In short
Clinical trials are investigating Vatiquinone in people with Friedreich ataxia. These studies aim to learn about long-term safety and possible benefit in patients who have already been exposed to Vatiquinone. The main focus is on safety checks, lab tests, and heart monitoring.
Key points
- Clinical trials of Vatiquinone are studying people with Friedreich ataxia. The trial data provided describes a long-term, open-label Phase 3 study in patients who had already been exposed to Vatiquinone. The main goal is to assess safety and possible benefit over time. Researchers are watching for adverse events, serious adverse events, lab changes, vital signs, and ECG results. The study is authorised and plans to enroll 134 participants. Vatiquinone is given by mouth in this trial.
Trial overview
The available trial is a long-term, open-label study of Vatiquinone in patients with Friedreich ataxia. The study is designed to assess both safety and efficacy in people who have already been exposed to Vatiquinone.
This is an interventional study, which means participants receive a treatment and the research team follows the results over time. The study is authorised and listed as Phase 3.
Who can participate
The trial is for patients with Friedreich ataxia who have previously been exposed to Vatiquinone. This makes the study different from a first-use trial, because it focuses on people who already have experience with the study treatment.
The source data does not give more details about age limits, symptom level, or other entry rules.
What is being measured
The main outcomes are adverse events, serious adverse events, laboratory abnormalities, vital signs, and electrocardiograms (ECGs). These measures help researchers see whether treatment is safe and whether it affects the body in a concerning way.
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. Laboratory abnormalities mean unusual blood or other test results, and ECGs are heart tests that record electrical activity.
Study design and phase
This trial is open-label, which means everyone knows that Vatiquinone is being given. 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.
Vatiquinone is given orally in this study, and the source data lists a dose of 200 mg. The trial plans to enroll 134 participants.
Key trial details
- Trial ID: NCT05515536.
- Title: Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia.
- Condition studied: Friedreich ataxia.
- Status: Authorised.
- Type of study: Interventional.
- Enrollment: 134 participants.