The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression. This is an open label study with comparison to natural history controls. All participants will receive vatiquinone. The primary outcome measure is change in modified Friedreich’s Ataxia Rating Scale (mFARS), a measure of neurological function, after 24 months.
Anyone considering participating in a clinical trial should discuss the matter with their physician. FARA does not endorse or recommend any particular studies.
Study Details:
Key Inclusion Criteria:
- Diagnosed with Friedreich’s ataxia with two GAA repeat expansions
- Ages 7-21 years
Key Exclusion Criteria:
- Individuals with FA who have point mutations, deletions, or other non-GAA expansion mutations
- Current use of omaveloxolone (SKYCLARYS)
- Current use of an investigational treatment
- Individuals who are already participating in an ongoing vatiquinone study or previously took vatiquinone
Additional inclusion and exclusion criteria apply and will be evaluated by a study doctor.