130 Participants Needed

Vatiquinone for Friedreich Ataxia

Recruiting at 13 trial locations
PA
Overseen ByPatient Advocacy
Age: Any Age
Sex: Any
Trial Phase: Phase 3
Sponsor: PTC Therapeutics
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

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.

Do I need to stop my current medications to join the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

I am willing to use effective birth control during and for 30 days after the study.
You cannot have participated in a previous study on vatiquinone for FA, called MOVE-FA or PTC743-NEU-005-FA.

Exclusion Criteria

Current participation in any other interventional study
Pregnancy or breastfeeding

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants continue to receive vatiquinone to assess long-term safety and efficacy

3 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • Vatiquinone
Trial Overview 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.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: VatiquinoneExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

PTC Therapeutics

Lead Sponsor

Trials
75
Recruited
6,300+

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

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