Trial Outcomes & Findings for Training in Ataxia - Individuals With Degenerative Cerebellar Diseases (NCT NCT05002218)

Last Updated: 2025-11-04

Results Overview

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.

Recruitment status

COMPLETED

Study phase

Target enrollment

64 participants

Primary outcome timeframe

Baseline, 6 months, 9 months, 12 months

Results posted on

2025-11-04

Participant Flow

64 participants were enrolled. Of those, 2 participants withdrew from the study prior to randomization.

Participant milestones

Participant milestones
Measure	Aerobic Training 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.	Balance Training 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.
Overall Study STARTED	31	31
Overall Study COMPLETED	26	25
Overall Study NOT COMPLETED	5	6

Reasons for withdrawal

Reasons for withdrawal
Measure	Aerobic Training 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.	Balance Training 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.
Overall Study Lost to Follow-up	5	6

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Aerobic Training n=31 Participants 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.	Balance Training n=31 Participants 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.	Total n=62 Participants Total of all reporting groups
Age, Continuous	55.0 years STANDARD_DEVIATION 12.5 • n=31 Participants	53.8 years STANDARD_DEVIATION 13.5 • n=31 Participants	54.4 years STANDARD_DEVIATION 12.9 • n=62 Participants
Sex: Female, Male Female	16 Participants n=31 Participants	13 Participants n=31 Participants	29 Participants n=62 Participants
Sex: Female, Male Male	15 Participants n=31 Participants	18 Participants n=31 Participants	33 Participants n=62 Participants
Race and Ethnicity Not Collected	—	—	0 Participants Race and Ethnicity were not collected from any participant.
Region of Enrollment United States	31 Participants n=31 Participants	31 Participants n=31 Participants	62 Participants n=62 Participants
Disease duration	8.2 years STANDARD_DEVIATION 7.0 • n=31 Participants	6.8 years STANDARD_DEVIATION 6.8 • n=31 Participants	7.6 years STANDARD_DEVIATION 6.9 • n=62 Participants
Disease type Spinocerebellar ataxia type 1 (SCA1)	2 Participants n=31 Participants	5 Participants n=31 Participants	7 Participants n=62 Participants
Disease type SCA2	5 Participants n=31 Participants	4 Participants n=31 Participants	9 Participants n=62 Participants
Disease type SCA3	4 Participants n=31 Participants	2 Participants n=31 Participants	6 Participants n=62 Participants
Disease type SCA4	1 Participants n=31 Participants	1 Participants n=31 Participants	2 Participants n=62 Participants
Disease type SCA6	5 Participants n=31 Participants	3 Participants n=31 Participants	8 Participants n=62 Participants
Disease type SCA7	0 Participants n=31 Participants	1 Participants n=31 Participants	1 Participants n=62 Participants
Disease type SCA8	2 Participants n=31 Participants	4 Participants n=31 Participants	6 Participants n=62 Participants
Disease type SCA14	1 Participants n=31 Participants	0 Participants n=31 Participants	1 Participants n=62 Participants
Disease type SCA15	1 Participants n=31 Participants	0 Participants n=31 Participants	1 Participants n=62 Participants
Disease type SCA19	0 Participants n=31 Participants	1 Participants n=31 Participants	1 Participants n=62 Participants
Disease type SCA27	0 Participants n=31 Participants	1 Participants n=31 Participants	1 Participants n=62 Participants
Disease type Idiopathic	8 Participants n=31 Participants	9 Participants n=31 Participants	17 Participants n=62 Participants
Disease type multiple system atrophy-cerebellar type	2 Participants n=31 Participants	0 Participants n=31 Participants	2 Participants n=62 Participants

PRIMARY outcome

Timeframe: Baseline, 6 months, 9 months, 12 months

Population: Some participants were lost to follow up at 6 months, 9 months, and 12 months, so data could not be collected from those participants.

Outcome measures

Outcome measures
Measure	Aerobic Training n=31 Participants 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.	Balance Training n=31 Participants 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.
Change in Assessment and Rating of Ataxia (SARA) Score Baseline to 12 months	-1.4 score on a scale Interval -2.1 to -0.7	0 score on a scale Interval -0.66 to 0.7
Change in Assessment and Rating of Ataxia (SARA) Score Baseline to 6 months	-2.4 score on a scale Interval -3.1 to -1.8	-0.9 score on a scale Interval -1.5 to -0.2
Change in Assessment and Rating of Ataxia (SARA) Score Baseline to 9 months	-1.5 score on a scale Interval -2.2 to -0.8	-0.2 score on a scale Interval -0.84 to 0.5

SECONDARY outcome

Timeframe: Baseline, 6 months, 9 months, 12 months

Population: Some participants were lost to follow up at 6 months, 9 months, and 12 months, so data could not be collected from those participants.

