Vatiquinone for Friedreich Ataxia

Not currently recruiting at 16 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the long-term safety of vatiquinone for individuals with Friedreich ataxia, a rare genetic disorder affecting balance and coordination. Participants who were part of a previous study with vatiquinone will continue using the treatment. It specifically targets those who have Friedreich ataxia and completed an earlier vatiquinone study. As a Phase 3 trial, this study represents the final step before FDA approval, giving participants the opportunity to contribute to the potential availability of a new treatment.

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.

Is there any evidence suggesting that vatiquinone is likely to be safe for humans?

Research shows that vatiquinone is generally safe and well-tolerated for people of all ages. Previous studies found no significant difference in unwanted effects between those taking vatiquinone and those taking a placebo (a pill with no active medicine). This suggests that any side effects are likely similar to not taking any treatment. The evidence indicates that vatiquinone could be a safe option for both children and adults with Friedreich ataxia. While more research is needed to confirm its effectiveness, its safety appears promising based on current studies. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Friedreich ataxia?

Vatiquinone is unique because it offers a new approach to treating Friedreich Ataxia by targeting oxidative stress, which is a key factor in the progression of the disease. Unlike traditional treatments that mainly focus on managing symptoms, vatiquinone works at the cellular level to protect nerve cells from damage. Researchers are excited about this treatment because it has the potential to slow down the progression of the disease, which could significantly improve the quality of life for patients.

What evidence suggests that vatiquinone might be an effective treatment for Friedreich ataxia?

Research suggests that vatiquinone, the treatment under study in this trial, might help treat Friedreich ataxia (FA). In one study, participants showed noticeable improvement in their FA symptoms, as measured by a specific assessment tool. Another study found that vatiquinone slowed the progression of FA, with participants showing a 3.7-point improvement in a neurological function score compared to those not receiving the treatment. However, not all results have been positive; one study did not meet its main goal of improving another FA-related score. Overall, some evidence indicates that vatiquinone could help improve neurological function and slow the disease's progression in FA. ^{2 3 4 6 7}

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?

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

Citations

1. neurologylive.com neurologylive.com/view/fda-denies-approval-friedreich-ataxia-agent-vatiquinone-citing-more-efficacy-needed

FDA Denies Approval for Friedreich Ataxia Agent ... Vatiquinone was considered more effective in the open-label study of adults with FA. After 24 weeks of treatment with the therapy, participants ...

2. curefa.org curefa.org/drug-development/vatiquinone-ptc-743/

Vatiquinone (PTC-743) In the analysis of the entire 24-month trial there was suggested evidence of a significant improvement in neurological function based on the FARS score in EPI- ...

3. mdaconference.org mdaconference.org/abstract-library/safety-and-efficacy-of-vatiquinone-treatment-in-friedreich-ataxia-patients-from-move-fa-a-phase-3-double-blind-placebo-controlled-trial/

Safety and Efficacy of Vatiquinone Treatment in Friedreich ... Vatiquinone treatment resulted in a clinically meaningful and statistically significant treatment effect on the USS, a sensitive and predictive endpoint for ...

4. ir.ptcbio.com ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-announces-topline-results-vatiquinone-move-fa?mobile=1

PTC Therapeutics Announces Topline Results from ... The study did not meet its primary endpoint of statistically significant change in mFARS score at 72 weeks in the primary analysis population.

5. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0960896625008144

357PLong-term vatiquinone treatment slows Friedreich's ... The matched FACOMS cohort progressed by 7.48 points over the same period. Vatiquinone treatment resulted in a 3.7-point benefit (p < 0.0001, n = 70) in mFARS ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT05515536

NCT05515536 | A Study to Assess the Safety and Efficacy ... The primary objective of this study is to assess the long-term safety of vatiquinone in participants with Friedreich ataxia (FA) previously exposed to ...

7. clinicaltrials.gov clinicaltrials.gov/study/NCT04577352

NCT04577352 | A Study to Assess the Efficacy and Safety ... The primary objective of the study is to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale [mFARS]) and safety of vatiquinone in ...