ClinicalTrials.gov record NCT07681713
Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)
Evaluation of Long-Term Efficacy of Vatiquinone for the Treatment of Friedreich's Ataxia
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This Phase 3, single-group (open-label) study tests vatiquinone in children and young people with genetically confirmed Friedreich's Ataxia (ages 7–21). Participants take oral vatiquinone three times daily (dose based on body weight) for 24 months. The study measures change from baseline in neurological rating scales and walking tests and records adverse events; its stated purpose is to confirm vatiquinone's effects on key measures of FA disease progression.
What it is studying. The study is testing vatiquinone's effects on neurological and functional measures of Friedreich's Ataxia over 24 months, and collecting safety data. The primary measurement is change from baseline in total modified Friedreich's Ataxia Rating Scale (mFARS) at Month 24; secondary measures include mFARS subscales, FARS-ADL, the 25-foot walk, and counts of adverse events.
What the researchers hope to learn. To confirm vatiquinone's effects on total mFARS score at Month 24; to evaluate changes in mFARS subscales (Upright Stability, Upper Limb, Lower Limb, Bulbar), FARS-ADL, and the 25-foot walk; and to collect data on treatment-emergent and serious adverse events.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- Vatiquinone
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- 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.
- Key
Exclusion criteria
- 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.
Study description and what participation involves
The registry record has no detailed description beyond the summary above.
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants take oral vatiquinone capsules three times a day for 24 months (200 mg TID if weighing <25 kg; 400 mg TID if ≥25 kg). They will attend study visits for clinical assessments including the modified Friedreich's Ataxia Rating Scale (mFARS) and its subscales, FARS‑ADL, the 25‑foot walk test, and safety monitoring for adverse events (safety follow-up extends to about month 25). Screening and baseline assessments (including pregnancy testing where required) must be completed and participants must avoid specified strong CYP3A4 inducers/inhibitors before and during the study. The study record notes ongoing safety and efficacy assessments but does not list visit frequency or all procedures in detail.
How the study is structured. Phase 3 interventional study with one treatment group and no masking. Participants receive vatiquinone capsules three times daily for 24 months (200 mg TID if weight <25 kg; 400 mg TID if weight ≥25 kg). Outcomes are measured at baseline and during follow-up, focusing on mFARS change at Month 24 and safety monitoring.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The registry says recruitment has not started. When is it expected to open, and can I register interest now?
- The listed sites are in Belgium, Brazil, Canada, France, Spain, United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on Friedreich ataxia
Important dates
- Study start
- Aug 15, 2026 (estimated)
- Primary completion
- Mar 15, 2029 (estimated)
- Study completion
- Mar 15, 2029 (estimated)
- Target enrollment
- 120 participants (estimated)
- Last updated on registry
- Aug 14, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- PTC Therapeutics
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Patient Advocacy 📞 1-866-562-4620 ✉ medinfo@ptcbio.com
Participating sites (9)
- University of California Los Angeles
Los Angeles, California, 90095 · United States
- Susan Perlman
- University of South Florida
Tampa, Florida, 33612 · United States
- Theresa Zesiewicz
- University of Iowa
Iowa City, Iowa, 52242 · United States
- Katherine Mathews
- Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104 · United States
- David Lynch
- Hôpital Érasme (Hôpital Universitaire de Bruxelles)
Brussels, 1070 · Belgium
- Gilles Naeije
- University of Campinas
Campinas, São Paulo, 13083-888 · Brazil
- Marcondes França
- Centre Hospitalier de l'Universite de Montreal CHUM
Montreal, Quebec, H2X 0A9 · Canada
- Antoine Duquette
- Institut du Cerveau et de la Moelle Epinière
Paris, 75013 · France
- Alexandra Durr
- Hospital Sant Joan de Deu
Barcelona, 08950 · Spain
- Alejandra Darling
Terms used on this page · explanations by MedicalUpdates.ai
- at Screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- Baseline
- Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
- childbearing potential
- Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
- investigational drug
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- exclusion criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- Key Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this website; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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