What side effects are associated with this type of intervention?

Common treatments for mitochondrial diseases, such as vatiquinone, often aim to improve cellular energy production and reduce oxidative stress. Known side effects of these treatments can include gastrointestinal issues (nausea, vomiting, diarrhea), fatigue, and potential liver toxicity. Other treatments like branched-chain amino acids, taurine, and vitamin E may also cause mild gastrointestinal discomfort. It is important to monitor for these side effects and discuss them with a healthcare provider to ensure safe and effective management of the condition.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for mitochondrial diseases that are directly comparable to vatiquinone. Vatiquinone is being studied for its safety in inherited mitochondrial diseases, and it represents a novel approach in this area. Generally, treatments for mitochondrial diseases focus on managing symptoms and improving quality of life, often involving supplements like coenzyme Q10, which has shown some benefits in mitochondrial function. However, these treatments are not FDA-approved specifically for mitochondrial diseases but are used off-label based on their potential benefits.

What other types of research exist?

Promising novel alternatives to Vatiquinone for mitochondrial disease patients may include treatments currently under investigation in clinical trials for neurodegenerative and metabolic disorders. These could involve agents targeting oxidative stress, mitochondrial biogenesis, or specific genetic mutations associated with mitochondrial dysfunction. Patients should consult with their healthcare provider to explore the latest clinical trials and emerging therapies tailored to their specific condition.

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Quinone Analog

Vatiquinone Safety for Mitochondrial Disease

Phase 3

Waitlist Available

Research Sponsored by PTC Therapeutics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial is testing the safety of vatiquinone, a medication that helps cells produce energy, in people with inherited mitochondrial disease. These participants have already used vatiquinone in previous studies. The study will continue for an extended period. Vatiquinone, also known as EPI-743, is a new oral antioxidant that has been evaluated for mitochondrial diseases.

Who is the study for?

This trial is for people with inherited mitochondrial diseases who have previously taken vatiquinone in a study or treatment plan. Participants must not be pregnant, breastfeeding, or involved in another interventional study. Women of childbearing age and fertile men must agree to use effective contraception during the study.

What is being tested?

The safety of vatiquinone is being tested in individuals with inherited mitochondrial disease who have used it before. The trial will monitor participants until vatiquinone is available commercially or the program ends.

What are the potential side effects?

Specific side effects are not listed here, but as this is a safety study for vatiquinone, all potential side effects experienced by participants will be closely monitored and recorded throughout the duration of the trial.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups

Experimental Treatment

Group I: VatiquinoneExperimental Treatment1 Intervention

Participants will receive vatiquinone oral solution (100 milligrams \[mg\]/milliliter \[mL\]), up to 400 mg, administered orally or via feeding tube 3 times daily (TID).

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Vatiquinone

2020

Completed Phase 3

~160

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for mitochondrial diseases, such as vatiquinone, often focus on enhancing mitochondrial function and reducing oxidative stress. Vatiquinone, for instance, is an antioxidant that helps to mitigate the damage caused by reactive oxygen species (ROS) within the mitochondria. This is crucial for patients with mitochondrial diseases because their cells are typically less efficient at producing energy and more prone to oxidative damage. By reducing oxidative stress, these treatments aim to improve cellular energy production and overall cell function, which can alleviate symptoms and slow disease progression.

Three-Year Follow-Up of High-Dose Ubiquinol Supplementation in a Case of Familial Multiple System Atrophy with Compound Heterozygous COQ2 Mutations.