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 a...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 readers who are less familiar with regulatory language to take maximum advantage of this review.展开更多
文摘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 readers who are less familiar with regulatory language to take maximum advantage of this review.