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).
  
  
  OBJECTIVE
  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.
authors.affiliation: University of Rochester Department of Neurology, Rochester, NY, USA. lahar_mehta@urmc.rochester.edu
authors.forename: L R
authors.initials: LR
authors.lastname: Mehta
authors.forename: S R
authors.initials: SR
authors.lastname: Schwid
authors.forename: D L
authors.initials: DL
authors.lastname: Arnold
authors.forename: G R
authors.initials: GR
authors.lastname: Cutter
authors.forename: S
authors.initials: S
authors.lastname: Aradhye
authors.forename: L J
authors.initials: LJ
authors.lastname: Balcer
authors.forename: P A
authors.initials: PA
authors.lastname: Calabresi
authors.forename: J A
authors.initials: JA
authors.lastname: Cohen
authors.forename: P E
authors.initials: PE
authors.lastname: Cole
authors.forename: R
authors.initials: R
authors.lastname: Glanzman
authors.forename: S
authors.initials: S
authors.lastname: Goelz
authors.forename: M
authors.initials: M
authors.lastname: Inglese
authors.forename: R
authors.initials: R
authors.lastname: Kapoor
authors.forename: L
authors.initials: L
authors.lastname: Kappos
authors.forename: R
authors.initials: R
authors.lastname: Kreitman
authors.forename: F D
authors.initials: FD
authors.lastname: Lublin
authors.forename: A
authors.initials: A
authors.lastname: Mann
authors.forename: R A
authors.initials: RA
authors.lastname: Marrie
authors.forename: P
authors.initials: P
authors.lastname: O'Looney
authors.forename: C H
authors.initials: CH
authors.lastname: Polman
authors.forename: B M
authors.initials: BM
authors.lastname: Ravina
authors.forename: S C
authors.initials: SC
authors.lastname: Reingold
authors.forename: J R
authors.initials: JR
authors.lastname: Richert
authors.forename: A W
authors.initials: AW
authors.lastname: Sandrock
authors.forename: E
authors.initials: E
authors.lastname: Waubant
mesh_terms: D006801:Humans; D009103:Multiple Sclerosis / Q000188:drug therapy* / Q000473:pathology*; D018696:Neuroprotective Agents / Q000627:therapeutic use*; D016032:Randomized Controlled Trials as Topic
chemical_list: D018696:Neuroprotective Agents