IPF clinical trial design and endpoints

Curr Opin Pulm Med. 2014 Sep;20(5):463-71. doi: 10.1097/MCP.0000000000000091.


Purpose of review: There remains a dire need for therapies that impact the clinical course of patients with idiopathic pulmonary fibrosis (IPF). Indeed, there is a surge of interest in IPF therapeutics, with many candidate agents in various stages of development. Optimal design and implementation of the appropriate prospective clinical trials are essential to demonstrate clinical efficacy of promising drugs for the treatment of IPF. A key element in the success of such clinical trials is the choice of the best endpoint(s) to match the design of the study.

Recent findings: Although the results of many IPF clinical trials have been disappointing, these trials have provided valuable insights into the epidemiology and natural history of the disease and have sparked debate into the best clinical trial designs and endpoints.

Summary: This review will discuss the various clinical trial endpoints that have been used or proposed with a focus on their potential utility, as well as possible pitfalls that investigators should consider in the design of such studies.

Video abstract: http://links.lww.com/COPM/A13.

Publication types

  • Review

MeSH terms

  • Biomarkers / blood
  • Clinical Trials as Topic
  • Exercise Test
  • Hospitalization
  • Humans
  • Idiopathic Pulmonary Fibrosis / drug therapy*
  • Treatment Outcome


  • Biomarkers