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
- Are there results?
- Not yet: the study is under way
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:
Show all 13Show fewer
- 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
Study at a glance
From the registry record, last updated by the sponsor on 14 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Long-Term Efficacy of Vatiquinone for the Treatment of Friedreich's Ataxia
- ClinicalTrials.gov ID
- NCT07681713
- Acronym / sponsor ID
- PROVE-FA / PTC743-FA-304
- Conditions
- Friedreich's Ataxia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- PTC TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 15 Aug 2026 (estimated)
- Primary completion
- 15 Mar 2029 (estimated)
- Study completion
- 15 Mar 2029 (estimated)
- First posted
- 2 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Vatiquinone | Experimental | Drug: VatiquinoneParticipants 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. |
Who may qualify
- Ages
- 7 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (13 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VatiquinoneAlso: PTC743Vatiquinone will be administered per dose and schedule specified in the arm. | Drug | Vatiquinone |
Primary outcome
Change From Baseline in Total Modified Friedreich's Ataxia Rating Scale (mFARS) Score at Month 24
Time frame: Baseline, Month 24
4 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Aug 2026.
Related trials for Friedreich's Ataxia
RecruitingPhase 1
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
Friedreich's Ataxia (FA)
Active, not recruiting
Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia
Friedreich Ataxia
Recruiting
Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.
Friedreich's Ataxia, Motor Disorders
Active, not recruitingObservational study
Characterisation of the Cognitive Profile of Patients Suffering From Friedreich's Ataxia
Friedreich Ataxia
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
9 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Vatiquinone in people with Friedreich's Ataxia, run by PTC Therapeutics at 9 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 14 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States, Belgium, Brazil, Canada, France and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Patient Advocacy (1-866-562-4620, medinfo@ptcbio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in Total Modified Friedreich's Ataxia Rating Scale (mFARS) Score at Month 24 (time frame: Baseline, Month 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Aug 2026.