A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia

  • End date
    Aug 31, 2024
  • participants needed
  • sponsor
    PTC Therapeutics
Updated on 7 October 2022
genetic testing
fxn gene


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.

Condition Friedreich Ataxia
Treatment Vatiquinone
Clinical Study IdentifierNCT05485987
SponsorPTC Therapeutics
Last Modified on7 October 2022


Yes No Not Sure

Inclusion Criteria

Friedreich ataxia diagnosis (homozygous for GAA repeat expansion in intron-1 of FXN gene), confirmed by clinical genetic testing
Must be able to abstain from anticoagulants and any aspirin (including 81 mg) for 30 days before 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 strong 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 and for the duration of the study
Must be able to take vatiquinone oral solution with food

Exclusion Criteria

Previous treatment with vatiquinone
Allergy to vatiquinone or sesame oil
Ejection fraction <50%
Participation in any other interventional clinical trial or receipt of any study drug in any other clinical trial within 60 days prior to the Baseline Visit. Participants may be rescreened after the exclusionary period of 60 days has passed
Concomitant use of interventional CoQ10, vitamin E, or any approved or non-approved medication for FA within 30 days prior to the Baseline Visit. These prohibited medications can be discontinued at the Screening Visit
Note: Other inclusion and exclusion criteria may apply
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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