Development of drugs for celiac disease: review of endpoints for Phase 2 and 3 trials.

ABSTRACT: Celiac disease is a lifelong disorder for which there is currently only one known, effective treatment: a gluten-free diet. New treatment approaches have recently emerged; several drugs are in Phase 2 trials and results appear promising; however, discussion around regulatory endpoints is in its infancy. We will briefly discuss the drugs that are under development and then shift our attention to potential trial endpoints, such as patient-reported outcomes, histology, serology, gene expression analysis and other tests. We will outline the differing requirements for proof-of-concept Phase 2 trials and Phase 3 registration trials, with a particular emphasis on current thinking in regulatory agencies. We conclude our paper with recommendations and a glossary of regulatory terms, to enable reade

SUBMITTER: Gottlieb K 

PROVIDER: S-EPMC4423465 | biostudies-literature | 2015 May

REPOSITORIES: biostudies-literature