Training in Ataxia - Individuals With Degenerative Cerebellar Diseases

NCT05002218 | Completed Results DMC

• 2 other identifiers: AAAT83881K23NS121518-01A1
• Study Type: interventional
• Target: 64
• Locations: 1 country
• Sites: 1

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.

Conditions

Cerebellar Ataxia

Keywords

ataxia; cerebellar ataxia; exercise; training; rehabilitation

Outcome Measures

Primary Outcomes (1)

• 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.

  Baseline, 6 months, 9 months, 12 months

Secondary Outcomes (11)

• Average Gait Speed

  Baseline, 6 months, 9 months, 12 months

• Dynamic Gait Index Score

  Baseline, 6 months, 9 months, 12 months

• Timed Up and Go (TUG)

  Baseline, 6 months, 9 months, 12 months

• Fatigue Severity Scale (FSS) Score

  Baseline, 6 months, 9 months, 12 months

• Quality of Life (QOL) - Physical Health

  Baseline, 6 months, 9 months, 12 months

Study Arms (2)

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.

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.

Behavioral: Balance Training

Interventions

Aerobic Training BEHAVIORAL

Aerobic training on stationary bike 5x a week for 30 minutes a day.

Aerobic Training

Balance Training BEHAVIORAL

Balance training 5x a week for 30 minutes as instructed by a therapist. Standard of care.

Balance Training

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosed with spinocerebellar ataxia
• Cerebellar atrophy on MRI
• Prevalence of ataxia on clinical exam
• Ability to safely ride a stationary exercise bike

You may not qualify if:

• Other neurologic conditions
• Heart disease
• Cognitive impairment
• Medical instability

Sponsors & Collaborators

• Columbia University: lead
• National Institute of Neurological Disorders and Stroke (NINDS): collaborator

Study Sites (1)

Columbia University Irving Medical Center

New York, New York, 10032, United States

Location

Related Publications (1)

• 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.

  PMID: 40946705 DERIVED

MeSH Terms

Conditions

Cerebellar Ataxia; Ataxia; Motor Activity

Condition Hierarchy (Ancestors)

Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Dyskinesias; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Behavior

Limitations and Caveats

The study had a small sample size and was conducted during the COVID-19 pandemic, precluding cardiopulmonary exercise testing. Recruitment was largely limited to individuals with multiple system atrophy-cerebellar type and idiopathic cerebellar ataxia, as individuals with other types of ataxia were recruited for other competing trials. Training adherence relied on training logs with no objective measures.

Results Point of Contact

• Title: Scott Barbuto, MD
• Organization: Columbia University Irving Medical Center

sb3779@cumc.columbia.edu

518-275-9588

Study Officials

• Scott M Barbuto, MD

  Assistant Professor of Rehabilitation and Regenerative Medicine

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: CARE PROVIDER, OUTCOMES ASSESSOR
• Masking Details: Care providers and outcome assessor will not know which group individuals are assigned. In addition, the primary outcome will be video-recorded and the videos will be scrambled so that outcome assessor will not know if the test was done pre- or post-training.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor of Rehabilitation and Regenerative Medicine

Model Details: This is a single-blinded randomized control trial.

Study Record Dates

• First Submitted: August 4, 2021
• First Posted: August 12, 2021
• Study Start: January 31, 2022
• Primary Completion: November 6, 2024
• Study Completion: November 6, 2024
• Last Updated: November 4, 2025
• Results First Posted: November 4, 2025

Record last verified: 2025-08

Locations

US (1)