Designing Clinical Trials in Amyotrophic Lateral Sclerosis

Clinical trials in amyotrophic lateral sclerosis have significantly evolved over the last decade. New outcome measures have been developed that have reduced the sample size requirement as compared with survival studies. There has been increasing recognition that dose-ranging studies are crucial to full evaluation of experimental agents. While the requirements of late stage trials have not changed, many new designs have been suggested for earlier phase development. While no design achieves the perfect balance of sensitivity and efficiency, clinical trialists continue to work toward the goals of smaller and shorter trials so that more compounds can be studied concurrently.