Vatiquinone for Friedreich Ataxia

Recruiting at 13 trial locations

Overseen By Patient Advocacy

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: PTC Therapeutics

Disqualifiers: Other study, Pregnancy, Breastfeeding

No Placebo Group All 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 Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 3 years

130 Participants Needed

Trial Summary

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.

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

Group I: Vatiquinone Experimental Treatment 1 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.

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