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

Klaus Gottlieb, Jill Dawson, Fez Hussain, Joseph A. Murray

Research output: Contribution to journalReview article

20 Scopus citations

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 readers who are less familiar with regulatory language to take maximum advantage of this review.

Original languageEnglish (US)
Pages (from-to)91-102
Number of pages12
JournalGastroenterology Report
Volume3
Issue number2
DOIs
StatePublished - May 1 2015

Keywords

  • Celiac disease
  • Clinical trials
  • Endpoints

ASJC Scopus subject areas

  • Gastroenterology

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