Effect of Nilotinib in Cerebellar Ataxia Patients

• Status: Completed
• Trial end date: 2020-08-13
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Seoul National University Hospital

Criteria

Inclusion Criteria:

• Diagnosed as chronic cerebellar ataxia

• Confirmed as spinocerebellar ataxia by gene test

Exclusion Criteria:

• Laboratory abnormalities that could interfere with the proper use of Nilotinib QTc interval >450ms on initial electrocardiograph, Hb <8.0, WBC<2000, ANC <1600, PLT <140,000, AST >200, ALT>200, ALP>575, Positive HIV serology, active hepatitis B

• Unstable mental or physical status that could interfere with precise evaluation and proper management of SCA Heart failure (NYHA Grade III or IV), history of major heart disease Pregnancy, on breast feeding