Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development

Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development

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...

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Veröffentlicht in:Annals of neurology 2022-02, Vol.91 (2), p.165-175
Hauptverfasser: Chase, Marianne, Sherman, Alexander V., Shefner, Jeremy, Chibnik, Lori B., Vestrucci, Matteo, Cudkowicz, Merit E., Macklin, Eric, Saville, Ben, Detry, Michelle A., Chibnik, Lori, Bind, Marie Abele, Paganoni, Sabrina, Nicholson, Katie, Ajroud‐Driss, Senda, Kolb, Stephen, (Simpson) Greene, Ericka, Thonhoff, Jason, Shroff, Sheetal, Goutman, Stephen A., Feldman, Eva L., Amber Malcolm, N. P., Newman, Daniel S., Owegi, Margaret Ayo, Ghasemi, Mehdi, Fernandes, Joseph Americo M., Thaisetthawatkul, Pariwat, Rad, Nassim, Ilieva, Hristelina, Judith Guarnieri, N. P., Jacobsen, Bill, Bowser, Robert, Foster, Laura, Rosenfeld, Jeffrey, Bhuvaneswaran, Karthikeyan, Quinn, Colin, Julia Yasek, N. P., Fee, Dominic, Deboo, Anahita, Swenson, Andrea, Nance, Christopher, Vu, Tuan, Suresh, Niraja, Caress, Jim, Cartwright, Michael, McIlduff, Courtney, Stommel, Elijah, Glass, Jonathan, Young, Eufrosina, Wymer, James P., Granit, Volkan, Lewis, Richard, Maragakis, Nicholas J., Clawson, Lora, Uchil, Alpa, Riley, Kristen, Mosmiller, Betsy, Twydell, Paul, Goyal, Namita A., Jabari, Duaa, Kasarskis, Edward J., Shah, Jaimin, Bedlack, Richard, Li, Xiaoyan, Olney, Nicholas, Pattee, Gary L., Johnson, Kristin, Govindarajan, Raghav, Jones, Vovanti, Sherman, Alex, Eydinov, Matthew, Korin, Alexander, Novak, Ilya, Sinani, Ervin, Katsovskiy, Igor, Whitworth, Isaac, Gladden, Catherine, Tustison, Eric, Jentoft, Katie, Lim, Sangwon, Changkuon, Genevive, Figueroa‐Szostek, Precious, Pothier, Lindsay, DiStefano, Sofia, Kennedy, Erin, Bailey, Abbey, Feulner, Jacob, Gomes, Jassira, DiMarzio, Marisa, Henrique, Jennifer, Grabert, Stacey, Garozzo, Tessa, Randall, Rebecca, Connolly, Mariah, De Santiago, Diana, Lavery, Courtney, Hamilton, Jenny, Small, Catherine, Bulat, Allison, McGarry, Andrew
Format: Artikel
Sprache:eng
Schlagworte:
Amyotrophic lateral sclerosis
Amyotrophic Lateral Sclerosis - therapy
Animals
Biomarkers
Clinical trials
Clinical Trials as Topic - legislation & jurisprudence
Endpoint Determination
Humans
Neurology
Research Design
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Zusammenfassung: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
ISSN:0364-5134
1531-8249
DOI:10.1002/ana.26285