TY - JOUR T1 - End-points and clinical trial design in pulmonary arterial hypertension: have we made progress? JF - European Respiratory Journal JO - Eur Respir J SP - 231 LP - 242 DO - 10.1183/09031936.00107108 VL - 34 IS - 1 AU - A. J. Peacock AU - R. Naeije AU - N. Galiè AU - L. Rubin Y1 - 2009/07/01 UR - http://erj.ersjournals.com/content/34/1/231.abstract N2 - There is enormous interest in the treatment of pulmonary arterial hypertension (PAH), so it is appropriate to consider the design of trials of new therapies and the end-points to be measured when trying to decide whether or not a therapy is effective. In May 2003, the first meeting devoted solely to the discussion of end-points and trial design in PAH was held in Gleneagles, UK. At that time, most of the randomised controlled trials in PAH had used 6-min walking distance and/or resting haemodynamics as their primary end-points. The present article considers the progress that has been made since 2003. It deals with aspects of clinical trial design (such as noninferiority, superiority and withdrawal trials), considers end-points used in previous and current studies (such as 6-min walking distance, time to clinical worsening, haemodynamics, imaging and plasma brain natriuretic peptide), and considers what end-points might be used in the future. The second end-points meeting was held in Turnberry, UK, in June 2007. It had a similar format to the first meeting. Much of what is presented here is a summary of the workshops from that meeting. An attempt has been made to both summarise the current state of end-points and trial design and suggest new ways in which they could be improved. The present article forms one of a series being published in the European Respiratory Journal on pulmonary hypertension. SERIES “PULMONARY HYPERTENSION: BASIC CONCEPTS FOR PRACTICAL MANAGEMENT” Edited by M.M. Hoeper and A.T. Dinh-Xuan Number 10 in this Series ER -