Development of drugs for celiac disease: review of endpoints for Phase 2 and 3 trials.
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.
Immunology and Internal Medicine - Medical Strategy & Science, Quintiles, Durham, NC, USA, Corporate Communications, Quintiles, Durham, NC, USA and Division of Gastroenterology and Hepatology, Mayo Clinic, Rochester, MN, USA klaus.gottlieb@quintiles.com.
Klaus
K
Gottlieb
Immunology and Internal Medicine - Medical Strategy & Science, Quintiles, Durham, NC, USA, Corporate Communications, Quintiles, Durham, NC, USA and Division of Gastroenterology and Hepatology, Mayo Clinic, Rochester, MN, USA.
Jill
J
Dawson
Immunology and Internal Medicine - Medical Strategy & Science, Quintiles, Durham, NC, USA, Corporate Communications, Quintiles, Durham, NC, USA and Division of Gastroenterology and Hepatology, Mayo Clinic, Rochester, MN, USA.
Fez
F
Hussain
Immunology and Internal Medicine - Medical Strategy & Science, Quintiles, Durham, NC, USA, Corporate Communications, Quintiles, Durham, NC, USA and Division of Gastroenterology and Hepatology, Mayo Clinic, Rochester, MN, USA.
Joseph A
JA
Murray
celiac disease; clinical trials; endpoints