The main criteria for participation
The trial is recruiting, Par invitation, 100 patients of all ages, suffering from mitochondrial disease and who have already received vatiquinone (PTC743), in several countries around the world, including France.
The objective
The trial aims to evaluate the safety of vatiquinone (PTC743).
In practice
The patient takes vatiquinone (PTC743) three times a day orally (oral solution), or through a feeding tube if they require enteral nutrition. During this period, all adverse events are recorded. The study is expected to last until vatiquinone receives marketing authorization (MA).
In France, the study is being conducted at the Angers University Hospital, the Montpellier University Hospital (Saint-Éloi Hospital), the Necker-Enfants Malades Hospital in Paris and the Hautepierre Hospital in Strasbourg.
Where are we?
Recruitment (by invitation) is underway.