Phase 3 Friedreich Ataxia Trial, Completed

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• You have a confirmed diagnosis of Friedreich's ataxia caused by a specific type of genetic change (a GAA repeat expansion in both copies of the frataxin gene), proven by genetic testing.
• Your disease severity score (called mFARS) falls between 20 and 70 at the start of the study, and your score stays stable (within 4 points) between your screening visit and your baseline visit.
• You are able to walk at least 10 feet within 1 minute, with or without help, and do not use a wheelchair full-time.
• You are willing and able to follow all study procedures; if you are under 18 (or the local age of consent), a parent or legal guardian must also agree to the study requirements.
• You are able to stop taking blood thinners (anticoagulants) and all forms of aspirin (including low-dose 81 mg aspirin) for 30 days before the study starts and throughout the entire study, under the supervision of a relevant specialist.
• You are able to avoid certain medications and foods that affect how drugs are processed in the body — such as ketoconazole, rifampin, St. John's Wort, grapefruit juice, or any grapefruit products — for at least 30 days before enrolling.
• You are able to swallow capsules.
• If you are male or female and able to have children, you are willing to use effective birth control from signing the consent form until 30 days after your last dose; male participants must also agree not to donate sperm during this period.

Who may not be able to join:

• Your Friedreich's ataxia is caused by a different type of genetic change (such as a point mutation, deletion, or other non-GAA mutation) rather than the GAA repeat expansion.
• You have previously been treated with the study drug, vatiquinone.
• You have a known allergy to vatiquinone or any of its ingredients, including sesame oil, gelatin (from cattle or pigs), titanium dioxide, or red iron oxide.
• Your heart's pumping function (ejection fraction) is below 50%.
• Your diabetes is not well controlled (based on a blood test called HbA1c being above 7.0%) at the time of your screening visit.
• You have had thoughts of suicide in the past 3 months before screening or between your screening and baseline visits, or any suicidal behaviour in the past year (assessed using a standard scale).
• You are currently pregnant or breastfeeding, or you are sexually active but unwilling to use appropriate birth control; females who could become pregnant must have a negative pregnancy test at both the screening and baseline visits.
• Your liver enzyme levels (called AST or ALT) are 2 or more times higher than the normal range at screening.
• Your blood clotting test result (called INR) is 1.5 or more times higher than the normal range, or you have significant bleeding issues, as judged by the study doctor.
• Your kidney function marker (called serum creatinine) is 1.5 or more times higher than the normal range at screening.
• You have other health conditions that the study doctor believes could affect the study results — for example, a condition that affects how your body absorbs fat, or another mitochondrial disorder (confirm with trial site).
• You have taken part in another interventional clinical trial or received any experimental drug within 60 days before your baseline visit (you may be able to re-apply after 60 days have passed).
• You have been taking CoQ10 supplements, vitamin E, or any approved or unapproved medication for Friedreich's ataxia within 30 days before the screening visit (these may be stopped at screening, but additional steps and re-testing will be required — confirm with trial site).
• You have used illegal/illicit drugs in the 30 days before screening or plan to do so during the study.

Important: Always verify eligibility with the trial site directly before applying.