Table of Contents
Trial overview
The available trial is a long-term, open-label study of Vatiquinone in patients with Friedreich ataxia.[1] The study is designed to assess both safety and efficacy in people who have already been exposed to Vatiquinone.[1]
This is an interventional study, which means participants receive a treatment and the research team follows the results over time.[1] The study is authorised and listed as Phase 3.[1]
Who can participate
The trial is for patients with Friedreich ataxia who have previously been exposed to Vatiquinone.[1] This makes the study different from a first-use trial, because it focuses on people who already have experience with the study treatment.[1]
The source data does not give more details about age limits, symptom level, or other entry rules.[1]
What is being measured
The main outcomes are adverse events, serious adverse events, laboratory abnormalities, vital signs, and electrocardiograms (ECGs).[1] These measures help researchers see whether treatment is safe and whether it affects the body in a concerning way.[1]
Adverse events are any medical problems that happen during the study, while serious adverse events are more severe problems that may need hospital care or may be life-threatening.[1] Laboratory abnormalities mean unusual blood or other test results, and ECGs are heart tests that record electrical activity.[1]
Study design and phase
This trial is open-label, which means everyone knows that Vatiquinone is being given.[1] The study is in Phase 3, a later stage of clinical testing that usually looks more closely at safety and benefit in a larger group of people.[1]
Vatiquinone is given orally in this study, and the source data lists a dose of 200 mg.[1] The trial plans to enroll 134 participants.[1]
