What is the mechanism of action of this treatment?

Common treatments for Friedreich Ataxia, such as Vatiquinone, focus on their antioxidant and anti-inflammatory properties. These treatments work by reducing oxidative stress and inflammation, which are key contributors to the neurodegenerative processes in FA. By mitigating these factors, the treatments aim to slow disease progression and improve neurological function, thereby addressing the underlying causes of the disease and potentially enhancing the quality of life for FA patients.

What side effects are associated with this type of intervention?

Common treatments for Friedreich Ataxia that have antioxidant and anti-inflammatory properties include idebenone and various forms of coenzyme Q10. Known side effects of these treatments can include gastrointestinal discomfort, such as nausea and diarrhea, as well as potential liver enzyme abnormalities. Additionally, some patients may experience headaches and dizziness. It is important to monitor these side effects and discuss them with a healthcare provider to ensure optimal management of the condition.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for Friedreich Ataxia that focus on antioxidant and anti-inflammatory properties similar to Vatiquinone. The current management of Friedreich Ataxia primarily involves supportive care and symptom management. Research is ongoing to find effective treatments, including the study of Vatiquinone, which aims to assess its pharmacokinetics and safety in young patients with Friedreich Ataxia.

What other types of research exist?

Promising novel alternatives to Vatiquinone for Friedreich Ataxia patients include quercetin, bisquercetin-diglucoside, alpha-lipoic acid, and omega-3 fatty acids. These compounds have demonstrated significant antioxidant and neuroprotective properties in various studies.

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Vatiquinone for Friedreich Ataxia

Phase 2

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

Must be able to abstain from anticoagulants and any aspirin (including 81 mg) for 30 days before the Baseline Visit and for the duration of the study; any possible discontinuation of anticoagulants should be monitored and indicated by a specialist (for example, cardiologist, neurologist, or hematologist), and discontinuation will be noted by the prescribing physician

Must be able to abstain from strong cytochrome P450 (CYP) 3A4 inducers/inhibitors (for example, ketoconazole, rifampin, St. John's wort, grapefruit juice, or any grapefruit product) for at least 30 days prior to enrollment and for the duration of the study.

Must not have

Previous treatment with vatiquinone

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a medication called vatiquinone to see how it behaves in the bodies of children under 7 years old who have a genetic disorder called Friedreich ataxia. The goal is to understand if the medication is safe and how it is processed by their bodies.

Who is the study for?

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.

What is being tested?

The trial is studying vatiquinone in young participants with Friedreich ataxia. It aims to understand how the body processes the drug (pharmacokinetics) and evaluate its safety when administered alongside a meal.

What are the potential side effects?

While not explicitly listed here, potential side effects may include reactions related to sesame oil (an ingredient), issues due to stopping anticoagulants or other drugs as required by the study protocol.

Eligibility Criteria

Inclusion Criteria

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

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I can stop taking blood thinners and aspirin for 30 days before and during the study, as advised by my doctor.

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I can avoid certain medications and products like grapefruit for 30 days before and during the study.

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I have been diagnosed with Friedreich ataxia through genetic testing.

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I can avoid certain medications and products like grapefruit for 30 days before and during the study.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I have been treated with vatiquinone before.

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.

Trial Design

1Treatment groups

Experimental Treatment

Group I: VatiquinoneExperimental Treatment1 Intervention

Participants will receive an oral solution (100 milligrams \[mg\]/milliliter \[mL\]) of vatiquinone (15 mg/kilogram \[kg\] if body weight \<13 kg and 200 mg if body weight ≥13 kg) 3 times a day (TID) for 72 weeks.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Vatiquinone

2020

Completed Phase 3

~160

0 / 5Eligibility 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

Common treatments for Friedreich Ataxia, such as Vatiquinone, focus on their antioxidant and anti-inflammatory properties. These treatments work by reducing oxidative stress and inflammation, which are key contributors to the neurodegenerative processes in FA. By mitigating these factors, the treatments aim to slow disease progression and improve neurological function, thereby addressing the underlying causes of the disease and potentially enhancing the quality of life for FA patients.

Therapeutic role of quercetin on oxidative damage induced by acrylamide in rat brain.Coenzyme Q10 in the central nervous system and its potential usefulness in the treatment of neurodegenerative diseases.