Effect of Nilotinib in Cerebellar Ataxia Patients

Ataxia, Cerebellar Clinical Trial

Official title:

Effect of Nilotinib in Cerebellar Ataxia Patients

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03932669
• Other study ID #: 1811-074-985
• Status: Completed
• Phase: Phase 2
• Start date: November 19, 2018
• Est. completion date: August 13, 2020

Study information

• Verified date: September 2021
• Source: Seoul National University Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 70
• Est. completion date: August 13, 2020
• Est. primary completion date: August 13, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 85 Years

Eligibility

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

Related Conditions & MeSH terms

• Ataxia
• Ataxia, Cerebellar
• Cerebellar Ataxia

Intervention

Drug:
• Nilotinib
150-300mg daily dose of nilotinib

Locations

Country	Name	City	State
Korea, Republic of	Seoul National University Hospital	Seoul

Sponsors (1)

Lead Sponsor	Collaborator
Seoul National University Hospital

Country where clinical trial is conducted

Korea, Republic of, 

References & Publications (1)

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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Occurrence of adverse events	CTCAE version 4.0	Baseline, 1 month, 3 month, 6 month, 12 month
Primary	Activity of daily living	Barthel index: score range 0-6	12 month
Secondary	Cerebellar function	Scale for the assessment and rating of ataxia (SARA): score range 0-36	12 month
Secondary	General function	Rating scale for Friedreich's ataxia I: score range 0-6	12 month
Secondary	Activity of daily living	Rating scale for Friedreich's ataxia II: score range 0-36	12 month