Friedreich's AtaxiaNCT07444333
Cardiac Output and Fatigue in Friedreich's Ataxia
A study testing Aerobic Exercise in people with Friedreich's Ataxia, run by Scott Barbuto at 1 site in United States.
Trial summary
This is a clinical trial examining to impact of aerobic training plus omaveloxolone in FRDA. Thirty individuals with FRDA will be recruited; 20 individuals will be on omaveloxolone treatment whereas the other ten individuals will not. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Friedreich's Ataxia
- What is being tested?
- Aerobic Exercise
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Jul 2026to 1 Apr 2028
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Genetically confirmed FRDA
- Ability to safely ride a stationary bicycle (mFARS sitting posture sub-score <2)
Who cannot join
- Beck depression score >19, a score that precludes ability to exercise.
- Montreal Cognitive Assessment (MoCA) score <23/30
- Disorders that interfere with ability to perform endurance exercise (e.g., stroke, respiratory problems, traumatic brain injury, or neuromuscular disease).
- Regular participation in vigorous endurance exercise (defined as >2 days/week for at least the past 4 months at max HR>65%).
- Evidence of serious arrhythmias or ischemic heart disease.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Columbia University Irving Medical Center
New York, New York, 10032
Scott M Barbuto, MD PhD, 212-305-3535, sb3779@cumc.columbia.edu
Scott M Barbuto, MD PhD
About this study
This is a clinical trial examining to impact of aerobic training plus omaveloxolone in FRDA. Thirty individuals with FRDA will be recruited; 20 individuals will be on omaveloxolone treatment whereas the other ten individuals will not. Individuals will undergo baseline assessment including cardiopulmonary exercise testing (CPET), mFARS, gait speed, Timed Up and Go (TUG), Fatigue Severity Scale (FSS), Fatigue Impact Scale (FIS), and 6-minute Walk Test (6MWT). Individuals will then perform 3-months of home aerobic training. Repeat assessments will be conducted at 3- and 6-months.
Read the detailed description
In February 2023, omaveloxolone became the first Food and Drug Administration (FDA) approved medication for Friedreich's Ataxia (FRDA). In its pivotal study, named MOXIe, researchers found that omaveloxolone significantly improved neurological function compared to placebo. The primary outcome measure was the modified Friedreich's Ataxia Rating Scale (mFARS), the gold standard for measuring ataxia symptoms in clinical trials for FRDA. However, there are several limitations with the use of mFARS:
- It does not detect subtle disease progression over short time frames. For example, in MOXIe, differences between placebo and omaveloxolone were not detected until 48 weeks.
- Some of the components of mFARS rely on clinical judgment, which can introduce variability between raters.
- The minimal clinical important difference (MCID) for mFARS has not been clearly defined.
- The scale lacks sensitivity to detect changes in individuals with minimal symptoms or advanced disease.
Given these limitations, development of additional outcome measures for clinical trials to determine the impact of drugs like omaveloxolone is desirable. In this study, investigators propose using maximal oxygen consumption (VO2max) as a future primary outcome measure for FRDA clinical trials. VO2max offers several advantages as an outcome measure:
- It is a direct physiological measure of aerobic capacity, reducing subjectivity compared to clinical scales like mFARS.
- It can detect subtle changes in aerobic function making it useful for monitoring intervention effects over time.
- It has a defined MCID, and higher VO2max is associated with better health outcomes making it a clinically meaningful endpoint.
- It is non-invasive and reproducible.
To examine the utility of VO2max as a primary outcome measure, this study will recruit 30 individuals with FRDA in this pilot study. Twenty individuals will be on omaveloxolone treatment whereas the other 10 individuals will not. Individuals will undergo baseline cardiopulmonary exercise testing (CPET) to determine VO2max. Secondary measures will include cardiac output. All participants will be given a recumbent bicycle for home use. Participants will be expected to cycle 30-minutes per session, 5x per week at 65-75% maximum heart rate (moderate intensity) for 3-months. To track training, individuals will be given a Fitbit Charge 6, and an exercise physiologist will call every two weeks to adjust training as needed. To determine benefits of training with omaveloxolone, repeat assessments will occur at 3- and 6-months.
