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Quinone Analog

Vatiquinone for Friedreich Ataxia

Q: Has Vatiquinone met all the requirements for drug safety by the FDA?

Vatiquinone has been studied in Phases 1-3, meaning there is data supporting both its efficacy and safety. Our analysts rated it a 3 on our safety scale.

Q: Are there any spaces available for new participants in this trial?

No, this particular clinical study is not actively recruiting right now. The trial was first posted on November 1st, 2022 and the last update occurred on October 21st, 2022. Currently, there are 48 other trials that are still enrolling patients.

Q: In how many different places is this study being conducted today?

There are 7 locations where this study is being conducted, these include Los Angeles, Montreal and Philadelphia. Additionally, there are 4 other sites. To limit travel requirements, enrolling at the location closest to you is advised.

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 baseline, year 5

Awards & highlights

Study Summary

This trial will study the long-term safety of a drug for people with Friedreich ataxia who have used it before.

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.Check my eligibility

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.See study design

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 ~ baseline, year 5

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, year 5

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, year 5 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Number of Participants With Adverse Events (AEs)

Secondary outcome measures

Change From Baseline in the Modified Friedreich Ataxia Rating Scale (mFARS) at Year 5

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 (PTC743-NEU-003-FA or PTC743-NEU-005-FA) 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

~210

0 / 1Eligibility criteria met

Find a Location

Who is running the clinical trial?

PTC TherapeuticsLead Sponsor

74 Previous Clinical Trials

6,090 Total Patients Enrolled

3 Trials studying Friedreich Ataxia

211 Patients Enrolled for Friedreich Ataxia

Media Library

Vatiquinone (Quinone Analog) Clinical Trial Eligibility Overview. Trial Name: NCT05515536 — Phase 3

Friedreich Ataxia Research Study Groups: Vatiquinone

Friedreich Ataxia Clinical Trial 2023: Vatiquinone Highlights & Side Effects. Trial Name: NCT05515536 — Phase 3

Vatiquinone (Quinone Analog) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05515536 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Has Vatiquinone met all the requirements for drug safety by the FDA?

"Vatiquinone has been studied in Phases 1-3, meaning there is data supporting both its efficacy and safety. Our analysts rated it a 3 on our safety scale."

Answered by AI

Are there any spaces available for new participants in this trial?

"No, this particular clinical study is not actively recruiting right now. The trial was first posted on November 1st, 2022 and the last update occurred on October 21st, 2022. Currently, there are 48 other trials that are still enrolling patients."

Answered by AI

In how many different places is this study being conducted today?

"There are 7 locations where this study is being conducted, these include Los Angeles, Montreal and Philadelphia. Additionally, there are 4 other sites. To limit travel requirements, enrolling at the location closest to you is advised."

Answered by AI