Vatiquinone for Friedreich's Ataxia
(PROVE-FA Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.
Are You a Good Fit for This Trial?
This trial is for people diagnosed with Friedreich's Ataxia confirmed by genetic testing, who can walk (even if limited), have a certain range of disease severity, and are able to avoid specific medications and supplements during the study.Inclusion Criteria
What Are the Treatments Tested in This Trial?
Interventions
- Vatiquinone
Trial Overview
The study is testing vatiquinone to see if it can slow down or improve symptoms of Friedreich's Ataxia over time. All participants receive the medication, and their progress will be closely monitored.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Participants will receive vatiquinone capsule at a dose of either 200 milligrams (mg) orally 3 times a day (TID) if weighing ˂25 kilograms (kg) or 400 mg orally TID if weighing ≥25 kg for 24 months.
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
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