Amyotroph Lateral Sc
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Amyotroph Lateral Sc · Oct 2007
Randomized Controlled Trial Clinical TrialOutcome measures for early phase clinical trials.
As the number of potential neuroprotective agents for ALS increases, the need for early phase trials that screen drugs before proceeding to efficacy trials also grows. However, it is not known which outcome measures perform best and also provide the most meaningful information in brief small trials. We assessed the performance of different outcome measures for use in early phase clinical trials in ALS, and determined what degree of change in the ALSFRS-R that patients could perceive. ⋯ One-unit change in patient-perceived clinical function paralleled a 9-point decrease in the ALSFRS-R (p = 0.025; 95% CI 8, 10). This trial assessed just 30 patients over six months, but the standard outcome measures each performed dependably; all could be used in short-duration, early phase trials. The ALSFRS-R most strongly predicted survival and provided the most complete data, but large changes may be necessary before patients perceive treatment effects.