AIM 1: Acceptability and Feasibility of Home Aerobic Exercise. Individuals with other types of ataxia have been able to train at the above levels safely. We hypothesize that there will be no serious adverse events related to aerobic training, and there will be an acceptable number of minor adverse events. The investigators further hypothesize that drop-out from the trial will be less than 25%.
AIM 2: Impact of Omaveloxolone on VO2max. Omaveloxolone works by activating and preventing the degradation of Nuclear factor-like 2 (Nrf2), which helps prevent oxidative damage within the mitochondria of individuals with FRDA. Improved mitochondrial function should significantly enhance VO2max by increasing ATP production and improving the rate of oxygen consumption. Thus, the investigators hypothesize that individuals on omaveloxolone will have a significantly larger increase in VO2max after the aerobic training when compared to individuals who are not on omaveloxolone.
AIM 3: Impact of Aerobic Training + Omaveloxolone on Fatigue. Omaveloxolone has been shown to cause a transient (12-week) increase in fatigue. Aerobic training, on the other hand, is known to improve fatigue in individuals with other hereditary ataxias. For this aim, the primary outcome measure will be the Fatigue Severity Scale (FSS) with secondary measures of Fatigue Impact Scale (FIS) and 6-minute walk test (6MWT). The investigators hypothesize improved fatigue with the incorporation of aerobic training and that individuals in the omaveloxolone group will have less fatigue than those not on omaveloxolone.
This pilot study will provide the foundation for the use of VO2max as an outcome measure in future FRDA trials. It will also examine the synergistic effect of omaveloxolone and aerobic training which may show greater improvement than either intervention alone, including improvements in fatigue.
- Conditions
- Friedreich's Ataxia
- Being tested
- Aerobic Exercise
- Keywords
- Friedreich's ataxia, Aerobic exercise, Omaveloxolone, Fatigue, Cardiac Output
- Medical subject headings
- Friedreich Ataxia, Fatigue
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Aerobic ExerciseBehavioural
Both groups will receive aerobic training. The difference will be whether individuals are on or off omaveloxolone.
| Study group | Role | What they receive |
|---|---|---|
| Home Aerobic Training on Omaveloxolone | Experimental | Participants who are currently taking Skyclarys (omaveloxolone) will be given a recumbent exercise bike (Marcy ME-709 Adjustable Recumbent Exercise Bike, Pomona, CA) for home use. They will be instructed to exercise five times a week. The regimen will include 5-minute warm up, 30 minutes of exercise at target heart rate, and 5 minutes of cool down. Receives: Behavioral: Aerobic Exercise |
| Home Aerobic Training off Omaveloxolone | Active comparator | Participants who are not taking Skyclarys (omaveloxolone) will be given a recumbent exercise bike (Marcy ME-709 Adjustable Recumbent Exercise Bike, Pomona, CA) for home use. They will be instructed to exercise five times a week. The regimen will include 5-minute warm up, 30 minutes of exercise at target heart rate, and 5 minutes of cool down. Receives: Behavioral: Aerobic Exercise |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events (Primary outcome for Aim 1)
Adverse events are defined as exercise-related discomforts (muscle and joint pain), minor injuries (strains, sprains), and non-injurious falls. Serious adverse events are defined as hospitalization, surgery, death, or permanent disability. We will compare adverse and serious adverse events for both groups.
Measured: 0, 3, 6 months
Maximal Oxygen Consumption (primary outcome measure for Aim 2)
Maximal oxygen consumption (VO2max) will be determined by a breath-by-breath measurement of VO2 with a Vmax Encore Metabolic System, while participants perform a progressive ramped exercise test using an electronic-braked lower body cycle ergometer (CareFusion Corp, San Diego, CA). The minimal clinically important difference (MCID) has been determined to be between 1.0-2.0 mL/kg/min.
Measured: 0, 3-, 6-months
Fatigue Severity scale (primary outcome measure for Aim 3)
A commonly used measure to assess fatigue in a variety of populations. A score change of 3.5 to 4.5 points is considered clinically meaningful.
Measured: 0, 3-, and 6-months
Secondary outcomes (7)
6-Minute Walk Test
A functional measure of exercise capacity, we will measure the maximum distance a participant can walk in 6 minutes over a 25-meter linear course. The MCID has been determined to be between 14.0 and 30.5 meters. Fatigability will be assessed comparing the distance traveled during the last minute compared to the first minute. This difference will be divided by distance walked in the first minute and expressed as a percentage.
