The primary objective of the study is to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale \[mFARS\]) and safety of vatiquinone in participants with Friedreich ataxia (FA).
During the double-blind, placebo-controlled phase, participants will be stratified by baseline mFARS score (\<40 versus ≥40), age of disease onset (\<14 versus ≥14), and age at screening (≤21 years or \>21 years) and randomized to receive either vatiquinone or placebo using interactive response technology (IRT). Following completion of the randomized, double-blind, placebo-controlled phase (72 weeks), participants will enter into an open-label extension phase (24 weeks) during which they will receive open-label treatment with vatiquinone at the dose they received in the randomized phase of the study (for participants entering the extension phase who initially received placebo, the dose of vatiquinone will be determined based on age and weight) and then a safety follow-up (approximately 30 days \[±5 days\] after last dose or termination visit, whichever is later). The primary efficacy analysis will be based on change from baseline in mFARS score of participants between 7 and 21 years old. In order to explore the treatment efficacy and safety, approximately an additional 20 participants \>21 years of age will be randomized for a total of approximately 126 participants.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
146
Vatiquinone will be administered per dose and schedule specified in the arm.
Placebo will be administered per schedule specified in the arm.
UCLA
Los Angeles, California, United States
University of Florida
Gainesville, Florida, United States
Change From Baseline in the Modified Friedreich Ataxia Rating Scale (mFARS) Score at Week 72
Friedreich Ataxia Rating Scale (FARS) is a disease-specific scale that measures progression of neurological effects of FA. The mFARS is a validated and reliable 93- item scale; comprised of the neurologic component of the FARS and evaluates bulbar, upper limb, lower limb, and upright stability/gait function. For each item, responses categorize the corresponding neurological finding, and the findings are assigned a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment.
Time frame: Baseline, Week 72
Change From Baseline in Friedreich Ataxia Rating Scale Activities of Daily Living (FARS-ADL) Score at Week 72
The FARS-ADL is a subsection of the FARS questionnaire that assesses activities of daily living, including speech, personal hygiene, feeding, and mobility. Participants rank each category using a scale of 0 (normal) to 4 (severe disability/ inability to carry out activity independently), with lower scores indicative of "normal" function/activity.
Time frame: Baseline, Week 72
Change From Baseline in 1-Minute Walk Test (1MWT) at Week 72
The 1MWT is a timed performance test used to measure functional ability, walking endurance, balance, and muscle performance by measuring maximal walking speed in 1 minute. Participants will be instructed to walk as quickly as possible for 1 minute without running. Maximal walking speed will be measured upon completion of the walk and recorded.
Time frame: Baseline, Week 72
Number of Falls Through Week 72
The fall log directly relate to a participant's ability to ambulate during normal daily activities. Thus, each participant will be required to maintain a fall log, which will include the date and time of each fall. Falls as defined by World Health Organization as "inadvertently coming to rest on the ground, floor or other lower level, excluding intentional change in position to rest in furniture, wall or other objects," will be reported.
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University of South Florida
Tampa, Florida, United States
University of Iowa
Iowa City, Iowa, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Murdoch Children's Research Institute
Parkville, Victoria, Australia
University of Campinas (UNICAMP) - School of Medical Sciences, Dept of Neurology
São Paulo, Brazil
Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)
Montreal, Quebec, Canada
CHU Sainte-Justine
Montreal, Quebec, Canada
Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute)
Paris, France
...and 4 more locations
Time frame: Baseline through Week 72