Value of <scp>MRI</scp> Outcomes for Preventive and Early‐Stage Trials in Spinocerebellar Ataxias 1 and 3
The estimated sample sizes to detect a 50% reduction in progression with 80% power using the pons volume as primary outcome measure indicate the feasibility of preventive trials (n = 73 per arm in a 1-year trial) in common SCAs and predict a 6-fold reduction in required sample sizes relative to SARA in early interventional trials. These findings support the use of MRI endpoints in early-stage SCA trials.
Authors
- Readisca Investigators
- R. Valabrègue
- Young Woo Park (ORCID: https://orcid.org/0000-0001-8737-6278)
- James M. Joers (ORCID: https://orcid.org/0000-0002-8444-2185)
- Philipp Ehses (ORCID: https://orcid.org/0000-0002-5839-6525)
- Emilien Petit (ORCID: https://orcid.org/0009-0000-0602-7122)
- Jeremy D. Schmahmann (ORCID: https://orcid.org/0000-0003-0706-5125)
- J. R. Rezende Thiago
- Guita Banan
- Thomas H. Mareci (ORCID: https://orcid.org/0000-0003-4700-2364)
- Christophe Lenglet (ORCID: https://orcid.org/0000-0003-4646-3185)
- Michal Považan (ORCID: https://orcid.org/0000-0002-5498-5162)
- İpek Özdemir (ORCID: https://orcid.org/0000-0001-6807-9390)
- P BARKER
- S. H. Subramony (ORCID: https://orcid.org/0000-0002-8052-9240)
- SUB SUBRAMONY
- Eva Ratai
- Gülin Öz
- A. Durr
- Jennifer Faber
- Sophie Tezenas du Montcel
- H. Moore Arnold
- Thomas Klockgether
- Jonathan M. DuBois
- Tetsuo Ashizawa
- Henry Paulson
- Chiadi U. Onyike
Institutions
- Henry M. Jackson Foundation (US)
- Centre National de la Recherche Scientifique (FR)
- Biogen (United States) (US)
- Johns Hopkins University (US)
- Uniformed Services University of the Health Sciences (US)
- Inserm (FR)
- Universidade Estadual de Campinas (UNICAMP) (BR)
- Cornell University (US)
- University of Michigan (US)
- University Hospital Bonn (DE)
- Johns Hopkins Medicine (US)
- German Center for Neurodegenerative Diseases (DE)
- University of Florida (US)
- Sorbonne Université (FR)
- Assistance Publique – Hôpitaux de Paris (FR)
- Center for Neuro-Oncology (US)
- Methodist Hospital (US)
- Athinoula A. Martinos Center for Biomedical Imaging (US)
- Institut de Psychiatrie et Neurosciences de Paris (FR)
- Resonance Research (United States) (US)
Publication Details
- Journal
- Annals of Clinical and Translational Neurology
- Published
- 2026-02-10
- DOI
- https://doi.org/10.1002/acn3.70325
- Primary Topic
- Genetic Neurodegenerative Diseases
- Type
- article
- Field-Weighted Citation Impact
- 0.00