Measured: 0, 3-, and 6-months
Fatigue Impact Scale
This scale assesses the perceived impact of fatigue on physical, cognitive, and psychosocial functioning during the participant's past four weeks. An MCID of 3.9 to 8.1 has been determined for other movement disorders such as Parkinson's Disease and Multiple Sclerosis.
Measured: 0, 3-, 6-months
Cardiac Output
PhysioFlow is a non-invasive device that allows you to determine cardiac output through impedence cardiography. It is FDA approved to determine cardiac output. Cardiac output will be determined both at rest and at maximal exertion during CPET testing.
Measured: 0, 3-, and 6-months
Timed Up and Go
To monitor balance and gait, TUG will be performed three times using the standard protocol, and the results will be averaged. Participants will be instructed to perform this test using the typical assistive device they use to walk outside if applicable. This test has been shown to be a reliable measure of balance in individuals with ataxia.
Measured: 0, 3-, and 6-months
Gait Speed
Gait Speed will be determined by asking participants to walk as fast as possible on a 10-meter runway three times, and the times will be averaged. The MCID has been determined to be between 0.1m/s to 0.2 m/s for various patient populations.
Measured: 0, 3-, 6-months
mFARS
mFARS is a validated measure of ataxia symptoms in individuals with FRDA. Scores range from 0-93 points, with higher scores indicating more ataxia. Research has shown the mFARS has good internal consistency, interrater and test-retest reliability. The minimal clinically important difference is not clearly defined, however.
Measured: 0, 3-, 6-months
Exercise Adherence
We will determine the average number of days per week exercised and duration of the exercise at target heart rate. The measure for achieving levels of exercise intensity is derived from the average heart rate during an exercise session expressed as a percentage of the maximal heart rate for the individual.
Measured: 0, 3-, 6-months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Mar 2026
- Study start15 Jul 2026
- Primary completion1 Feb 2028 (estimated)
- Study completion1 Apr 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Friedreich's AtaxiaStudies of the same condition, open and under way.
- 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
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- This is a clinical trial examining to impact of aerobic training plus omaveloxolone in FRDA. Thirty individuals with FRDA will be recruited; 20 individuals will be on omaveloxolone treatment whereas the other ten individuals will not. Individuals will undergo baseline assessment including cardiopulmonary exercise testing (CPET), mFARS, gait speed, Timed Up and Go (TUG), Fatigue Severity Scale (FSS), Fatigue Impact Scale (FIS), and 6-minute Walk Test (6MWT). Individuals will then perform 3-months of home aerobic training. Repeat assessments will be conducted at 3- and 6-months.
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Scott Barbuto
- Sponsor type
- University, hospital or other
- Collaborators
- Biogen
- Responsible party
- Sponsor-investigator, Scott Barbuto, Columbia University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07444333
- Sponsor's protocol ID
- AAAV7687
- Other IDs
- US-SKY-12378
- Status last verified
- Sep 2026
- First submitted
- 17 Nov 2025
- Last updated
- 22 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Data will be made available upon request
Study at a glance
From the registry record, last updated by the sponsor on 22 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cardiac Output and Fatigue in Friedreich's Ataxia
- ClinicalTrials.gov ID
- NCT07444333
- Acronym / sponsor ID
- AAAV7687
- Conditions
- Friedreich's Ataxia
- Keywords
- Friedreich's ataxia, Aerobic exercise, Omaveloxolone, Fatigue, Cardiac Output
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Scott BarbutoUniversity, hospital or other
- Collaborators
- Biogen
- Study start
- 15 Jul 2026 (actual)
- Primary completion
- 1 Feb 2028 (estimated)
- Study completion
- 1 Apr 2028 (estimated)
- First posted
- 2 Mar 2026 (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 a clinical trial examining to impact of aerobic training plus omaveloxolone in FRDA. Thirty individuals with FRDA will be recruited; 20 individuals will be on omaveloxolone treatment whereas the other ten individuals will not. Individuals will undergo baseline assessment including cardiopulmonary exercise testing (CPET), mFARS, gait speed, Timed Up and Go (TUG), Fatigue Severity Scale (FSS), Fatigue Impact Scale (FIS), and 6-minute Walk Test (6MWT). Individuals will then perform 3-months of home aerobic training. Repeat assessments will be conducted at 3- and 6-months.
