Training in Ataxia - Individuals With Degenerative Cerebellar Diseases
- Conditions
- Cerebellar Ataxia
- Registration Number
- NCT05002218
- Lead Sponsor
- Columbia University
- Brief 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. 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 64
- Diagnosed with spinocerebellar ataxia
- Cerebellar atrophy on MRI
- Prevalence of ataxia on clinical exam
- Ability to safely ride a stationary exercise bike
- Other neurologic conditions
- Heart disease
- Cognitive impairment
- Medical instability
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Primary Outcome Measures
Name Time Method Change in Assessment and Rating of Ataxia (SARA) Score 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.
- Secondary Outcome Measures
Name Time Method Average Gait Speed Baseline, 6 months, 9 months, 12 months 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).
Dynamic Gait Index Score Baseline, 6 months, 9 months, 12 months 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.
Timed Up and Go (TUG) Baseline, 6 months, 9 months, 12 months 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.
Fatigue Severity Scale (FSS) Score Baseline, 6 months, 9 months, 12 months 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.
Quality of Life (QOL) - Physical Health Baseline, 6 months, 9 months, 12 months 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.
Quality of Life (QOL) - Psychological Baseline, 6 months, 9 months, 12 months 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.
Quality of Life (QOL) - Social Relationships Baseline, 6 months, 9 months, 12 months 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.
Quality of Life (QOL) - Environment Baseline, 6 months, 9 months, 12 months 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.
Prevalence of Desired Changes in Diffuse Tensor Imaging 6 months 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.
Prevalence of Cerebellar Volume Baseline, 6 months, 9 months, 12 months 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.
Prevalence of Desired Changes in Resting State fMRI Scans 6 months 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.
Trial Locations
- Locations (1)
Columbia University Irving Medical Center
🇺🇸New York, New York, United States
Columbia University Irving Medical Center🇺🇸New York, New York, United States