Clinical trial NCT03932669: Effect of Nilotinib in Cerebellar Ataxia Patients
Official title: Effect of Nilotinib in Cerebellar Ataxia Patients
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Ataxia, Cerebellar
Ataxia, Progressive
Keywords: spinocerebellar ataxia
treatment
nilotinib
bcr-abl
receptor-tyrosine-kinase-inhibitor
Lead sponsor: Seoul National University Hospital
Sponsor class: OTHER
First posted: 2019-05-01
Enrollment: 70
Sex: ALL
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

Eligibility 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

Primary outcomes:
measure: Activity of daily living
timeFrame: 12 month
Secondary outcomes:
measure: Cerebellar function
timeFrame: 12 month
measure: General function
timeFrame: 12 month
measure: Activity of daily living
timeFrame: 12 month
Other outcomes:
measure: Occurrence of adverse events
timeFrame: Baseline, 1 month, 3 month, 6 month, 12 month
Locations:
city: Seoul
country: Korea, Republic of
facility: Seoul National University Hospital
Study officials:
affiliation: Seoul National University Hospital
name: Kon Chu, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.