Effect of Nilotinib in Cerebellar Ataxia Patients

Phase 2

Completed

• Conditions: Ataxia, Cerebellar; Ataxia, Progressive
• Interventions: Drug: Nilotinib

• Registration Number: NCT03932669
• Lead Sponsor: Seoul National University Hospital

Brief 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.

Detailed Description

1. Patients taking Nilotinib 1-1.Baseline information: demographics, genetic type of SCA, brain MRI, electrocardiography, routine blood evaluations 1-2. Time point of evaluation: At initiation of taking Nilotinib, 1, 3, 6, and 12 months 1-3. Evaluation items (at each time point) Routine check-up: electrocardiography, routine blood evaluations Adverse events: CTCAE version 4.0 Daily living performance: Barthel index General function: Friedreich's ataxia I and II 1-4. Change of drug dose Based on the attending physician's decision

2. Patients planning to take Nilotinib 2-1.Baseline information: demographics, genetic type of SCA, brain MRI, electrocardiography, routine blood evaluations 2-2. Time point of evaluation: Baseline, 1, 3, 6, and 12 months 2-3. Evaluation items (at each time point) Routine check-up: electrocardiography, routine blood evaluations Adverse events: CTCAE version 4.0 Cerebellar function: Scale for assessment and rating of ataxia (SARA) Daily living performance: Barthel index General function: Friedreich's ataxia I and II 2-4. Change of drug dose Based on the attending physician's decision

Study Timeline

• Study Start: 2018-11-19
• Study First Submitted: 2019-04-25
• Study First Submitted QC: 2019-04-26
• Study First Posted: 2019-05-01
• Primary Completion: 2020-08-13
• Study Completion: 2020-08-13
• Status Verified: 2021-09
• Last Update Submitted: 2021-09-23
• Last Update Posted: 2021-09-29

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 70

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Nilotinib group	Nilotinib	Patients with SCA and taking nilotinib treatment

Primary Outcome Measures

Name	Time	Method
Activity of daily living	12 month	Barthel index: score range 0-6

Secondary Outcome Measures

Name	Time	Method
Cerebellar function	12 month	Scale for the assessment and rating of ataxia (SARA): score range 0-36
Activity of daily living	12 month	Rating scale for Friedreich's ataxia II: score range 0-36
General function	12 month	Rating scale for Friedreich's ataxia I: score range 0-6

Trial Locations

Locations (1)

Seoul National University Hospital; 🇰🇷 Seoul, Korea, Republic of