Cerebellar AtaxiaNCT05002218
Training in Ataxia - Individuals With Degenerative Cerebellar Diseases
A study testing Aerobic Training and Balance Training in people with Cerebellar Ataxia, run by Columbia University at 1 site in United States.
Trial summary
Balance and aerobic training show promise as treatments for degenerative cerebellar diseases, but the neural effects of both training methods are unknown. The goal of this project is to evaluate how each training method impacts the brain, and particularly, the degenerating cerebellum. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cerebellar Ataxia
- What is being tested?
- Aerobic Training and Balance Training
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 64 participants
- Where
- 1 siteUnited States
- Study dates
- 31 Jan 2022to 6 Nov 2024
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosed with spinocerebellar ataxia
- Cerebellar atrophy on MRI
- Prevalence of ataxia on clinical exam
- Ability to safely ride a stationary exercise bike
Who cannot join
- Other neurologic conditions
- Heart disease
- Cognitive impairment
- Medical instability
Trial locations and contacts
1 site in 1 country.
United States1 site
Columbia University Irving Medical Center
New York, New York, 10032
About this study
Official title: The Neural Effects of Balance Versus Aerobic Training in Individuals With Degenerative Cerebellar Diseases
Balance and aerobic training show promise as treatments for degenerative cerebellar diseases, but the neural effects of both training methods are unknown. The goal of this project is to evaluate how each training method impacts the brain, and particularly, the degenerating cerebellum. Various neuroimaging techniques will be used to accomplish this goal and test the hypothesis that balance training impacts brain structures outside the cerebellum whereas aerobic training causes more neuroplastic changes within the cerebellum.
Read the detailed description
Degenerative cerebellar diseases are a group of disorders that cause severe disability and can be fatal. There are currently no known disease-modifying treatments available for use, and there is a critical need to find treatments that slow disease progression and allow affected individuals to live more functional lives. Balance and aerobic training show promise as treatments for degenerative cerebellar diseases, but the neural effects of both training methods have not been thoroughly investigated. It is crucial to understand how the training impacts the brain, and particularly the cerebellum, in order to determine if one training method is better at slowing disease progression than the other. The goal of this proposal is to compare the neural effects of balance versus aerobic training in individuals with degenerative cerebellar diseases. The investigator hypothesizes that aerobic training causes neuroplastic changes within the cerebellum whereas balance training causes improvements for people with cerebellar degeneration by impacting brain structures outside the cerebellum. If this hypothesis is true, aerobic training may have more influence on disease progression than balance training as it directly impacts the cerebellum.
To investigate the hypothesis, various neuroimaging techniques will be used. In AIM 1, the investigator will compare cerebellar volume before and after the participants perform either 6-months of balance or aerobic training. In AIM 2, the investigator will investigate whether neural changes have clinical significance by correlating cerebellar volume changes with clinical measures of ataxia. Finally, for AIM 3, the investigator will use diffusion tensor imaging and resting state fMRI scans to examine how both training methods impact cerebellar microstructure and functional cerebellar connections. The investigator hopes that a detailed understanding of how each training method impacts the cerebellum will lead to more targeted training regimens with the goal of slowing disease progression of these devastating diseases.
- Conditions
- Cerebellar Ataxia
- Being tested
- Aerobic Training, Balance Training
- Keywords
- ataxia, cerebellar ataxia, exercise, training, rehabilitation
- Medical subject headings
- Cerebellar Ataxia, Ataxia, Motor Activity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Aerobic TrainingBehavioural
Aerobic training on stationary bike 5x a week for 30 minutes a day.
Balance TrainingBehavioural
Balance training 5x a week for 30 minutes as instructed by a therapist. Standard of care.
| Study group | Role | What they receive |
|---|---|---|
| Aerobic Training | Experimental | Participants will be given a stationary exercise bike for home use. They will be instructed to use the exercise bike five times a week for thirty-minute sessions. The exercise intensity prescription will be based on the subject's VO2max determined on pre-test day. The exercise program will start at 60% of intensity per session, and then will be increased by steps of 5% intensity every 2 sessions until participants reach 30 minutes of training at 80% intensity. Participants will be contacted weekly by e-mail or phone to answer any questions about the exercise protocol and will be instructed to log each training session. Subjects will record duration of exercise, perceived exertion, average heart rate, maximum heart rate, and distance. Receives: Behavioral: Aerobic Training |
| Balance Training | Active comparator | A physical therapist will tailor a home balance training program for each participant based on pre- training capabilities. Subjects will be asked to perform exercises five times a week for thirty-minute sessions. Both dynamic and static exercises will be performed in sitting and standing positions. Exercises will start with stabilizing in a challenging static position and progress to dynamic arm and leg movements in the same or modified position. Participants will be contacted weekly by e-mail or phone to answer any questions about the exercise protocol and will be required to log their exercise effort in terms of frequency and level of balance challenge. Receives: Behavioral: Balance Training |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Assessment and Rating of Ataxia (SARA) Score
This is to measure ataxia severity. The Scale for the Assessment and Rating of Ataxia (SARA) will be administered before and after training. SARA is an 8-item performance based scale, yielding a total score of 0 (no ataxia) to 40 (most severe ataxia) - with higher scores indicating more severe ataxia. The scores are based on patient performance of gait, stance, sitting, speech disturbance, finger chase, nose-finger test, fast alternating hand movements and heel-shin slide. The change in score from baseline to 6 months, 9 months, and 12 months will be reported.
