Clinical trial designs and endpoints

Trials should be designed with consideration of the individual disease context and research question. Many different approaches may be justified. In this chapter, we therefore consider some of the principal components of trial design in general and within the context of the emerging field of gene and cell therapies. Many aspects of developing a trial protocol require striking a balance between scientific rigor and practicalities for which the voice of patients and their families should form an integral part. We outline the importance of the acceptability of trial designs to participants, the determination of a target population and eligibility criteria, stratification methods that ensure a balanced control of variance across the trial, adequate controls to answer research questions including considerations of placebo allocation, blinding, and endpoints.