Vatiquinone for Friedreich Ataxia
Trial Summary
What is the purpose of this trial?
This trial is testing a medication called vatiquinone to see how it behaves in the bodies of children under 7 years old who have a genetic disorder called Friedreich ataxia. The goal is to understand if the medication is safe and how it is processed by their bodies.
Will I have to stop taking my current medications?
You will need to stop taking anticoagulants, aspirin, and strong CYP3A4 inducers/inhibitors at least 30 days before the study and during the study. Also, you cannot use interventional CoQ10, vitamin E, or any medication for Friedreich ataxia 30 days before the study.
Eligibility Criteria
This trial is for children under 7 with Friedreich ataxia, confirmed by genetic testing. They must be able to take vatiquinone orally with food and avoid certain medications like anticoagulants, aspirin, and strong CYP3A4 inducers/inhibitors for specific periods before and during the study.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive vatiquinone orally 3 times a day for 72 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- Vatiquinone
Find a Clinic Near You
Who Is Running the Clinical Trial?
PTC Therapeutics
Lead Sponsor
Dr. Matthew B. Klein
PTC Therapeutics
Chief Executive Officer since 2023
BA from the University of Pennsylvania, MD from Yale University School of Medicine, MS in Epidemiology from the University of Washington School of Public Health
Dr. Stuart W. Peltz
PTC Therapeutics
Chief Medical Officer since 2023
MD from Harvard Medical School