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

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Friedreich's Ataxia
• Intervention / Treatment: Drug: Vatiquinone

• Study Type: Interventional
• Enrollment (Estimated): 120
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Patient Advocacy; Phone Number: 1-866-562-4620; Email: medinfo@ptcbio.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

Key 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:

  1. Use was less than 3 cumulative months and participants have been off treatment for >30 days.
  2. 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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Vatiquinone; 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; Vatiquinone will be administered per dose and schedule specified in the arm.; Other Names: PTC743

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Change From Baseline in Total Modified Friedreich's Ataxia Rating Scale (mFARS) Score at Month 24	Baseline, Month 24

Secondary Outcome Measures

Outcome Measure	Time Frame
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

Collaborators and Investigators

• Sponsor: PTC Therapeutics

Study record dates

Study Major Dates

• Study Start (Estimated): August 15, 2026
• Primary Completion (Estimated): March 15, 2029
• Study Completion (Estimated): March 15, 2029

Study Registration Dates

• First Submitted: June 26, 2026
• First Submitted That Met QC Criteria: June 26, 2026
• First Posted (Actual): July 2, 2026

Study Record Updates

• Last Update Posted (Actual): July 2, 2026
• Last Update Submitted That Met QC Criteria: June 26, 2026
• Last Verified: June 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Metabolic Diseases; Neurodegenerative Diseases; Heredodegenerative Disorders, Nervous System; Spinal Cord Diseases; Mitochondrial Diseases; Cerebellar Diseases; Spinocerebellar Degenerations; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases; Friedreich Ataxia; alpha-tocotrienol quinone
• Other Study ID Numbers: PTC743-FA-304; 2026-526947-37-00 (Ctis)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No