Effect of Nilotinib in Cerebellar Ataxia Patients

Sponsor
Seoul National University Hospital (Other)
Overall Status
Completed
CT.gov ID
NCT03932669
Collaborator
(none)
70
1
1
20.8
3.4

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
Phase 2

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 Design

Study Type:
Interventional
Actual Enrollment :
70 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Effect of Nilotinib in Cerebellar Ataxia Patients
Actual Study Start Date :
Nov 19, 2018
Actual Primary Completion Date :
Aug 13, 2020
Actual Study Completion Date :
Aug 13, 2020

Arms and Interventions

Arm Intervention/Treatment
Experimental: Nilotinib group

Patients with SCA and taking nilotinib treatment

Drug: Nilotinib
150-300mg daily dose of nilotinib

Outcome Measures

Primary Outcome Measures

  1. Activity of daily living [12 month]

    Barthel index: score range 0-6

Secondary Outcome Measures

  1. Cerebellar function [12 month]

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

  2. General function [12 month]

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

  3. Activity of daily living [12 month]

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

Other Outcome Measures

  1. Occurrence of adverse events [Baseline, 1 month, 3 month, 6 month, 12 month]

    CTCAE version 4.0

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years to 85 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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

Contacts and Locations

Locations

Site City State Country Postal Code
1 Seoul National University Hospital Seoul Korea, Republic of 03080

Sponsors and Collaborators

  • Seoul National University Hospital

Investigators

  • Principal Investigator: Kon Chu, MD, PhD, Seoul National University Hospital

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Kon Chu, Professor, Seoul National University Hospital
ClinicalTrials.gov Identifier:
NCT03932669
Other Study ID Numbers:
  • 1811-074-985
First Posted:
May 1, 2019
Last Update Posted:
Sep 29, 2021
Last Verified:
Sep 1, 2021
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Keywords provided by Kon Chu, Professor, Seoul National University Hospital
Additional relevant MeSH terms:

Study Results

No Results Posted as of Sep 29, 2021