Measured: Baseline, 6 months, 9 months, 12 months
Secondary outcomes (11)
Average Gait Speed
This is to measure average time to complete 8-meter walk test. Participants will walk 8 meters as fast as possible three different times. Gait will be reported as meters per second (m/s).
Measured: Baseline, 6 months, 9 months, 12 months
Dynamic Gait Index Score
The dynamic gait index (DGI) will be performed to assess balance. Patients will be asked to walk 20 feet and conditions such as speed and head position will be varied as previously described. The examiner will then grade the subject's movement on a four-point ordinal scale, ranging from 0 (lowest level of function) to 3 (highest level of function). The total score range is 0 to 24, with higher scores indicating better dynamic balance and functional mobility.
Measured: Baseline, 6 months, 9 months, 12 months
Timed Up and Go (TUG)
The Timed Up and Go will be performed to assess balance. Once the tester says "Go", participants will stand from seated and walk around a cone that is 3 meters away, then walk back to the chair and sit back down. Participants will be timed from the moment the tester says "Go" until seated again.
Measured: Baseline, 6 months, 9 months, 12 months
Fatigue Severity Scale (FSS) Score
The FSS is a nine-item questionnaire that measures the severity of fatigue. Each item is rated on a 7-point Likert scale, from 1 (strongly disagree) to 7 (strongly agree). Scores for all nine items are summed to calculate the total score, which ranges from 9 to 63. Higher scores indicate greater fatigue severity.
Measured: Baseline, 6 months, 9 months, 12 months
Quality of Life (QOL) - Physical Health
The WHOQOL-BREF measures self-perceived quality of life across four domains: Physical Health, Psychological, Social Relationships, and Environment. Scores are derived from a 5-point Likert scale, with higher scores indicating better quality of life in that specific area. To calculate the domain scores, the mean score for items within each domain is multiplied by 4, and these scores are transformed to a 0 - 100 scale for interpretation.
Measured: Baseline, 6 months, 9 months, 12 months
Quality of Life (QOL) - Psychological
The WHOQOL-BREF measures self-perceived quality of life across four domains: Physical Health, Psychological, Social Relationships, and Environment. Scores are derived from a 5-point Likert scale, with higher scores indicating better quality of life in that specific area. To calculate the domain scores, the mean score for items within each domain is multiplied by 4, and these scores are transformed to a 0 - 100 scale for interpretation.
Measured: Baseline, 6 months, 9 months, 12 months
Quality of Life (QOL) - Social Relationships
The WHOQOL-BREF measures self-perceived quality of life across four domains: Physical Health, Psychological, Social Relationships, and Environment. Scores are derived from a 5-point Likert scale, with higher scores indicating better quality of life in that specific area. To calculate the domain scores, the mean score for items within each domain is multiplied by 4, and these scores are transformed to a 0 - 100 scale for interpretation.
Measured: Baseline, 6 months, 9 months, 12 months
Quality of Life (QOL) - Environment
The WHOQOL-BREF measures self-perceived quality of life across four domains: Physical Health, Psychological, Social Relationships, and Environment. Scores are derived from a 5-point Likert scale, with higher scores indicating better quality of life in that specific area. To calculate the domain scores, the mean score for items within each domain is multiplied by 4, and these scores are transformed to a 0 - 100 scale for interpretation.
Measured: Baseline, 6 months, 9 months, 12 months
Prevalence of Desired Changes in Diffuse Tensor Imaging
Diffusion data will be preprocessed for motion and corrected for geometrical distortion using ExploreDTI. For each participant, the bmatrix will be reoriented to provide a more accurate estimate of diffusion tensor orientations. Diffusion tensor estimation will be performed using a non-linear least square fitting method. FA and Mean Diffusivity (MD) maps will be generated. Whole brain tractography will be performed using all brain voxels with FA ≤ 0.2 as seed region.
