The primary objective of the study is to assess the pharmacokinetics (PK) and safety of vatiquinone administered in participants with Friedreich ataxia (FA) younger than 7 years.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
5
Vatiquinone will be administered per dose and schedule specified in the arm.
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Plasma Concentration of Vatiquinone
Time frame: Pre-morning dose (0 hour) at Week 4; 1 to 3 hours and 3 to 6 hours post-morning dose at Weeks 4, 12, and 24
Area Under the Curve (AUC) of Vatiquinone
Time frame: Pre-morning dose (0 hour) at Week 4; 1 to 3 hours and 3 to 6 hours post-morning dose at Weeks 4, 12, and 24
Number of Participants With Adverse Events
Time frame: Baseline up to Week 76
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