Scott Barbuto
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: * It does not detect subtle disease progression over short time frames. For example, in MOXIe, diff…
Inclusion Criteria: 1. Genetically confirmed FRDA 2. Ability to safely ride a stationary bicycle (mFARS sitting posture sub-score \<2) Exclusion Criteria: 1. Beck depression score \>19, a score that precludes ability to exercise. 2. Montreal Cognitive Assessment (MoCA) score \<23/30 3. Disorders that interfere with ability to perform endurance exercise (e.g., stroke, respiratory problems, traumatic brain injury, or neuromuscular disease). 4. Regular participation in vigorous endurance exercise (defined as \>2 days/week for at least the past 4 months at max HR\>65%). 5. Evidence of serious a…
Both groups will receive aerobic training. The difference will be whether individuals are on or off omaveloxolone.
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