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).

Outcome measures

Outcome measures
Measure	Aerobic Training n=31 Participants 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.	Balance Training n=31 Participants 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.
Average Gait Speed Baseline	0.90 m/s Standard Error 0.28	0.95 m/s Standard Error 0.32
Average Gait Speed 6 months	0.80 m/s Standard Error 0.26	0.89 m/s Standard Error 0.30
Average Gait Speed 9 months	0.80 m/s Standard Error 0.26	0.80 m/s Standard Error 0.26
Average Gait Speed 12 months	0.80 m/s Standard Error 0.26	0.79 m/s Standard Error 0.26

SECONDARY outcome

Timeframe: Baseline, 6 months, 9 months, 12 months

Population: Some participants were lost to follow up at 6 months, 9 months, and 12 months, so data could not be collected from those participants.

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.

Outcome measures

Outcome measures
Measure	Aerobic Training n=31 Participants 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.	Balance Training n=31 Participants 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.
Dynamic Gait Index Score 12 months	13.3 score on a scale Standard Error 2.0	12.8 score on a scale Standard Error 2.0
Dynamic Gait Index Score Baseline	11.8 score on a scale Standard Error 2.0	11.7 score on a scale Standard Error 2.0
Dynamic Gait Index Score 6 months	12.8 score on a scale Standard Error 2.0	13.2 score on a scale Standard Error 2.0
Dynamic Gait Index Score 9 months	13.0 score on a scale Standard Error 2.0	13.2 score on a scale Standard Error 2.0

SECONDARY outcome

Timeframe: Baseline, 6 months, 9 months, 12 months

Population: Some participants were lost to follow up at 6 months, 9 months, and 12 months, so data could not be collected from those participants.

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.

Outcome measures

Outcome measures
Measure	Aerobic Training n=31 Participants 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.	Balance Training n=31 Participants 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.
Timed Up and Go (TUG) Baseline	17.0 seconds Standard Error 3.1	16.6 seconds Standard Error 3.1
Timed Up and Go (TUG) 6 months	15.5 seconds Standard Error 3.1	15.7 seconds Standard Error 3.1
Timed Up and Go (TUG) 9 months	15.5 seconds Standard Error 3.1	15.7 seconds Standard Error 3.1
Timed Up and Go (TUG) 12 months	16.8 seconds Standard Error 3.1	16.7 seconds Standard Error 3.1

SECONDARY outcome

Timeframe: Baseline, 6 months, 9 months, 12 months

Population: Some participants were lost to follow up at 6 months, 9 months, and 12 months, so data could not be collected from those participants.

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.

Outcome measures

Outcome measures
Measure	Aerobic Training n=31 Participants 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.	Balance Training n=31 Participants 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.
Fatigue Severity Scale (FSS) Score 12 months	35.3 score on a scale Standard Error 5.2	36.2 score on a scale Standard Error 5.2
Fatigue Severity Scale (FSS) Score Baseline	41.2 score on a scale Standard Error 5.1	33.5 score on a scale Standard Error 5.2
Fatigue Severity Scale (FSS) Score 6 months	33.3 score on a scale Standard Error 5.2	35.0 score on a scale Standard Error 5.1
Fatigue Severity Scale (FSS) Score 9 months	35.6 score on a scale Standard Error 5.2	34.8 score on a scale Standard Error 5.2

SECONDARY outcome

Timeframe: Baseline, 6 months, 9 months, 12 months

Population: Some participants were lost to follow up at 6 months, 9 months, and 12 months, so data could not be collected from those participants.

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.

Outcome measures

Outcome measures
Measure	Aerobic Training n=31 Participants 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.	Balance Training n=31 Participants 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.
Quality of Life (QOL) - Physical Health Baseline	55.8 score on a scale Standard Error 7.0	59.1 score on a scale Standard Error 7.1
Quality of Life (QOL) - Physical Health 9 months	57.1 score on a scale Standard Error 7.1	59.8 score on a scale Standard Error 7.2
Quality of Life (QOL) - Physical Health 12 months	57.1 score on a scale Standard Error 7.1	60.7 score on a scale Standard Error 7.2
Quality of Life (QOL) - Physical Health 6 months	54.3 score on a scale Standard Error 7.1	60.1 score on a scale Standard Error 7.1

SECONDARY outcome

Timeframe: Baseline, 6 months, 9 months, 12 months

Population: Some participants were lost to follow up at 6 months, 9 months, and 12 months, so data could not be collected from those participants.

