Effect of Nilotinib in Cerebellar Ataxia Patients | NCT03932669

Studies
Effect of Nilotinib in Cerebellar Ataxia Patients

NCT03932669

Sponsor

Seoul National University Hospital

Status

Completed

Last Update Posted

Sep 29, 2021Actual

Enrollment

70Actual

Phase

Phase 2

Conditions

Ataxia, Cerebellar
Ataxia, Progressive

Interventions

Nilotinib

Countries

South Korea

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

Annotation Module

Unposted Annotation

Unposted Responsible Party

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Sep 2021

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Nov 19, 2018Actual

Primary Completion Date

Aug 13, 2020Actual

Completion Date

Aug 13, 2020Actual

First Submitted Date

Apr 25, 2019

First Submission Date that Met QC Criteria

Apr 26, 2019

First Posted Date

May 1, 2019Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Sep 23, 2021

Last Update Posted Date

Sep 29, 2021Actual

Sponsor/Collaborators Module

Responsible Party, by Official Title

Kon Chu, Professor, Seoul National University HospitalPrincipal Investigator

Lead Sponsor

Seoul National University HospitalOTHER

Collaborators

Not provided

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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

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
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

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

70Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Nilotinib group	Experimental	Patients with SCA and taking nilotinib treatment	Drug: Nilotinib

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Nilotinib	Drug	150-300mg daily dose of nilotinib	Nilotinib group

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Activity of daily living	Barthel index: score range 0-6	12 month

Secondary Outcomes

Measure	Description	Time Frame
Cerebellar function	Scale for the assessment and rating of ataxia (SARA): score range 0-36	12 month
General function	Rating scale for Friedreich's ataxia I: score range 0-6

Other Outcomes

Measure	Description	Time Frame
Occurrence of adverse events	CTCAE version 4.0	Baseline, 1 month, 3 month, 6 month, 12 month

Eligibility Module

Eligibility 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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

85 Years

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Kon Chu, MD, PhD	Seoul National University Hospital	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Seoul National University Hospital		Seoul		03080	South Korea

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
28601294	Result	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.
39009709	Derived

Type	Date	Date Unknown
Release	Sep 5, 2023
Reset	Mar 14, 2024

ID	Term
D002524	Cerebellar Ataxia
D001259	Ataxia
D020754	Spinocerebellar Ataxias

ID	Term
D002526	Cerebellar Diseases
D001927	Brain Diseases
D002493	Central Nervous System Diseases
D009422	Nervous System Diseases