The Evolution of Randomized Clinical Trial Designs to Assess Therapeutics in Alzheimer Disease
This methodology research of AD RCTs highlights substantial changes in key features of AD clinical trials from 1992 to 2024. AD RCTs have become larger and longer, such that they are powered to detect smaller clinical differences. The increased sample sizes and duration should enable the detection of smaller and more slowly occurring outcomes, which may lead to successful RCTs of therapies with slower and more subtle efficacy.
Authors
- Angela T.H. Kwan (ORCID: https://orcid.org/0000-0003-4013-1112)
- Tharick Ali Pascoal (ORCID: https://orcid.org/0000-0001-9057-8014)
- Joseph Therriault (ORCID: https://orcid.org/0000-0002-7826-4781)
- Eduardo Rigon Zimmer (ORCID: https://orcid.org/0000-0002-5349-0053)
- Saman Arfaie (ORCID: https://orcid.org/0000-0001-5240-4275)
- Marcel S. Woo (ORCID: https://orcid.org/0000-0002-1306-2708)
- Giovanna Carello‐Collar (ORCID: https://orcid.org/0000-0003-4644-0151)
- Nesrine Rahmouni (ORCID: https://orcid.org/0009-0004-3081-8468)
- Arthur C. Macedo (ORCID: https://orcid.org/0000-0002-3957-0284)
- Stijn Servaes (ORCID: https://orcid.org/0000-0002-4431-957X)
- Brandon J. Hall (ORCID: https://orcid.org/0009-0007-4578-2033)
- Cécile Tissot (ORCID: https://orcid.org/0000-0003-2711-3833)
- Étienne Aumont (ORCID: https://orcid.org/0000-0002-8997-7090)
- Jean‐Paul Soucy
- Pedro Rosa‐Neto (ORCID: https://orcid.org/0000-0001-9116-1376)
- Paolo Vitali (ORCID: https://orcid.org/0000-0001-9204-2678)
- Serge Gauthier
- Lydia Trudel (ORCID: https://orcid.org/0009-0002-5593-7717)
- João-Pedro Ferrari-Souza
Institutions
- Universidade Federal do Rio Grande do Sul (BR)
- Lawrence Berkeley National Laboratory (US)
- University of Ottawa (CA)
- Universität Hamburg (DE)
- University of Pittsburgh (US)
- Southwestern Medical Center (US)
- University Medical Center Hamburg-Eppendorf (DE)
- McGill University (CA)
- The University of Texas Southwestern Medical Center (US)
- University of California, Berkeley (US)
Publication Details
- Journal
- JAMA Network Open
- Published
- 2025-08-29
- DOI
- https://doi.org/10.1001/jamanetworkopen.2025.29665
- Citations
- 1
- Primary Topic
- Health Systems, Economic Evaluations, Quality of Life
- Type
- article
- Field-Weighted Citation Impact
- 3.13