Outcome measures

Outcome measures
Measure	Aerobic Training n=31 Participants 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.	Balance Training n=31 Participants 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.
Quality of Life (QOL) - Psychological Baseline	65.6 score on a scale Standard Error 7.1	68.1 score on a scale Standard Error 7.2
Quality of Life (QOL) - Psychological 6 months	63.1 score on a scale Standard Error 7.2	67.6 score on a scale Standard Error 7.2
Quality of Life (QOL) - Psychological 9 months	68.8 score on a scale Standard Error 7.2	67.0 score on a scale Standard Error 7.2
Quality of Life (QOL) - Psychological 12 months	66.2 score on a scale Standard Error 7.2	66.2 score on a scale Standard Error 7.2

SECONDARY outcome

Timeframe: Baseline, 6 months, 9 months, 12 months

Population: Some participants were lost to follow up at 6 months, 9 months, and 12 months, so data could not be collected from those participants.

Outcome measures

Outcome measures
Measure	Aerobic Training n=31 Participants 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.	Balance Training n=31 Participants 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.
Quality of Life (QOL) - Social Relationships Baseline	60.1 score on a scale Standard Error 8.4	64.8 score on a scale Standard Error 8.5
Quality of Life (QOL) - Social Relationships 6 months	65.5 score on a scale Standard Error 8.4	53.0 score on a scale Standard Error 8.4
Quality of Life (QOL) - Social Relationships 9 months	64.2 score on a scale Standard Error 8.5	60.1 score on a scale Standard Error 8.5
Quality of Life (QOL) - Social Relationships 12 months	65.4 score on a scale Standard Error 8.6	65.7 score on a scale Standard Error 8.5

SECONDARY outcome

Timeframe: Baseline, 6 months, 9 months, 12 months

Population: Some participants were lost to follow up at 6 months, 9 months, and 12 months, so data could not be collected from those participants.

Outcome measures

Outcome measures
Measure	Aerobic Training n=31 Participants 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.	Balance Training n=31 Participants 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.
Quality of Life (QOL) - Environment Baseline	65.5 score on a scale Standard Error 5.7	68.3 score on a scale Standard Error 5.7
Quality of Life (QOL) - Environment 6 months	70.5 score on a scale Standard Error 5.7	66.1 score on a scale Standard Error 5.7
Quality of Life (QOL) - Environment 9 months	69.5 score on a scale Standard Error 5.7	69.6 score on a scale Standard Error 5.7
Quality of Life (QOL) - Environment 12 months	68.9 score on a scale Standard Error 5.7	72.2 score on a scale Standard Error 5.7

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome data not reported

Adverse Events

Aerobic Training

Serious events: 2 serious events

Other events: 8 other events

Deaths: 0 deaths

Balance Training

Serious events: 1 serious events

Other events: 10 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Aerobic Training n=31 participants at risk 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.	Balance Training n=31 participants at risk 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.
Infections and infestations Infection, hospitalized	0.00% 0/31 • AE data was collected during in-person assessments at baseline, 6 months, 9 months, and 12 months	3.2% 1/31 • AE data was collected during in-person assessments at baseline, 6 months, 9 months, and 12 months
Social circumstances Car Accident, hospitalized	6.5% 2/31 • AE data was collected during in-person assessments at baseline, 6 months, 9 months, and 12 months	0.00% 0/31 • AE data was collected during in-person assessments at baseline, 6 months, 9 months, and 12 months

Other adverse events

Other adverse events
Measure	Aerobic Training n=31 participants at risk 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.	Balance Training n=31 participants at risk 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.
Musculoskeletal and connective tissue disorders Muscle Pain	12.9% 4/31 • AE data was collected during in-person assessments at baseline, 6 months, 9 months, and 12 months	3.2% 1/31 • AE data was collected during in-person assessments at baseline, 6 months, 9 months, and 12 months
General disorders Falls	6.5% 2/31 • AE data was collected during in-person assessments at baseline, 6 months, 9 months, and 12 months	25.8% 8/31 • AE data was collected during in-person assessments at baseline, 6 months, 9 months, and 12 months
General disorders Fatigue	6.5% 2/31 • AE data was collected during in-person assessments at baseline, 6 months, 9 months, and 12 months	3.2% 1/31 • AE data was collected during in-person assessments at baseline, 6 months, 9 months, and 12 months
Infections and infestations Infection, not hospitalized	12.9% 4/31 • AE data was collected during in-person assessments at baseline, 6 months, 9 months, and 12 months	12.9% 4/31 • AE data was collected during in-person assessments at baseline, 6 months, 9 months, and 12 months

Additional Information

Scott Barbuto, MD

Columbia University Irving Medical Center

Phone: 518-275-9588

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place