Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development. Issue 2 (10th January 2022)

• Record Type: Journal Article
• Authors: Paganoni, Sabrina; Berry, James D.; Quintana, Melanie; Macklin, Eric; Saville, Benjamin R.; Detry, Michelle A.; Chase, Marianne; Sherman, Alexander V.; Yu, Hong; Drake, Kristin; Andrews, Jinsy; Shefner, Jeremy; Chibnik, Lori B.; Vestrucci, Matteo; Cudkowicz, Merit E.
• Other Names: investigator.; investigator.; Macklin Eric investigator.; Quintana Melanie investigator.; Saville Ben investigator.; Detry Michelle A. investigator.; Chibnik Lori investigator.; Bind Marie Abele investigator.; Chan James investigator.; Vestrucci Matteo investigator.; investigator.; Locatelli Eduardo investigator.; Alameda Gustavo investigator.; Paganoni Sabrina investigator.; Cudkowicz Merit investigator.; McCaffrey Alexandra investigator.; Berry James investigator.; Babu Suma investigator.; Nicholson Katie investigator.; Scalia Jennifer investigator.; Simmons Zachary investigator.; Grogan James investigator.; Su Xiaowei investigator.; Mamarabadi Mansoureh investigator.; Heitzman Daragh investigator.; Nasri Mohamad Asaad investigator.; Katz Jonathan investigator.; Jenkins Liberty investigator.; Felice Kevin investigator.; Whitaker Charles investigator.; Ajroud‐Driss Senda investigator.; Quick Adam investigator.; Kolb Stephen investigator.; Sarah Heintzman N. P. investigator.; Appel Stanley H. investigator.; (Simpson) Greene Ericka investigator.; Thonhoff Jason investigator.; Shroff Sheetal investigator.; Liao Bing investigator.; Goutman Stephen A. investigator.; Feldman Eva L. investigator.; Miller Timothy investigator.; Amber Malcolm N. P. investigator.; Newman Daniel S. investigator.; Arcila‐Londono Ximena investigator.; Steijlen Kara investigator.; Owegi Margaret Ayo investigator.; Brown Robert H. investigator.; Ghasemi Mehdi investigator.; Fernandes Joseph Americo M. investigator.; Piccione Ezequiel investigator.; Thaisetthawatkul Pariwat investigator.; Weiss Michael D. investigator.; Rad Nassim investigator.; Ilieva Hristelina investigator.; Pasinelli Piera investigator.; Judith Guarnieri N. P. investigator.; Ladha Shafeeq investigator.; Jacobsen Bill investigator.; Bowser Robert investigator.; Shefner Jeremy investigator.; Foster Laura investigator.; Jackson Carlayne investigator.; Rosenfeld Jeffrey investigator.; Bhuvaneswaran Karthikeyan investigator.; Elman Lauren investigator.; Quinn Colin investigator.; Andrews Jinsy investigator.; Julia Yasek N. P. investigator.; Fee Dominic investigator.; Heiman‐Patterson Terry investigator.; Deboo Anahita investigator.; Swenson Andrea investigator.; Nance Christopher investigator.; Vu Tuan investigator.; Suresh Niraja investigator.; Farias Jerrica investigator.; Caress Jim investigator.; Cartwright Michael investigator.; Rezania Kourosh investigator.; Elliott Matthew investigator.; Rutkove Seward investigator.; McIlduff Courtney investigator.; Cohen Jeffrey A. investigator.; Stommel Elijah investigator.; Glass Jonathan investigator.; Fournier Christina investigator.; Young Eufrosina investigator.; Wymer James P. investigator.; Granit Volkan investigator.; Benatar Michael investigator.; Lewis Richard investigator.; Burford Matthew investigator.; Diaz Frank investigator.; Maragakis Nicholas J. investigator.; Clawson Lora investigator.; Uchil Alpa investigator.; Riley Kristen investigator.; Mosmiller Betsy investigator.; Twydell Paul investigator.; Goyal Namita A. investigator.; Jawdat Omar investigator.; Jabari Duaa investigator.; Statland Jeffrey investigator.; Kasarskis