study of active treatment versus placebo focusing on changes in brain volume from a post-baseline scan (3-6 months after starting treatment) to the final visit 1 year later. Study designs aimed at reducing residual deficits following acute exacerbations are less straightforward, depending greatly on the anticipated rapidity of treatment effect onset. Conclusions: The next step would be to perform one or more studies of potential tissue-protective agents with these designs in mind, creating the longitudinal data necessary to refine endpoint selection, eligibility criteria, and sample size estimates for future trials.

KW - Clinical trial designs

KW - Multiple sclerosis

KW - Outcome measurement

KW - Tissue protection

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U2 - 10.1177/1352458508101939

DO - 10.1177/1352458508101939

M3 - Review article

C2 - 19389749

AN - SCOPUS:65349186498

SN - 1352-4585

VL - 15

SP - 542

EP - 546

JO - Multiple Sclerosis Journal

JF - Multiple Sclerosis Journal

IS - 5

ER -

Proof of concept studies for tissue-protective agents in multiple sclerosis

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Keywords

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