Platform Trials to Expedite Drug Development in Alzheimer’s Disease: A Report from the EU/US CTAD Task Force - 21/11/24

Doi : 10.14283/jpad.2021.21 
P.S. Aisen 1, 9, , R.J. Bateman 2, M. Carrillo 3, R. Doody 4, K. Johnson 5, J.R. Sims 6, R. Sperling 7, B. Vellas 8

EU/US CTAD Task Force

1 Alzheimer’s Therapeutic Research Institute (ATRI), Keck School of Medicine, University of Southern California, San Diego, CA, USA 
2 Washington University School of Medicine, St. Louis, MO, USA 
3 Alzheimer’s Association, Chicago, IL, USA 
4 Genentech/Roche, Basel, Switzerland 
5 Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA 
6 Eli Lilly and Company, Indianapolis, IN, USA 
7 Brigham and Women’s Hospital, Boston, MA, USA 
8 Gerontopole, INSERM U1027, Alzheimer’s Disease Research and Clinical Center, Toulouse University Hospital, Toulouse, France 
9 University of Southern California Alzheimer’s Therapeutic Research Institute, San Diego, CA, USA 

a paisen@usc.edu paisen@usc.edu

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Abstract

A diverse range of platforms has been established to increase the efficiency and speed of clinical trials for Alzheimer’s disease (AD). These platforms enable parallel assessment of multiple therapeutics, treatment regimens, or participant groups; use uniform protocols and outcome measures; and may allow treatment arms to be added or dropped based on interim analyses of outcomes. The EU/US CTAD Task Force discussed the lessons learned from the Dominantly Inherited Alzheimer’s Network Trials Unit (DIAN-TU) platform trial and the challenges addressed by other platform trials that have launched or are in the planning stages. The landscape of clinical trial platforms in the AD space includes those testing experimental therapies such as DIAN-TU, platforms designed to test multidomain interventions, and those designed to streamline trial recruitment by building trial-ready cohorts. The heterogeneity of the AD patient population, AD drugs, treatment regimens, and analytical methods complicates the design and execution of platform trials, yet Task Force members concluded that platform trials are essential to advance the search for effective AD treatments, including combination therapies.

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Keywords : Alzheimer’s disease, anti-amyloid therapies, anti-tau therapies, platform trials, adaptive trial design, shared placebos, master protocols, secondary prevention


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 *EU/US/CTAD TASK FORCE: Rebecca Amariglio (Boston); Sandrine Andrieu (Toulouse); Rhoda Au (Boston); Clive Ballard (Exeter); Matthew Barton (Raleigh); Monika Baudler (Basel); Joanne Bell (Willmington); Kevin Biglan (Indianapolis); Adam Boxer (San Francisco); Sasha Bozeat (Basel); Claudine Brisard (Issy les Moulineaux); Miroslaw Brys (Indianapolis); Marc Cantillon (Gilbert); Bill Chan (Beijing); Ivan Cheung (Woodcliff Lake); Min Cho (Woodcliff Lake); Julia Coelho (San Francisco); Shobha Dhadda (Woodcliff Lake); Daniel Di Giusto (Basel); Michael Donohue (San Diego); John Dwyer (Washington); Michael Egan (North Wales); Rianne Esquivel (Malvern); Howard Fillit (New York); Wendy Galpern (New Jersey); Harald Hampel (Woodcliff Lake); Jason Hassenstab (St Louis); David Henley (New Jersey); Joseph Herring (North Wales); Carole Ho (South San Francisco); Michael Irizarry (Woodcliff Lake); Takeshi Iwatsubo (Tokyo); Gustavo Jimenez-Maggiora (San Diego); Jeffrey Kaye (Portland); Geoffrey Kerchner (South San Francisco); Gene Kinney (South San Francisco); Shailaja Korukonda (Woodcliff Lake); Lynn Kramer (Woodcliff Lake); Jaren Landen (Cambridge); Ishani Landri (Woodcliff Lake); Lars Lannfelt (Uppsala); Valérie Legrand (Nanterre); Simon Lovestone (Oxford); Manoj Malhotra (Woodcliff Lake); Eric McDade (St Louis); Fanni Natanegara (Indianapolis); Francisco Nogueira (South San Francisco); Gerald Novak (New Jersey); Gunilla Osswald (Stockholm); Susanne Ostrowitzki (South San Francisco); Amanda Paley (New York); Kathryn Papp (Boston); Martin Rabe (Woodcliff Lake); Rema Raman (San Diego); Elena Ratti (Cambridge); Laurie Ryan (Bethesda); Stephen Salloway (Providence); Peter Schüler (Langen); Hiroshi Sekiya (Malvern); Jiong Shi (Las Vegas); Melanie Shulman (Cambridge); Eric Siemers (Zionsville); Kaycee Sink (South San Francisco); Maria Soto-Martin (Toulouse); Joyce Suhy (Newark); Jina Swartz (London); Pierre Tariot (Phoenix); Edmond Teng (South San Francisco); Jacques Touchon (Montpellier); Martin Traber (Basel); Andrea Vergallo (Woodcliff Lake); Judy Walker (Singapore); Jon Walsh (San Francisco); Michael Weiner (San Francisco); Alette Wessels (Indianapolis); Haichen Yang (North Wales); Wagner Zago (San Francisco); Kenton Zavitz (Cambridge)


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Vol 8 - N° 3

P. 306-312 - Marzo 2021 Ritorno al numero
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