Methodology study: novel outcome measures to assess progression of ALS

Diagnosis ALS
Study phase Active
Participating centres UMC Utrecht

Observational methodology study of novel electrophysiological, physical, and imaging outcome measures to assess the progression of ALS.

The primary objectives of the study are to estimate and rank-order the longitudinal standardized mean changes over 6 months and over 12 months, for a set of novel outcome measures administered to participants with ALS, in order to identify measures that are more sensitive to disease progression than ALSFRS-R. The secondary objectives of this study are: To evaluate the test-retest reproducibility of each outcome measure; To determine correlations between 6 and 12-month changes in all exploratory measures with 18 and 24-month changes in ALSFRS-R and survival; To assess correlations between/among the various measures; To obtain biological samples in order to identify molecular correlates to the clinical measures and to further characterize previously identified and novel molecular biomarkers of disease progression for incorporation into future clinical studies.

Centers from nine countries are involved.