Challenges of Clinical Trial Design, Conduct & Endpoints Workstream

The Challenges of Clinical Trial Design, Conduct & Endpoints Workstream aims to provide solutions to complex questions and hot topics in the field: 

• What’s the future of clinical trial design?
• How do we transform clinical endpoints into validated regulatory endpoints?
• How can we maximise outcomes for patients participating in these trials? 

The Workstream is currently working on synthesising the group’s experience and knowledge of clinical trial designs into a guidance document. We hope this will inspire the next generation of scientists to facilitate the development of novel drugs and treatments for PH patients. If you’re interested in joining this Workstream please get in touch.

Publications 

• Weatherald, J., Fleming, T.R., Wilkins, M.R., Cascino, T.M., Psotka, M.A., et al. (2024) 'Clinical trial design, end-points, and emerging therapies in pulmonary arterial hypertension'. European Respiratory Journal, 64(4), e2401205
• Win Statistics in Pulmonary Arterial Hypertension Clinical Trials, Jason Weatherald, Jude Moutchia, Nadine Al-Naamani, Robyn L. McClelland, Corey E. Ventetuolo, Harold I. Palevsky, Michael O. Harhay, Steven M. Kawut, 1 December 2023.
• Evolution and optimization of clinical trial endpoints and design in pulmonary arterial hypertension, Marco Caccamo, Frank E. Harrell, Anna R. Hemnes, 7 August 2023.
• The evolving landscape of pulmonary arterial hypertension clinical trials, Jason Weatherald, Athénaïs Boucly, Anthony Peters, David Montani, Krishna Prasad, Mitchell A Psotka et al. November 26, 2022.
• Repurposing of medications for pulmonary arterial hypertension, Mark Toshner, Edda Spiekerkoetter, Harm Bogaard, Georg Hansmann, Sylvia Nikkho, Kurt W. Prins, November 18, 2020. Presentation at PVRI Annual Congress, IDDI Pre-meeting.
• Novel composite clinical endpoints and risk scores used in clinical trials in PAH, Olivier Sitbon, Sylvia Nikkho, Raymond Benza, Chunqin (CQ) Deng, Harrison W. Farber, Mardi Gomberg-Maitland, Paul Hassoun, Christian Meier, Joanna Pepke-Zaba, Krishna Prasad, Werner Seeger, Paul A Corris, November 18, 2020.
• Role of biomarkers in evaluation, treatment and clinical studies of PAH , Anna Hemnes, Alexander M.K. Rothman, Andrew J. Swift, Lawrence S. Zisman, November 18, 2020.
• Clinical Trial Design in phase 2 and 3 trials for pulmonary hypertension, Sylvia Nikkho, Peter Fernandes, R. James White, Chunqin (CQ) Deng, Harrison W. Farber, Paul A Corris, July 20, 2020.