Functional outcome measures as clinical trial endpoints in ALS

Neurology. 2004 Nov 23;63(10):1933-5. doi: 10.1212/01.wnl.0000144345.49510.4e.

Abstract

The topiramate study was a 12-month randomized placebo-controlled trial in patients with ALS. Follow-up evaluation of the placebo group (n = 97) constituted a well-described cohort of patients with ALS, in whom multiple outcome measures were assessed at 3-month intervals. During the 12-month study period, the decline of forced vital capacity (FVC%) and ALS functional rating scale (ALSFRS) was linear, whereas the decline of maximum voluntary isometric contraction-arm (MVIC-arm) and MVIC-grip Z scores was curvilinear. Rates of FVC% and ALFRS decline, but not of MVIC-arm or MVIC-grip, were independent predictors of survival.

Publication types

  • Clinical Trial
  • Randomized Controlled Trial
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Amyotrophic Lateral Sclerosis / drug therapy*
  • Amyotrophic Lateral Sclerosis / mortality
  • Amyotrophic Lateral Sclerosis / physiopathology
  • Arm / physiopathology*
  • Biomarkers
  • Female
  • Fructose / analogs & derivatives*
  • Fructose / therapeutic use
  • Hand Strength*
  • Humans
  • Isometric Contraction
  • Life Tables
  • Male
  • Middle Aged
  • Outcome Assessment, Health Care / methods*
  • Predictive Value of Tests
  • Proportional Hazards Models
  • Randomized Controlled Trials as Topic / methods
  • Severity of Illness Index*
  • Survival Analysis
  • Topiramate
  • Treatment Outcome
  • Vital Capacity*

Substances

  • Biomarkers
  • Topiramate
  • Fructose