Measured: 6 months
Prevalence of Cerebellar Volume
To determine cerebellar volume, each T1 scan will be visually inspected to ensure inclusion of only minimal movement artifacts. All images will be processed in a blinded manner in order to maintain accuracy and consistency of volume calculation. Regional cerebellar volumes will be calculated using the SUIT toolbox of the SPM12 software.
Measured: Baseline, 6 months, 9 months, 12 months
Prevalence of Desired Changes in Resting State fMRI Scans
This measure will be a primary outcome for Aim 3. The anatomical and functional data will be pre-processed and analyzed using Statistical Parametric Mapping (SPM12) and the CONN toolbox Version 14p.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Aug 2021
- Study start31 Jan 2022
- Primary completion6 Nov 2024
- Study completion6 Nov 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cerebellar AtaxiaStudies of the same condition, open and under way.
- More trials run by Columbia UniversityThe 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 N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- This is a single-blinded randomized control trial.
- Masking
- Double blind
- Who is masked
- Care provider, Outcomes assessor
- Enrolment
- 64 participants (actual)
Sponsor and investigators
- Lead sponsor
- Columbia University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Scott Barbuto, Columbia University
- Investigators
- Scott M Barbuto, MD, Principal investigator, Assistant Professor of Rehabilitation and Regenerative Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05002218
- Sponsor's protocol ID
- AAAT8388
- Other IDs
- 1K23NS121518-01A1
- Status last verified
- Aug 2025
- First submitted
- 4 Aug 2021
- Last updated
- 4 Nov 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Barbuto S, Lee S, Stein J, Kuo SH, Quinn L, Spinner M, Stern Y. Home Training for Cerebellar Ataxias: A Randomized Clinical Trial. JAMA Neurol. 2025 Nov 1;82(11):1162-1170. doi: 10.1001/jamaneurol.2025.3421.
PubMed 40946705
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Neural Effects of Balance Versus Aerobic Training in Individuals With Degenerative Cerebellar Diseases
- ClinicalTrials.gov ID
- NCT05002218
- Acronym / sponsor ID
- AAAT8388
- Conditions
- Cerebellar Ataxia
- Keywords
- ataxia, cerebellar ataxia, exercise, training, rehabilitation
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 64 participants (actual)
- Lead sponsor
- Columbia UniversityUniversity, hospital or other
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS)
- Study start
- 31 Jan 2022 (actual)
- Primary completion
- 6 Nov 2024 (actual)
- Study completion
- 6 Nov 2024 (actual)
- First posted
- 12 Aug 2021 (actual)
- Results
- Posted 4 Nov 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Balance and aerobic training show promise as treatments for degenerative cerebellar diseases, but the neural effects of both training methods are unknown. The goal of this project is to evaluate how each training method impacts the brain, and particularly, the degenerating cerebellum. Various neuroimaging techniques will be used to accomplish this goal and test the hypothesis that balance training impacts brain structures outside the cerebellum whereas aerobic training causes more neuroplastic changes within the cerebellum.
Detailed description
Degenerative cerebellar diseases are a group of disorders that cause severe disability and can be fatal. There are currently no known disease-modifying treatments available for use, and there is a critical need to find treatments that slow disease progression and allow affected individuals to live more functional lives. Balance and aerobic training show promise as treatments for degenerative cerebellar diseases, but the neural effects of both training methods have not been thoroughly investigated. It is crucial to understand how the training impacts the brain, and particularly the cerebellum, in order to determine if one training method is better at slowing disease progression than the other. The goal of this proposal is to compare the neural effects of balance versus aerobic training in individuals with degenerative cerebellar diseases. The investigator hypothesizes that aerobic training causes neuroplastic changes within the cerebellum whereas balance training causes improvements for people with cerebellar degeneration by impacting brain structures outside the cerebellum. If this hypothesis is true, aerobic training may have more influence on disease progression than balance training as it directly impacts the cerebellum.
To investigate the hypothesis, various neuroimaging techniques will be used. In AIM 1, the investigator will compare cerebellar volume before and after the participants perform either 6-months of balance or aerobic training. In AIM 2, the investigator will investigate whether neural changes have clinical significance by correlating cerebellar volume changes with clinical measures of ataxia. Finally, for AIM 3, the investigator will use diffusion tensor imaging and resting state fMRI scans to examine how both training methods impact cerebellar microstructure and functional cerebellar connections. The investigator hopes that a detailed understanding of how each training method impacts the cerebellum will lead to more targeted training regimens with the goal of slowing disease progression of these devastating diseases.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (care provider, outcomes assessor)
- Number of arms
- 2
- Model description
- This is a single-blinded randomized control trial.
