Effect of Nilotinib in Cerebellar Ataxia Patients

Summary

This is an institutional cohort study. Patients confirmed with spinocerebellar ataxia (SCA) and taking or planning to take Nilotinib (Tasigna®) are enrolled in this study. The daily dose of Nilotinib is 150mg-300mg and the patients will be followed up at 1, 3, 6, and 12 months. Rating scale for Friedreich's ataxia I and II and Barthel index are used as general function and daily living performance index. Scale for assessment and rating of ataxia (SARA) are used as an objective measure of cerebellar function. Adverse drug reactions are evaluated based on CTCAE version 4.0.

Timeline

Start

2018-11-19

Primary completion

2020-08-13

Completion

2020-08-13

Publications

• Results Lee WJ, Moon J, Kim TJ, Jun JS, Lee HS, Ryu YJ, Lee ST, Jung KH, Park KI, Jung KY, Kim M, Lee SK, Chu K. The c-Abl inhibitor, nilotinib, as a potential therapeutic agent for chronic cerebellar ataxia. J Neuroimmunol. 2017 Aug 15;309:82-87. doi: 10.1016/j.jneuroim.2017.05.015. Epub 2017 May 24.
• Lee WJ, Moon J, Jang Y, Shin YW, Son H, Shin S, Jeon D, Han D, Lee ST, Park KI, Jung KH, Lee SK, Chu K. Nilotinib treatment outcomes in autosomal dominant spinocerebellar ataxia over one year. Sci Rep. 2024 Jul 15;14(1):16303. doi: 10.1038/s41598-024-67072-z.

Drugs

Indications