Detailed description
In February 2023, omaveloxolone became the first Food and Drug Administration (FDA) approved medication for Friedreich's Ataxia (FRDA). In its pivotal study, named MOXIe, researchers found that omaveloxolone significantly improved neurological function compared to placebo. The primary outcome measure was the modified Friedreich's Ataxia Rating Scale (mFARS), the gold standard for measuring ataxia symptoms in clinical trials for FRDA. However, there are several limitations with the use of mFARS:
- It does not detect subtle disease progression over short time frames. For example, in MOXIe, differences between placebo and omaveloxolone were not detected until 48 weeks.
- Some of the components of mFARS rely on clinical judgment, which can introduce variability between raters.
- The minimal clinical important difference (MCID) for mFARS has not been clearly defined.
- The scale lacks sensitivity to detect changes in individuals with minimal symptoms or advanced disease.
Given these limitations, development of additional outcome measures for clinical trials to determine the impact of drugs like omaveloxolone is desirable. In this study, investigators propose using maximal oxygen consumption (VO2max) as a future primary outcome measure for FRDA clinical trials. VO2max offers several advantages as an outcome measure:
- It is a direct physiological measure of aerobic capacity, reducing subjectivity compared to clinical scales like mFARS.
- It can detect subtle changes in aerobic function making it useful for monitoring intervention effects over time.
- It has a defined MCID, and higher VO2max is associated with better health outcomes making it a clinically meaningful endpoint.
- It is non-invasive and reproducible.
To examine the utility of VO2max as a primary outcome measure, this study will recruit 30 individuals with FRDA in this pilot study. Twenty individuals will be on omaveloxolone treatment whereas the other 10 individuals will not. Individuals will undergo baseline cardiopulmonary exercise testing (CPET) to determine VO2max. Secondary measures will include cardiac output. All participants will be given a recumbent bicycle for home use. Participants will be expected to cycle 30-minutes per session, 5x per week at 65-75% maximum heart rate (moderate intensity) for 3-months. To track training, individuals will be given a Fitbit Charge 6, and an exercise physiologist will call every two weeks to adjust training as needed. To determine benefits of training with omaveloxolone, repeat assessments will occur at 3- and 6-months.
AIM 1: Acceptability and Feasibility of Home Aerobic Exercise. Individuals with other types of ataxia have been able to train at the above levels safely. We hypothesize that there will be no serious adverse events related to aerobic training, and there will be an acceptable number of minor adverse events. The investigators further hypothesize that drop-out from the trial will be less than 25%.
AIM 2: Impact of Omaveloxolone on VO2max. Omaveloxolone works by activating and preventing the degradation of Nuclear factor-like 2 (Nrf2), which helps prevent oxidative damage within the mitochondria of individuals with FRDA. Improved mitochondrial function should significantly enhance VO2max by increasing ATP production and improving the rate of oxygen consumption. Thus, the investigators hypothesize that individuals on omaveloxolone will have a significantly larger increase in VO2max after the aerobic training when compared to individuals who are not on omaveloxolone.
AIM 3: Impact of Aerobic Training + Omaveloxolone on Fatigue. Omaveloxolone has been shown to cause a transient (12-week) increase in fatigue. Aerobic training, on the other hand, is known to improve fatigue in individuals with other hereditary ataxias. For this aim, the primary outcome measure will be the Fatigue Severity Scale (FSS) with secondary measures of Fatigue Impact Scale (FIS) and 6-minute walk test (6MWT). The investigators hypothesize improved fatigue with the incorporation of aerobic training and that individuals in the omaveloxolone group will have less fatigue than those not on omaveloxolone.
