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Trial record 2 of 219 for:    Ataxia

Effect of Nilotinib in Cerebellar Ataxia Patients

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ClinicalTrials.gov Identifier: NCT03932669
Recruitment Status : Active, not recruiting
First Posted : May 1, 2019
Last Update Posted : May 1, 2019
Sponsor:
Information provided by (Responsible Party):
Kon Chu, Seoul National University Hospital

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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 70 participants
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


Arm Intervention/treatment
Experimental: Nilotinib group
Patients with SCA and taking nilotinib treatment
Drug: Nilotinib
150-300mg daily dose of nilotinib




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



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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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03932669


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

Publications of Results:
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Responsible Party: Kon Chu, Professor, Seoul National University Hospital
ClinicalTrials.gov Identifier: NCT03932669     History of Changes
Other Study ID Numbers: 1811-074-985
First Posted: May 1, 2019    Key Record Dates
Last Update Posted: May 1, 2019
Last Verified: April 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: The IPD will be provided upon request from any qualified investigator.

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Kon Chu, Seoul National University Hospital:
spinocerebellar ataxia, treatment, nilotinib, bcr-abl inhibitor

Additional relevant MeSH terms:
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Ataxia
Cerebellar Ataxia
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases