Systematic, comprehensive, evidence-based approach to

Authors

Charis Wong, Jenna M Gregory, Jing Liao, Kieren Egan, Hanna M Vesterinen, Aimal Ahmad Khan, Maarij Anwar, Caitlin Beagan, Fraser S Brown, John Cafferkey, Alessandra Cardinali, Jane Yi Chiam, Claire Chiang, Victoria Collins, Joyce Dormido, Elizabeth Elliott, Peter Foley, Yu Cheng Foo, Lily Fulton-Humble, Angus B Gane, Stella A Glasmacher, Áine Heffernan, Kiran Jayaprakash, Nimesh Jayasuriya, Amina Kaddouri, Jamie Kiernan, Gavin Langlands, D Leighton, Jiaming Liu, James Lyon, Arpan R Mehta, Alyssa Meng, Vivienne Nguyen, Na Hyun Park, Suzanne Quigley, Yousuf Rashid, Andrea Salzinger, Bethany Shiell, Ankur Singh, Tim Soane, Alexandra Thompson, Olaf Tomala, Fergal M Waldron, Bhuvaneish T Selvaraj, Jeremy Chataway, Robert Swingler, Peter Connick, Suvankar Pal, Siddharthan Chandran, Malcolm Macleod

Abstract

Motor neuron disease (MND) is an incurable progressive neurodegenerative disease with limited treatment options. There is a pressing need for innovation in identifying therapies to take to clinical trial. Here, we detail a systematic and structured evidence-based approach to inform consensus decision making to select the first two drugs for evaluation in Motor Neuron Disease-Systematic Multi-arm Adaptive Randomised Trial (MND-SMART: NCT04302870), an adaptive platform trial. We aim to identify and prioritise candidate drugs which have the best available evidence for efficacy, acceptable safety profiles and are feasible for evaluation within the trial protocol.

PMID:36725099 | DOI:10.1136/bmjopen-2022-064169