Friedreich's AtaxiaPhase 3PROVE-FANCT07681713
Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)
A Phase 3 study testing Vatiquinone in people with Friedreich's Ataxia, run by PTC Therapeutics at 9 sites in 6 countries.
Trial summary
The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression. Read more about this study
- Who is it for?
- Ages 7 to 21 years
- Conditions the study is for
- Friedreich's Ataxia
- What is being tested?
- Vatiquinone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 120 participants
- Where
- 9 sitesin 6 countries
- Study dates
- 15 Aug 2026to 15 Mar 2029
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 7 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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:
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- 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.
Trial locations and contacts
9 sites in 6 countries. Contact the study team or a site near you to ask about taking part.
9 sites in 6 countries
United States4 sites
University of Iowa
Iowa City, Iowa, 52242
Katherine Mathews
University of California Los Angeles
Los Angeles, California, 90095
Susan Perlman
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
David Lynch
University of South Florida
Tampa, Florida, 33612
Theresa Zesiewicz
Belgium1 site
Hôpital Érasme (Hôpital Universitaire de Bruxelles)
Brussels, 1070
Gilles Naeije
Brazil1 site
University of Campinas
Campinas, São Paulo, 13083-888
Marcondes França
Canada1 site
Centre Hospitalier de l'Universite de Montreal CHUM
Montreal, Quebec, H2X 0A9
Antoine Duquette
France1 site
Institut du Cerveau et de la Moelle Epinière
Paris, 75013
Alexandra Durr
About this study
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.
- Conditions
- Friedreich's Ataxia
- Being tested
- Vatiquinone
- Medical subject headings
- Friedreich Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VatiquinoneDrug
Also called PTC743
Vatiquinone will be administered per dose and schedule specified in the arm.
| Study group | Role | What they receive |
|---|---|---|
| Vatiquinone | Experimental | Participants will receive vatiquinone capsule at a dose of either 200 milligrams (mg) orally 3 times a day (TID) if weighing ˂25 kilograms (kg) or 400 mg orally TID if weighing ≥25 kg for 24 months. Receives: Drug: Vatiquinone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Total Modified Friedreich's Ataxia Rating Scale (mFARS) Score at Month 24
Measured: Baseline, Month 24
Secondary outcomes (4)
Change From Baseline in mFARS Subscale Scores (Upright Stability Subscale [USS], Upper Limb [UL], Lower Limb [LL], Bulbar [BUL]) at Month 24
Measured: Baseline, Month 24
Change From Baseline in Friedreich's Ataxia Rating Scale - Activities of Daily Living (FARS-ADL) Score at Month 24
Measured: Baseline, Month 24
Change From Baseline in 25-Foot Walk Test (T25FW) at Month 24
Measured: Baseline, Month 24
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Measured: Baseline up to Month 25
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jul 2026
- Study start15 Aug 2026 (estimated)
- Primary completion15 Mar 2029 (estimated)
- Study completion15 Mar 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Friedreich's AtaxiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 120 participants (estimated)
Sponsor and investigators
- Lead sponsor
- PTC Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07681713
- Sponsor's protocol ID
- PTC743-FA-304
- Other IDs
- 2026-526947-37-00
- Acronym
- PROVE-FA
- Status last verified
- Aug 2026
- First submitted
- 26 Jun 2026
- Last updated
- 14 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No