Operational Development and Launch of an Adaptive Platform Trial in Amyotrophic Lateral Sclerosis: Processes and Learnings From the First Four Regimens of the <scp>HEALEY ALS</scp> Platform Trial
The HEALEY ALS Platform Trial capitalized on the benefits of the platform approach, including an adaptable operational infrastructure, concurrent enrollment into four distinct regimens, and an accelerated start-up time for a new regimen added after initial trial launch.
Authors
- Eric Tustison
- Jeremy M. Shefner (ORCID: https://orcid.org/0000-0001-5067-5602)
- Gale Kittle
- Lori B. Chibnik (ORCID: https://orcid.org/0000-0001-6293-806X)
- Sabrina Paganoni (ORCID: https://orcid.org/0000-0003-0505-1168)
- Elisa Giacomelli (ORCID: https://orcid.org/0000-0001-9233-235X)
- Suma Babu (ORCID: https://orcid.org/0000-0002-2648-7625)
- James D. Berry (ORCID: https://orcid.org/0000-0003-0898-6081)
- Annette De Mattos
- Sofia DiStefano
- Merit Cudkowicz (ORCID: https://orcid.org/0000-0002-7075-1681)
- Marianne Chase
- Hong Yu (ORCID: https://orcid.org/0000-0002-2838-5809)
- Derek D’Agostino
- Jaclyn Pagliaro (ORCID: https://orcid.org/0000-0003-4289-3073)
- Meghan Hall
- Alexander V. Sherman (ORCID: https://orcid.org/0000-0003-2097-5289)
- Kristin Drake
- Andrew J. McGarry (ORCID: https://orcid.org/0009-0009-2819-6029)
- Margherita Torti (ORCID: https://orcid.org/0000-0002-5027-0469)
- Catherine B. Small (ORCID: https://orcid.org/0000-0002-0601-6615)
- Eric A. Macklin (ORCID: https://orcid.org/0000-0003-1618-3502)
- Mariah Connolly
- Brittney Harkey
- Courtney Igne
- Cornelia Kamp (ORCID: https://orcid.org/0000-0003-4158-8691)
- Genevive Changkuon
- Allison Bulat
- Erica Scirocco
- Lindsay Heyd
Institutions
- Barrow Neurological Institute (US)
- Harvard University (US)
- Spaulding Rehabilitation Hospital (US)
- Massachusetts General Hospital (US)
- Clinical Research Institute (US)
- University of Rochester (US)
Publication Details
- Journal
- Muscle & Nerve
- Published
- 2025-05-26
- DOI
- https://doi.org/10.1002/mus.28442
- Citations
- 4
- Primary Topic
- Amyotrophic Lateral Sclerosis Research
- Type
- article
- Field-Weighted Citation Impact
- 5.56
Funders
- ALS Association
- Sanofi
- Muscular Dystrophy Association
- Biogen
- Alexion Pharmaceuticals
- Regeneron Pharmaceuticals
- UCB
- Acorda Therapeutics
- Cytokinetics
- Sarepta Therapeutics
- Apellis Pharmaceuticals
- Eikonizo Therapeutics
- Mitsubishi Tanabe Pharma Corporation
- National Institutes of Health
- National Institute of Neurological Disorders and Stroke
- UCB Pharma