What is the mechanism of action of this treatment?

The most common treatments for Friedreich Ataxia (FA) focus on mitigating oxidative stress, a key factor in the disease's progression. Vatiquinone, an antioxidant, targets this oxidative stress by neutralizing free radicals and reducing mitochondrial damage. This is crucial for FA patients as oxidative stress contributes significantly to the neurodegeneration and muscle weakness characteristic of the disease. By reducing oxidative damage, treatments like vatiquinone aim to slow disease progression and improve quality of life for patients.

What side effects are associated with this type of intervention?

Common treatments for Friedreich Ataxia that target oxidative stress include antioxidants such as idebenone and vatiquinone. Known side effects of these treatments can include gastrointestinal issues like nausea and diarrhea, as well as potential liver enzyme abnormalities. Patients may also experience fatigue and headache. It is important to monitor for these side effects and discuss any concerns with a healthcare provider.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for Friedreich Ataxia that target oxidative stress similar to Vatiquinone. The primary focus of current research, including the study of Vatiquinone, is on managing symptoms and slowing disease progression through antioxidant mechanisms. Patients and healthcare providers often rely on supportive therapies and investigational drugs in clinical trials to address the oxidative stress associated with Friedreich Ataxia.

What other types of research exist?

Promising, novel alternatives to Vatiquinone for Friedreich Ataxia patients may include ultra-high-dose methylcobalamin and antioxidants such as vitamin E, which have shown neuroprotective effects in other neurodegenerative diseases.

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Quinone Analog

Vatiquinone for Friedreich Ataxia

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

Males and females of childbearing potential must be willing to use an effective method of contraception (for example, implants, injectables, transdermal patches, combined oral contraceptives, barrier methods, and intrauterine devices) from the time consent is signed until 30 days after the last dose of study drug or Early Termination Visit.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Pivotal Trial

No Placebo-Only Group

Summary

This trial is testing the long-term safety and effectiveness of vatiquinone in people with Friedreich ataxia who have already used this medication. The goal is to ensure it remains safe and helpful over an extended period. Idebenone, a predecessor to vatiquinone, has been used in Friedreich's ataxia patients and shown positive effects on cardiac hypertrophy.

Who is the study for?

This trial is for people with Friedreich ataxia who were previously in a vatiquinone study. Participants must be willing to use effective contraception from consent until 30 days after the last dose or if they leave the study early.

What is being tested?

The trial is testing the long-term safety and effectiveness of a drug called vatiquinone in patients with Friedreich ataxia, specifically those who have taken it before in earlier trials.

What are the potential side effects?

While specific side effects are not listed here, vatiquinone may cause reactions that can vary from person to person. It's important to monitor any changes and report them to the healthcare team.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I am willing to use effective birth control during and for 30 days after the study.

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

Pivotal Trial

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

No Placebo-Only Group

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

Trial Design

1Treatment groups

Experimental Treatment

Group I: VatiquinoneExperimental Treatment1 Intervention

Participants with FA who were previously treated with vatiquinone at an investigational site and completed participation in a prior PTC Therapeutics (PTC)-sponsored clinical study will continue to receive the same dose/formulation of vatiquinone (unless there has been a change in age and/or weight that meets the criteria for a different dose/formulation as described below; dose/formulation will also be changed if participants meet the criteria for a different dose/formulation during study treatment): If \<7 years of age, participants will receive an oral solution (100 milligrams \[mg\]/milliliter \[mL\]) 3 times a day (TID) at one of the following doses: 15 mg/kilogram (kg) if body weight \<13 kg, or 200 mg if body weight ≥13 kg. If ≥7 years of age, participants will receive a capsule formulation (200 mg) orally TID at one of the following doses: 200 mg if body weight ˂25 kg, or 400 mg if body weight ≥25 kg.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Vatiquinone

2020

Completed Phase 3

~160

0 / 1Eligibility criteria met

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

The most common treatments for Friedreich Ataxia (FA) focus on mitigating oxidative stress, a key factor in the disease's progression. Vatiquinone, an antioxidant, targets this oxidative stress by neutralizing free radicals and reducing mitochondrial damage. This is crucial for FA patients as oxidative stress contributes significantly to the neurodegeneration and muscle weakness characteristic of the disease. By reducing oxidative damage, treatments like vatiquinone aim to slow disease progression and improve quality of life for patients.

Therapeutic role of quercetin on oxidative damage induced by acrylamide in rat brain.