Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development

for the Healey ALS Platform Trial Study Group, HEALEY ALS Platform Trial Study Group, Biostatisticians, Investigators, Clinical Operations Team, Medical Monitors

Research output: Contribution to journalArticlepeer-review

33 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)165-175
Number of pages11
JournalAnnals of Neurology
Issue number2
StatePublished - Feb 1 2022

Bibliographical note

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.

PubMed: MeSH publication types

  • Journal Article
  • Research Support, Non-U.S. Gov't
  • Review


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