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

NCT07681713 | Not Yet Recruiting Phase 3 FDA Drug

• 2 other identifiers: PTC743-FA-3042026-526947-37-00
• Study Type: interventional
• Target: 120
• Locations: 0 countries
• Sites: N/A

Brief Summary

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

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Total Modified Friedreich's Ataxia Rating Scale (mFARS) Score at Month 24

  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

  Baseline, Month 24

• Change From Baseline in Friedreich's Ataxia Rating Scale - Activities of Daily Living (FARS-ADL) Score at Month 24

  Baseline, Month 24

• Change From Baseline in 25-Foot Walk Test (T25FW) at Month 24

  Baseline, Month 24

• Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

  Baseline up to Month 25

Study Arms (1)

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.

Drug: Vatiquinone

Interventions

Vatiquinone DRUG

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

Also known as: PTC743

Vatiquinone

Eligibility Criteria

• Age: 7 Years - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• PTC Therapeutics: lead

MeSH Terms

Conditions

Friedreich Ataxia

Interventions

alpha-tocotrienol quinone

Condition Hierarchy (Ancestors)

Spinocerebellar Degenerations; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Patient Advocacy

CONTACT

1-866-562-4620; medinfo@ptcbio.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 26, 2026
• First Posted: July 2, 2026
• Study Start (Estimated): August 15, 2026
• Primary Completion (Estimated): March 15, 2029
• Study Completion (Estimated): March 15, 2029
• Last Updated: July 2, 2026

Record last verified: 2026-06