Effect of Nilotinib in Cerebellar Ataxia Patients

Seoul National University Hospital70 enrolled

Overview

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.

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 Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Ataxia, Cerebellar Ataxia, Progressive

Interventions

NilotinibDRUG

150-300mg daily dose of nilotinib

Eligibility

Sex: ALLMin age: 18 YearsMax age: 85 Years

Medical Language ↔ Plain English

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

Locations (1)

Seoul National University Hospital

Seoul, South Korea

Outcomes

Primary Outcomes

Activity of daily living

Barthel index: score range 0-6

Time frame: 12 month

Secondary Outcomes

Cerebellar function

Scale for the assessment and rating of ataxia (SARA): score range 0-36

Time frame: 12 month

General function

Rating scale for Friedreich's ataxia I: score range 0-6

Time frame: 12 month

Activity of daily living

Rating scale for Friedreich's ataxia II: score range 0-36

Time frame: 12 month

Data from ClinicalTrials.gov

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