Clinical Trials Logo

Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT03932669
Other study ID # 1811-074-985
Secondary ID
Status Completed
Phase Phase 2
First received
Last updated
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
Health authority
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

Study Design


Related Conditions & MeSH terms


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
See also
  Status Clinical Trial Phase
Active, not recruiting NCT03703830 - tDCS Associated With Locomotor Training on Functional Mobility of Cerebellar Ataxia N/A
Completed NCT04937725 - Reading Problems Associated With Central Nervous System (CNS) Pathologies.
Completed NCT03120013 - Rehabilitative Trial With Cerebello-Spinal tDCS in Neurodegenerative Ataxia N/A
Recruiting NCT04830904 - Efficacy of Lycra Garments in Ataxic Subjects N/A
Not yet recruiting NCT04297540 - Neuromodulation of Social Skills in Childhood Ataxia N/A
Terminated NCT03901638 - Tllsh2910 for Ataxia and Gut Microbiota Alteration in Patients of Multiple System Atrophy Phase 3
Recruiting NCT03624374 - Natural History Study of Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation (LBSL)