Randomized controlled trial
A randomized controlled trial (RCT) refers to clinical trial design in which the participants are assigned by chance to separate groups to evaluate the therapy being studied. The study also includes a control group, which is undergoing the same rigors of the study with placebo or standard clinical therapy. In most cases, RCT trials are also double-blind.
Double-blind RCTs are considered the Gold Standard to determine the safety and efficacy of a therapy. However, this study design may not always be possible in therapies being developed for a rare or genetic disease due to the limited number of participants meeting the eligibility criteria. This type of study may also not be considered ethical in conditions the progress quickly.