Cardiac Output and Fatigue in Friedreich's Ataxia
ConditionFriedreich's Ataxia
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
AgeNot listed
SponsorScott Barbuto
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Genetically confirmed FRDA
Ability to safely ride a stationary bicycle (mFARS sitting posture sub-score <2)
Disqualifiers
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%).
Trial design
Design model
Parallel
Treatments tested in this trial
Aerobic Exercise
BehavioralBoth groups will receive aerobic training. The difference will be whether individuals are on or off omaveloxolone.
Treatment groups
30 Participants
are divided into 2 treatment groupsGroup A: Home Aerobic Training on OmaveloxoloneExperimental treatment 1 intervention
Group B: Home Aerobic Training off OmaveloxoloneActive comparator 1 intervention
Trial outcomes
Primary outcomes
1
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 months2
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-months3
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-monthsSecondary outcomes
1
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-months2
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-months3
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-months4
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-monthsSponsors and contacts
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Source ClinicalTrials.gov