A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With F… (NCT04577352) | Clinical Trial Compass
CompletedPhase 2/3
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
United States146 participantsStarted 2020-12-17
Plain-language summary
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).
Who can participate
Age range7 Years
SexALL
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Inclusion Criteria:
* mFARS ≥20 to ≤70 at baseline
* Must be able to ambulate at least 10 feet in 1 minute with or without assistance (non-wheelchair).
* Friedreich ataxia diagnosis (homozygous for guanine-adenine-adenine \[GAA\] repeat expansion in intron-1 of frataxin \[FXN\] gene), confirmed by clinical testing (Note: size of GAA repeat is not required for eligibility)
* Consent to comply with study procedures. For participants under the age of 18 (or age of consent), parent(s)/legal guardian(s) of the participant must agree to comply with the requirements of the study, including the need for frequent and prolonged follow up; parent(s)/legal guardian(s) with custody of the participant must give their consent for participant to enroll in the study.
* Difference in the mFARS at screening and baseline of no more than 4 points.
* Must be able to abstain from anticoagulants and any aspirin (including 81 mg) for 30 days prior to the baseline visit and for the duration of the study; any possible discontinuation of anticoagulants should be monitored and indicated by a specialist (for example, cardiologist, neurologist, or hematologist) and discontinuation will be noted by the prescribing physician.
* Must be able to abstain from potent cytochrome P450 (CYP) 3A4 inducers/inhibitors (for example, ketoconazole, rifampin, St. John's wort, grapefruit juice or any grapefruit product) for at least 30 days prior to enrollment
* Must be able to swallow capsules
* Males and females of chil…
What they're measuring
1
Change From Baseline in the mFARS Score at Week 72 - Modified Intent-to-treat (mITT) Analysis Set
Timeframe: Baseline, Week 72
2
Change From Baseline in the mFARS Score at Week 72 - Intent-to-treat (ITT) Analysis Set