Clinical Trial Designs in Amyotrophic Lateral Sclerosis: Does One Design Fit All?

ABSTRACT: The last 2 decades have seen a surge in the number of amyotrophic lateral sclerosis (ALS) clinical trials with the hope of finding successful treatments. Clinical trialists aim to repurpose existing drugs and test novel compounds to target potential ALS disease pathophysiology. Recent technological advancements have led to the discovery of new causative genetic agents and modes of delivering potential therapy, calling for increasingly sophisticated trial design. The standard ALS clinical trial design may be modified depending on study needs: type of therapy; route of therapy delivery; phase of therapy development; applicable subpopulation; market availability of therapy; and utility of telemedicine. Novel biomarkers of diagnostic, predictive, prognostic, and pharmacodynamic value are under

SUBMITTER: Nicholson KA 

PROVIDER: S-EPMC4404442 | biostudies-literature | 2015 Apr

REPOSITORIES: biostudies-literature