Edward J. investigator.; Russell James W. investigator.; Jin Peter investigator.; Walk David investigator.; Maiser Sam investigator.; Shah Jaimin investigator.; Oskarsson Bjorn investigator.; Bedlack Richard investigator.; Li Xiaoyan investigator.; Chen I‐Hweii Amy investigator.; Olney Nicholas investigator.; Bazan Tracy investigator.; Hayat Ghazala investigator.; Pattee Gary L. investigator.; Donofrio Peter D. investigator.; Peltier Amanda C. investigator.; Martinez‐Thompson Jennifer investigator.; Nathan investigator.; Johnson Kristin investigator.; Govindarajan Raghav investigator.; Jones Vovanti investigator.; Bertorini Tulio investigator.; Tourenne Clotilde Lagier investigator.; Vakili Ghazaleh Sadri investigator.; investigator.; Chase Marianne investigator.; Sherman Alex investigator.; Yu Hong investigator.; Dagostino Derek investigator.; Bocchio Scott investigator.; Eydinov Matthew investigator.; Faulconer Kenneth investigator.; Kharakozova Olga investigator.; Korin Alexander investigator.; Novak Ilya investigator.; Sinani Ervin investigator.; Tarasenko Natalia investigator.; Vigneswaran Prasha investigator.; Katsovskiy Igor investigator.; Whitworth Isaac investigator.; Wahab Yusra investigator.; Gladden Catherine investigator.; Shaughnessy Aileen investigator.; Tustison Eric investigator.; Jentoft Katie investigator.; Ostrow Joe investigator.; Lim Sangwon investigator.; Changkuon Genevive investigator.; Mattos Annette De investigator.; Patterson Janae investigator.; Scott Robin investigator.; Figueroa‐Szostek Precious investigator.; Pothier Lindsay investigator.; Harkey Brittney investigator.; DiStefano Sofia investigator.; Kennedy Erin investigator.; Bailey Abbey investigator.; DiOrio Nicole investigator.; Feulner Jacob investigator.; Davis Matt investigator.; Gomes Jassira investigator.; DiMarzio Marisa investigator.; Henrique Jennifer investigator.; Benson Molly investigator.; Grabert Stacey investigator.; Garozzo Tessa investigator.; Hall Meghan investigator.; Randall Rebecca investigator.; Connolly Mariah investigator.; De Santiago Diana investigator.; Lavery Courtney investigator.; Kittle Gale investigator.; Hamilton Jenny investigator.; Bolger Patrick investigator.; Fetouh Ahmed investigator.; Kamp Cornelia investigator.; Kennedy Julie investigator.; Small Catherine investigator.; Bulat Allison investigator.; Drake Kristin investigator.; investigator.; McGarry Andrew investigator.; Torti Margherita investigator.;
• Is Part Of: Annals of neurology. Volume 91:Issue 2(2022)
• Page Start: 165
• Page End: 175
• Publication Date: 2022-01-10
• Subjects: Neurology -- Periodicals; Pediatric neurology -- Periodicals; Nervous system -- Surgery -- Periodicals; 616.8
• DOI: 10.1002/ana.26285
• Languages: English
• ISSNs: 0364-5134

Abstract

Abstract : Current therapeutic development in amyotrophic lateral sclerosis (ALS) relies on individual randomized clinical trials to test a specific investigational product in a single patient population. This approach has intrinsic limitations, including cost, time, and lack of flexibility. Adaptive platform trials represent a novel approach to investigate several interventions for a single disease in a continuous manner. Already in use in oncology, this approach is now being employed more often in neurology. Here, we describe a newly launched platform trial for ALS. The Healey ALS Platform Trial is testing multiple investigational products concurrently in people with ALS, with the goal of rapidly identifying novel treatments, biomarkers, and trial endpoints. ANN NEUROL 2022;91:165–175