| Arm | Type | Receives |
|---|---|---|
| Aerobic Training | Experimental | Behavioral: Aerobic TrainingParticipants will be given a stationary exercise bike for home use. They will be instructed to use the exercise bike five times a week for thirty-minute sessions. The exercise intensity prescription will be based on the subject's VO2max determined on pre-test day. The exercise program will start at 60% of intensity per session, and then will be increased by steps of 5% intensity every 2 sessions until participants reach 30 minutes of training at 80% intensity. Participants will be contacted weekly by e-mail or phone to answer any questions about the exercise protocol and will be instructed to log each training session. Subjects will record duration of exercise, perceived exertion, average heart rate, maximum heart rate, and distance. |
| Balance Training | Active comparator | Behavioral: Balance TrainingA physical therapist will tailor a home balance training program for each participant based on pre- training capabilities. Subjects will be asked to perform exercises five times a week for thirty-minute sessions. Both dynamic and static exercises will be performed in sitting and standing positions. Exercises will start with stabilizing in a challenging static position and progress to dynamic arm and leg movements in the same or modified position. Participants will be contacted weekly by e-mail or phone to answer any questions about the exercise protocol and will be required to log their exercise effort in terms of frequency and level of balance challenge. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Diagnosed with spinocerebellar ataxia
- Cerebellar atrophy on MRI
- Prevalence of ataxia on clinical exam
- Ability to safely ride a stationary exercise bike
Key exclusion criteria (4 listed)
- Other neurologic conditions
- Heart disease
- Cognitive impairment
- Medical instability
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 |
|---|---|---|
| Aerobic TrainingAerobic training on stationary bike 5x a week for 30 minutes a day. | Behavioural | Aerobic Training |
| Balance TrainingBalance training 5x a week for 30 minutes as instructed by a therapist. Standard of care. | Behavioural | Balance Training |
Primary outcome
Change in Assessment and Rating of Ataxia (SARA) Score
Time frame: Baseline, 6 months, 9 months, 12 months
This is to measure ataxia severity. The Scale for the Assessment and Rating of Ataxia (SARA) will be administered before and after training. SARA is an 8-item performance based scale, yielding a total score of 0 (no ataxia) to 40 (most severe ataxia) - with higher scores indicating more severe ataxia. The scores are based on patient performance of gait, stance, sitting, speech disturbance, finger chase, nose-finger test, fast alternating hand movements and heel-shin slide. The change in score from baseline to 6 months, 9 months, and 12 months will be reported.
11 secondary outcomes
Average Gait Speed
Time frame: Baseline, 6 months, 9 months, 12 months
Dynamic Gait Index Score
Time frame: Baseline, 6 months, 9 months, 12 months
Timed Up and Go (TUG)
Time frame: Baseline, 6 months, 9 months, 12 months
Fatigue Severity Scale (FSS) Score
Time frame: Baseline, 6 months, 9 months, 12 months
Quality of Life (QOL) - Physical Health
Time frame: Baseline, 6 months, 9 months, 12 months
Quality of Life (QOL) - Psychological
Time frame: Baseline, 6 months, 9 months, 12 months
Quality of Life (QOL) - Social Relationships
Time frame: Baseline, 6 months, 9 months, 12 months
Quality of Life (QOL) - Environment
Time frame: Baseline, 6 months, 9 months, 12 months
Prevalence of Desired Changes in Diffuse Tensor Imaging
Time frame: 6 months
Prevalence of Cerebellar Volume
Time frame: Baseline, 6 months, 9 months, 12 months
Prevalence of Desired Changes in Resting State fMRI Scans
Time frame: 6 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Nov 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40946705Barbuto S, Lee S, Stein J, Kuo SH, Quinn L, Spinner M, Stern Y. Home Training for Cerebellar Ataxias: A Randomized Clinical Trial. JAMA Neurol. 2025 Nov 1;82(11):1162-1170. doi: 10.1001/jamaneurol.2025.3421.
- Individual participant dataSharing planned: Undecided
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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.
Scott M Barbuto, MD principal investigator
Assistant Professor of Rehabilitation and Regenerative Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Aerobic Training and Balance Training in people with Cerebellar Ataxia, run by Columbia University at 1 site in United States.
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 4 inclusion and 4 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 4 Nov 2025. 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 United States. 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.
Are results available for this study?
Yes. Results are posted in the registry (first posted 4 Nov 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Change in Assessment and Rating of Ataxia (SARA) Score (time frame: Baseline, 6 months, 9 months, 12 months). 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 NCT05002218, which also lists 1 linked publication.
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 4 Nov 2025.