Modeling Disease Progression and Placebo Response in Huntington Disease
Our results suggest that clinical scores in Huntington disease trials are susceptible to long-term placebo responses. These effects should be considered in future trial designs, especially when comparing trial data with natural history studies. Although based on the largest Huntington's trial, our results rely on limited placebo data and may not generalize to other trials with different populations, treatments, and designs.
Authors
- Cristina Sampaio (ORCID: https://orcid.org/0000-0002-7052-9079)
- Patricia Sanwald Ducray (ORCID: https://orcid.org/0000-0002-2904-5857)
- Cheikh Diack (ORCID: https://orcid.org/0000-0002-6320-7522)
- Jeffrey D. Long (ORCID: https://orcid.org/0000-0001-7181-9652)
- Peter McColgan (ORCID: https://orcid.org/0000-0002-5452-0648)
- Marcelo Boareto (ORCID: https://orcid.org/0000-0002-9915-6376)
- Yumi Yamamoto (ORCID: https://orcid.org/0000-0003-1798-3472)
Institutions
- Roche (Switzerland) (CH)
- University of Iowa (US)
- CHDI Foundation (US)
- Roche (United Kingdom) (GB)
Publication Details
- Journal
- Neurology
- Published
- 2025-05-02
- DOI
- https://doi.org/10.1212/wnl.0000000000213646
- Citations
- 4
- Primary Topic
- Genetic Neurodegenerative Diseases
- Type
- article
- Field-Weighted Citation Impact
- 3.64