Vatiquinone for Friedreich Ataxia
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called vatiquinone for young children with Friedreich ataxia, a rare genetic disorder affecting movement and coordination. The study aims to understand how the body processes the drug and to assess its safety. Children with a confirmed diagnosis of Friedreich ataxia who can avoid certain medications and foods, such as grapefruit, may be suitable for this trial. Participants will take an oral solution of vatiquinone three times a day for about 72 weeks. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important advancements in care.
Will I have to stop taking my current medications?
You will need to stop taking anticoagulants, aspirin, and strong CYP3A4 inducers/inhibitors at least 30 days before the study and during the study. Also, you cannot use interventional CoQ10, vitamin E, or any medication for Friedreich ataxia 30 days before the study.
Is there any evidence suggesting that vatiquinone is likely to be safe for humans?
Research has shown that vatiquinone is generally safe and well tolerated. Studies have found that people of all ages taking vatiquinone did not experience more side effects than those taking a placebo (a pill with no active ingredients). No major differences in unwanted effects appeared between the two groups, suggesting that vatiquinone is unlikely to cause harmful side effects.12345
Why do researchers think this study treatment might be promising?
Unlike the standard treatments for Friedreich Ataxia, which primarily focus on managing symptoms, Vatiquinone offers a new approach by targeting the underlying oxidative stress associated with the disease. Researchers are excited about Vatiquinone because it is a small molecule that works by inhibiting an enzyme called 15-lipoxygenase, potentially reducing oxidative damage in nerve cells. This could lead to better outcomes for patients by addressing the root cause rather than just alleviating symptoms, offering a glimmer of hope for more effective management of this challenging condition.
What evidence suggests that vatiquinone might be an effective treatment for Friedreich ataxia?
Research has shown that vatiquinone, the investigational treatment in this trial, may help treat Friedreich ataxia, a rare genetic disorder affecting the nervous system and movement. In studies with adults, those treated with vatiquinone improved by 4.8 points on a scale measuring symptom severity after 24 weeks. Patients also reported feeling less tired, as measured by a fatigue scale. These findings suggest that vatiquinone could offer real benefits for people with Friedreich ataxia. However, more research is needed to confirm its effectiveness, especially in younger patients.23567
Are You a Good Fit for This Trial?
This trial is for children under 7 with Friedreich ataxia, confirmed by genetic testing. They must be able to take vatiquinone orally with food and avoid certain medications like anticoagulants, aspirin, and strong CYP3A4 inducers/inhibitors for specific periods before and during the study.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive vatiquinone orally 3 times a day for 72 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Vatiquinone
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