An interventional study of Aerobic Exercise in Friedreich's Ataxia, sponsored by Scott Barbuto. Recruiting at 1 site in United States. Per ClinicalTrials.gov, last updated 2026-09-22.
Sponsored by Scott Barbuto · Not applicable, Interventional, and Treatment
Among 70 studies in Friedreich Ataxia with a site in United States — 17 open to participants now, 24 under way, 4 of them phase 3. Friedreich Ataxia · United States.
Among the 70 studies in Friedreich Ataxia with a site in United States, this one is the 26th largest by planned enrollment of the 48 interventional ones that give a count, is one of 7 that started in 2026, is one of 5 with no phase, reaches primary completion later than 9 of the 23 other live ones, and was the 64th registered.
110 studies on the registry are indexed under Friedreich Ataxia; 27 are open to participants now.
Browse Friedreich Ataxia studies →17 of the 70 studies in Friedreich Ataxia with a site in United States are open now. Recruiting first, the newest first:
Biogen · Sites in United States
Larimar Therapeutics, Inc. · Sites in United States
Lexeo Therapeutics · Sites in United States
Solid Biosciences Inc. · Sites in United States
Lexeo Therapeutics · Sites in United States, Brazil, Canada and 5 more countries
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 18 Friedreich Ataxia studies recruiting now, 9 (50%) set no upper age limit and 9 (50%) stop at 75 or younger; 15 take children under 18; 3 take healthy volunteers.
This study: all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
70 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 17 · Phase 2 19 · Phase 3 12
First in 2001; this study’s year is marked. 7 started in 2026.
This study's planned enrollment of 30 is close to the median of 30 across 75 interventional studies indexed under Friedreich Ataxia.
Median enrollment 33 across the 48 interventional studies, 65 across the 21 observational. Of the 43 that completed or were terminated, 16 (37%) have results posted.
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38 of 70 have an industry lead sponsor. The 10 most frequent:
This is the only study on the registry with Scott Barbuto as lead sponsor.
Scott Barbuto has 1 study filed under Friedreich Ataxia — fewer than ten, so there is no comparison to draw.
6 of the studies in Friedreich Ataxia with a site in United States that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT06953583Phase 3BiogenA Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia | 2027-11-16 |
| NCT06447025Larimar Therapeutics, Inc.An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia | 2027-01 |
| NCT07869654Adjuvia Therapeutics, Inc.A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ATI-105 in Healthy Adults | 2027-05-27 |
| NCT07870590Graviton Bioscience CorporationGV101 for Friedreich's Ataxia (FA) Treatment | 2027-07 |
| NCT02316314Weill Medical College of Cornell UniversityCharacterization of the Cardiac Phenotype of Friedreich's Ataxia (FRDA) | 2027-08 |
Among the 70 studies in Friedreich Ataxia with a site in United States, from their registry updates:
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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.
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:
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:
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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Short tests of memory and thinking, each scored out of 30. Lower scores suggest more difficulty; criteria give a range, either to include people with memory problems or to leave them out.
MMSE and MoCA
Short tests of memory and thinking, each scored out of 30. Lower scores suggest more difficulty; criteria give a range, either to include people with memory problems or to leave them out.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Behavioral: Aerobic Exercise
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.
Behavioral: Aerobic Exercise
Both groups will receive aerobic training. The difference will be whether individuals are on or off omaveloxolone.
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.
Time frame: 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.
Time frame: 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.
Time frame: 0, 3-, and 6-months
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 0, 3-, 6-months
Plan to share: Yes — Data will be made available upon request
Supporting information: Study protocol, Statistical analysis plan
Time frame: It will be available after study completion around 12/14/2027
Access criteria: They will have access to the study protocol and de-identified data. It will be deposited in an NIH database.
No publications or documents are linked to this record.
Started Jul 2026 (actual). Primary completion is estimated for Feb 2028, 18 months after the start.
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