This pilot study will provide the foundation for the use of VO2max as an outcome measure in future FRDA trials. It will also examine the synergistic effect of omaveloxolone and aerobic training which may show greater improvement than either intervention alone, including improvements in fatigue.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
- Model description
- This is a clinical trial examining to impact of aerobic training plus omaveloxolone in FRDA. Thirty individuals with FRDA will be recruited; 20 individuals will be on omaveloxolone treatment whereas the other ten individuals will not. Individuals will undergo baseline assessment including cardiopulmonary exercise testing (CPET), mFARS, gait speed, Timed Up and Go (TUG), Fatigue Severity Scale (FSS), Fatigue Impact Scale (FIS), and 6-minute Walk Test (6MWT). Individuals will then perform 3-months of home aerobic training. Repeat assessments will be conducted at 3- and 6-months.
| Arm | Type | Receives |
|---|---|---|
| Home Aerobic Training on Omaveloxolone | Experimental | Behavioral: Aerobic ExerciseParticipants who are currently taking Skyclarys (omaveloxolone) will be given a recumbent exercise bike (Marcy ME-709 Adjustable Recumbent Exercise Bike, Pomona, CA) for home use. They will be instructed to exercise five times a week. The regimen will include 5-minute warm up, 30 minutes of exercise at target heart rate, and 5 minutes of cool down. |
| Home Aerobic Training off Omaveloxolone | Active comparator | Behavioral: Aerobic ExerciseParticipants who are not taking Skyclarys (omaveloxolone) will be given a recumbent exercise bike (Marcy ME-709 Adjustable Recumbent Exercise Bike, Pomona, CA) for home use. They will be instructed to exercise five times a week. The regimen will include 5-minute warm up, 30 minutes of exercise at target heart rate, and 5 minutes of cool down. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Genetically confirmed FRDA
- Ability to safely ride a stationary bicycle (mFARS sitting posture sub-score <2)
Key exclusion criteria (5 listed)
- Beck depression score >19, a score that precludes ability to exercise.
- Montreal Cognitive Assessment (MoCA) score <23/30
- Disorders that interfere with ability to perform endurance exercise (e.g., stroke, respiratory problems, traumatic brain injury, or neuromuscular disease).
- Regular participation in vigorous endurance exercise (defined as >2 days/week for at least the past 4 months at max HR>65%).
- Evidence of serious arrhythmias or ischemic heart disease.
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 ExerciseBoth groups will receive aerobic training. The difference will be whether individuals are on or off omaveloxolone. | Behavioural | Home Aerobic Training off Omaveloxolone, Home Aerobic Training on Omaveloxolone |
Primary outcome
Adverse events (Primary outcome for Aim 1)
Time frame: 0, 3, 6 months
Adverse events are defined as exercise-related discomforts (muscle and joint pain), minor injuries (strains, sprains), and non-injurious falls. Serious adverse events are defined as hospitalization, surgery, death, or permanent disability. We will compare adverse and serious adverse events for both groups.
Maximal Oxygen Consumption (primary outcome measure for Aim 2)
Time frame: 0, 3-, 6-months
Maximal oxygen consumption (VO2max) will be determined by a breath-by-breath measurement of VO2 with a Vmax Encore Metabolic System, while participants perform a progressive ramped exercise test using an electronic-braked lower body cycle ergometer (CareFusion Corp, San Diego, CA). The minimal clinically important difference (MCID) has been determined to be between 1.0-2.0 mL/kg/min.
Fatigue Severity scale (primary outcome measure for Aim 3)
Time frame: 0, 3-, and 6-months
A commonly used measure to assess fatigue in a variety of populations. A score change of 3.5 to 4.5 points is considered clinically meaningful.
7 secondary outcomes
6-Minute Walk Test
Time frame: 0, 3-, and 6-months
Fatigue Impact Scale
Time frame: 0, 3-, 6-months
Cardiac Output
Time frame: 0, 3-, and 6-months
Timed Up and Go
Time frame: 0, 3-, and 6-months
Gait Speed
Time frame: 0, 3-, 6-months
mFARS
Time frame: 0, 3-, 6-months
Exercise Adherence
Time frame: 0, 3-, 6-months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Sep 2026.
- Individual participant dataSharing planned: Yes. Data will be made available upon request
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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.
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 Exercise in people with Friedreich's Ataxia, run by Scott Barbuto 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 2 inclusion and 5 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: looking for participants now, as of the sponsor's last update on 22 Sep 2026. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Scott Barbuto, MD PhD (12123054818, sb3779@cumc.columbia.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse events (Primary outcome for Aim 1) (time frame: 0, 3, 6 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 22 Sep 2026.