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出境医 / 临床实验 / Effect of Nilotinib in Cerebellar Ataxia Patients

Effect of Nilotinib in Cerebellar Ataxia Patients

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
Ataxia, Cerebellar Drug: Nilotinib 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
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Study Type : Interventional  (Clinical Trial)
Estimated 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 : November 19, 2018
Estimated Primary Completion Date : April 30, 2020
Estimated Study Completion Date : June 30, 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 [ Time Frame: 12 month ]
    Barthel index: score range 0-6


Secondary Outcome Measures :
  1. Cerebellar function [ Time Frame: 12 month ]
    Scale for the assessment and rating of ataxia (SARA): score range 0-36

  2. General function [ Time Frame: 12 month ]
    Rating scale for Friedreich's ataxia I: score range 0-6

  3. Activity of daily living [ Time Frame: 12 month ]
    Rating scale for Friedreich's ataxia II: score range 0-36


Other Outcome Measures:
  1. Occurrence of adverse events [ Time Frame: Baseline, 1 month, 3 month, 6 month, 12 month ]
    CTCAE version 4.0


Eligibility Criteria
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Ages Eligible for Study:   18 Years to 85 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations

Locations
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Korea, Republic of
Seoul National University Hospital
Seoul, Korea, Republic of, 03080
Sponsors and Collaborators
Seoul National University Hospital
Investigators
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Principal Investigator: Kon Chu, MD, PhD Seoul National University Hospital
Tracking Information
First Submitted Date  ICMJE April 25, 2019
First Posted Date  ICMJE May 1, 2019
Last Update Posted Date May 1, 2019
Actual Study Start Date  ICMJE November 19, 2018
Estimated Primary Completion Date April 30, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 26, 2019) 		Activity of daily living [ Time Frame: 12 month ]
Barthel index: score range 0-6
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: April 26, 2019)
  • Cerebellar function [ Time Frame: 12 month ]
    Scale for the assessment and rating of ataxia (SARA): score range 0-36
  • General function [ Time Frame: 12 month ]
    Rating scale for Friedreich's ataxia I: score range 0-6
  • Activity of daily living [ Time Frame: 12 month ]
    Rating scale for Friedreich's ataxia II: score range 0-36
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures
 (submitted: April 26, 2019) 		Occurrence of adverse events [ Time Frame: Baseline, 1 month, 3 month, 6 month, 12 month ]
CTCAE version 4.0
Original Other Pre-specified Outcome Measures Same as current
 
Descriptive Information
Brief Title  ICMJE Effect of Nilotinib in Cerebellar Ataxia Patients
Official Title  ICMJE Effect of Nilotinib in Cerebellar Ataxia Patients
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
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Ataxia, Cerebellar
Intervention  ICMJE Drug: Nilotinib
150-300mg daily dose of nilotinib
Study Arms  ICMJE Experimental: Nilotinib group
Patients with SCA and taking nilotinib treatment
Intervention: Drug: Nilotinib
Publications * 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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Unknown status
Estimated Enrollment  ICMJE
 (submitted: April 26, 2019) 		70
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 30, 2020
Estimated Primary Completion Date April 30, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 85 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Korea, Republic of
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03932669
Other Study ID Numbers  ICMJE 1811-074-985
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Plan Description: The IPD will be provided upon request from any qualified investigator.
Responsible Party Kon Chu, Seoul National University Hospital
Study Sponsor  ICMJE Seoul National University Hospital
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Kon Chu, MD, PhD Seoul National University Hospital
PRS Account Seoul National University Hospital
Verification Date April 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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