Not yet recruiting NCT07681713

Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)

Phase III Interventional Friedreich's Ataxia

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied: The protocol lists: Vatiquinone.
Who it may be relevant to: Registry conditions: Friedreich's Ataxia. Basic parameters: 7 years — 21 years · All.
What needs checking: Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place: Center list to be confirmed — check the primary protocol.
Next step: Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Official title

Evaluation of Long-Term Efficacy of Vatiquinone for the Treatment of Friedreich's Ataxia

The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.

Drug Vatiquinone
Vatiquinone will be administered per dose and schedule specified in the arm.

Change From Baseline in Total Modified Friedreich's Ataxia Rating Scale (mFARS) Score at Month 24 [Time frame: Baseline, Month 24]

Secondary outcome measures (4)

Change From Baseline in mFARS Subscale Scores (Upright Stability Subscale [USS], Upper Limb [UL], Lower Limb [LL], Bulbar [BUL]) at Month 24 [Time frame: Baseline, Month 24]
Change From Baseline in Friedreich's Ataxia Rating Scale - Activities of Daily Living (FARS-ADL) Score at Month 24 [Time frame: Baseline, Month 24]
Change From Baseline in 25-Foot Walk Test (T25FW) at Month 24 [Time frame: Baseline, Month 24]
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [Time frame: Baseline up to Month 25]

mFARS ≥20 to ≤70 at Screening (4 weeks prior to Day 1) and Baseline (Day 1).
Must be ambulatory as defined by a E7 score of 4 or less on the USS at Screening and Baseline.
Documentation that participants reached maximum score on items E4, E5, and E3b on the USS of mFARS at Screening and Baseline.
FA diagnosis (homozygous for guanine-adenine-adenine \[GAA\] repeat expansion in intron-1 of the frataxin gene), confirmed and documented with GAA repeat length for both alleles by clinical genetic testing.
Difference in the mFARS score between Screening and Baseline of no more than 4 points.
Ability to abstain from strong cytochrome P450 (CYP) 3A4 inducers/inhibitors (for example, ketoconazole, rifampin, St. John's wort, grapefruit juice) for at least 4 weeks prior to Baseline and for the duration of the study.

Individuals with clinical diagnosis of FA who have point mutations, deletions, or other non-GAA expansion mutations.
Allergy to vatiquinone, sesame oil, gelatin (bovine and/or porcine), titanium dioxide, or red iron oxide.
Pregnant or lactating participants or those sexually active participants who are unwilling to comply with proper birth control methods; females of childbearing potential must have a negative pregnancy test at Screening and during the Baseline Visit.
Comorbidities that may confound study results (for example, fat malabsorption syndrome, other mitochondrial disorder) in the opinion of the investigator.
Participation in an interventional clinical study or received investigational drug (other than SKYCLARYS® \[omaveloxolone\]) within 60 days prior to Screening. Participants may be screened after the exclusionary period of 60 days has passed.
Current use of omaveloxolone. Previous use of omaveloxolone will be allowed if:

Use was less than 3 cumulative months and participants have been off treatment for >30 days.
Use was less than 6 cumulative months and participants have been off treatment for >90 days
Previous or concurrent use of other investigational treatment for FA.
Participation in any cell or gene therapy-based treatment for FA.
Participation in an ongoing study for vatiquinone or current or previous use of vatiquinone.
Illicit drug use 30 days prior to Screening and during the study.

Note: Other protocol-defined inclusion and exclusion criteria may apply.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Center list to be confirmed — check the primary protocol.

NCT: NCT07681713 · PTC743-FA-304 · 2026-526947-37-00