Ataxia, CerebellarPhase 2NCT03932669
Effect of Nilotinib in Cerebellar Ataxia Patients
A Phase 2 study testing Nilotinib in people with Ataxia, Cerebellar and Ataxia, Progressive, run by Seoul National University Hospital at 1 site in South Korea.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Ataxia, Cerebellar, Ataxia, Progressive
- What is being tested?
- Nilotinib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- 1 siteSouth Korea
- Study dates
- 19 Nov 2018to 13 Aug 2020
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosed as chronic cerebellar ataxia
- Confirmed as spinocerebellar ataxia by gene test
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read the 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
- Conditions
- Ataxia, Cerebellar, Ataxia, Progressive
- Being tested
- Nilotinib
- Keywords
- spinocerebellar ataxia, treatment, nilotinib, bcr-abl, receptor-tyrosine-kinase-inhibitor
- Medical subject headings
- Cerebellar Ataxia, Ataxia, Spinocerebellar Ataxias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NilotinibDrug
150-300mg daily dose of nilotinib
| Study group | Role | What they receive |
|---|---|---|
| Nilotinib group | Experimental | Patients with SCA and taking nilotinib treatment Receives: Drug: Nilotinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Activity of daily living
Barthel index: score range 0-6
Measured: 12 month
Secondary outcomes (3)
Cerebellar function
Scale for the assessment and rating of ataxia (SARA): score range 0-36
Measured: 12 month
General function
Rating scale for Friedreich's ataxia I: score range 0-6
Measured: 12 month
Activity of daily living
Rating scale for Friedreich's ataxia II: score range 0-36
Measured: 12 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 May 2019
- Study start19 Nov 2018
- Primary completion13 Aug 2020
- Study completion13 Aug 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Ataxia, CerebellarStudies of the same condition, open and under way.
- More trials run by Seoul National University HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 70 participants (actual)
Sponsor and investigators
- Lead sponsor
- Seoul National University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Kon Chu, Seoul National University Hospital
- Investigators
- Kon Chu, MD, PhD, Principal investigator, Seoul National University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03932669
- Sponsor's protocol ID
- 1811-074-985
- Status last verified
- Sep 2021
- First submitted
- 25 Apr 2019
- Last updated
- 29 Sep 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: The IPD will be provided upon request from any qualified investigator.
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.
PubMed 28601294Lee WJ, Moon J, Jang Y, Shin YW, Son H, Shin S, Jeon D, Han D, Lee ST, Park KI, Jung KH, Lee SK, Chu K. Nilotinib treatment outcomes in autosomal dominant spinocerebellar ataxia over one year. Sci Rep. 2024 Jul 15;14(1):16303. doi: 10.1038/s41598-024-67072-z.
PubMed 39009709
Study at a glance
From the registry record, last updated by the sponsor on 29 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Nilotinib in Cerebellar Ataxia Patients
- ClinicalTrials.gov ID
- NCT03932669
- Acronym / sponsor ID
- 1811-074-985
- Conditions
- Ataxia, Cerebellar, Ataxia, Progressive
- Keywords
- spinocerebellar ataxia, treatment, nilotinib, bcr-abl, receptor-tyrosine-kinase-inhibitor
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 70 participants (actual)
- Lead sponsor
- Seoul National University HospitalUniversity, hospital or other
- Study start
- 19 Nov 2018 (actual)
- Primary completion
- 13 Aug 2020 (actual)
- Study completion
- 13 Aug 2020 (actual)
- First posted
- 1 May 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Nilotinib group | Experimental | Drug: NilotinibPatients with SCA and taking nilotinib treatment |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Diagnosed as chronic cerebellar ataxia
- Confirmed as spinocerebellar ataxia by gene test
Key exclusion criteria (2 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Nilotinib150-300mg daily dose of nilotinib | Drug | Nilotinib group |
Primary outcome
Activity of daily living
Time frame: 12 month
Barthel index: score range 0-6
3 secondary outcomes
Cerebellar function
Time frame: 12 month
General function
Time frame: 12 month
Activity of daily living
Time frame: 12 month
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Sep 2021.
- Results publication: PubMed 28601294Lee 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.
- Derived from this study: PubMed 39009709Lee WJ, Moon J, Jang Y, Shin YW, Son H, Shin S, Jeon D, Han D, Lee ST, Park KI, Jung KH, Lee SK, Chu K. Nilotinib treatment outcomes in autosomal dominant spinocerebellar ataxia over one year. Sci Rep. 2024 Jul 15;14(1):16303. doi: 10.1038/s41598-024-67072-z.
- Individual participant dataSharing planned: No. The IPD will be provided upon request from any qualified investigator.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kon Chu, MD, PhD principal investigator
Seoul National University Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Nilotinib in people with Ataxia, Cerebellar and Ataxia, Progressive, run by Seoul National University Hospital at 1 site in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 Sep 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Activity of daily living (time frame: 12 month). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03932669, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Sep 2021.