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. 

Publications 

• 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 
  First Published July 20, 2020 
   
• Role of biomarkers in evaluation, treatment and clinical studies of PAH 
  Anna Hemnes, Alexander M.K. Rothman, Andrew J. Swift, Lawrence S. Zisman 
  First Published November 18, 2020 
   
• 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 
  First Published November 18, 2020 
   
• Repurposing of medications for pulmonary arterial hypertension 
  Mark Toshner, Edda Spiekerkoetter, Harm Bogaard, Georg Hansmann, Sylvia Nikkho, Kurt W. Prins 
  First Published November 18, 2020 
  Presentation at PVRI Annual Congress, IDDI Pre-meeting