The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
120
Vatiquinone will be administered per dose and schedule specified in the arm.
University of California Los Angeles
Los Angeles, California, United States
University of South Florida
Tampa, Florida, United States
University of Iowa
Iowa City, Iowa, United States
Children's Hospital of Philadelphia
Change From Baseline in Total Modified Friedreich's Ataxia Rating Scale (mFARS) Score at Month 24
Time frame: Baseline, Month 24
Change From Baseline in mFARS Subscale Scores (Upright Stability Subscale [USS], Upper Limb [UL], Lower Limb [LL], Bulbar [BUL]) at Month 24
Time frame: Baseline, Month 24
Change From Baseline in Friedreich's Ataxia Rating Scale - Activities of Daily Living (FARS-ADL) Score at Month 24
Time frame: Baseline, Month 24
Change From Baseline in 25-Foot Walk Test (T25FW) at Month 24
Time frame: Baseline, Month 24
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: Baseline up to Month 25
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Philadelphia, Pennsylvania, United States
Hôpital Érasme (Hôpital Universitaire de Bruxelles)
Brussels, Belgium
University of Campinas
Campinas, São Paulo, Brazil
Centre Hospitalier de l'Universite de Montreal CHUM
Montreal, Quebec, Canada
Institut du Cerveau et de la Moelle Epinière
Paris, France
Hospital Sant Joan de Deu
Barcelona, Spain