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Clinical trial information and results are updated daily from ClinicalTrials.gov. The latest data update was conducted on 01/21/2021.
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Drug Interventions

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Clinical trial information and results are updated daily from ClinicalTrials.gov. The latest data update was conducted on 01/21/2021.

Effect of Nilotinib in Cerebellar Ataxia Patients

Clinicaltrials.gov identifier NCT03932669

Recruitment Status Active, not recruiting

First Posted May 1, 2019

Last update posted May 1, 2019

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:Ataxia, Cerebellar
  • Intervention/Treatment: Drug: Nilotinib
  • 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
  • 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 2018
  • Estimated Primary Completion Date: April 2020
  • Estimated Study Completion Date: June 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
  • Ages Eligible for Study: 18 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 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
Contacts
Locations

Korea, Republic of
Seoul National University Hospital
Seoul

Sponsors and Collaborators

Seoul National University Hospital

Investigators

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

More Information