The main criteria for participation
The trial is recruiting 130 patients of all ages with Friedreich’s ataxia in several countries around the world, including France, who have already received vatiquinone (PTC743) in previous clinical trials.
The objective
The trial aims to evaluate the long-term efficacy and safety of vatiquinone (PTC743).
In practice
The patient continues treatment with vatiquinone (PTC743) as in the previous clinical trial, unless there have been changes in age and/or weight that require adjusting the treatment, for example, switching from an oral solution to a capsule and/or increasing the dose.
In France, the study is being conducted at the Brain Institute in Paris.
Where are we?
Recruitment is over.