TY - JOUR
T1 - Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development
AU - Paganoni, Sabrina
AU - Berry, James D.
AU - Quintana, Melanie
AU - Macklin, Eric
AU - Saville, Benjamin R.
AU - Detry, Michelle A.
AU - Chase, Marianne
AU - Sherman, Alexander V.
AU - Yu, Hong
AU - Drake, Kristin
AU - Andrews, Jinsy
AU - Shefner, Jeremy
AU - Chibnik, Lori B.
AU - Vestrucci, Matteo
AU - Cudkowicz, Merit E.
AU - Macklin, Eric
AU - Quintana, Melanie
AU - Saville, Ben
AU - Detry, Michelle A.
AU - Chibnik, Lori
AU - Bind, Marie Abele
AU - Chan, James
AU - Vestrucci, Matteo
AU - Locatelli, Eduardo
AU - Alameda, Gustavo
AU - Paganoni, Sabrina
AU - Cudkowicz, Merit
AU - McCaffrey, Alexandra
AU - Berry, James
AU - Babu, Suma
AU - Nicholson, Katie
AU - Scalia, Jennifer
AU - Simmons, Zachary
AU - Grogan, James
AU - Su, Xiaowei
AU - Mamarabadi, Mansoureh
AU - Heitzman, Daragh
AU - Nasri, Mohamad Asaad
AU - Katz, Jonathan
AU - Jenkins, Liberty
AU - Felice, Kevin
AU - Whitaker, Charles
AU - Ajroud-Driss, Senda
AU - Quick, Adam
AU - Kolb, Stephen
AU - Sarah Heintzman, N. P.
AU - Appel, Stanley H.
AU - Greene, Ericka
AU - Thonhoff, Jason
AU - Kasarskis, Edward J.
N1 - Funding Information:
Healey Center philanthropic funding and foundation grants supported upfront planning costs, parent protocol core/infrastructure, the endpoint development engine, and part of the costs of the first 4 regimens. This initial upfront capital investment facilitated trial launch. For the future, the financial model is for industry partners to assume all the costs of their respective regimens. For potential investigator‐initiated regimens, grant opportunities might be considered. Each regimen is projected to cost substantially less than a traditional RCT of a similar size and design, due to the shared placebo arm and the substantial time savings from startup to close‐out.
Publisher Copyright:
© 2021 American Neurological Association.
PY - 2022/2
Y1 - 2022/2
N2 - 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.
AB - 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.
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U2 - 10.1002/ana.26285
DO - 10.1002/ana.26285
M3 - Article
C2 - 34935174
AN - SCOPUS:85122780992
VL - 91
SP - 165
EP - 175
IS - 2
ER -