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

The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.

PTC Identifier:
PTC743-FA-304
ClinicalTrials.gov Identifier:
NCT07681713
EudraCT Identifier:
N/A
EU CT ID:
2026-526947-37-00
Not Recruiting

Trial Purpose

Age
7 - 21 Years
Sex
Both (Female and Male)
Healthy Volunteers
No

Key Participation Requirements

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.

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.
Inclusion and Exclusion Criteria
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.
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:
  • a) Use was less than 3 cumulative months and participants have been off treatment for >30 days.
  • b) 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 Summary

Total Participants

This is the number of individuals who are taking part in the trial.

Total Participants
120

Trial Dates

These are the start and end dates for the trial. Note that future dates are estimates that may be updated before or during the trial.

Trial Dates
Aug 2026 - Mar 2029

Phase

These are the different steps that a treatment goes through during clinical development before it is approved for use.

Phase
Phase 3

Placebo Controlled

In this type of study, some participants receive a “placebo,” which looks like the study treatment, but does not actually contain the active treatment.

Placebo Controlled
No

Product

This is any type of drug, device, or other treatment that is being studied in the trial.

Product
Vatiquinone

Accepts Healthy Volunteers

Healthy volunteers are individuals who participate in the trial and do not have that specific disease or condition.

Accepts Healthy Volunteers
No
Discuss with the trial center for details

Trial Design

Trial Type

This is the general nature of the study.

Trial Type
Interventional

Interventional

Participants in these studies are subject to an intervention by researchers. These trials are used to understand the effects of a particular intervention.

Discuss with the trial center for details

Blinding

Study participants know which treatments they might get if they join the trial, however during a trial they may not be informed which treatment they are actually taking. The act of not disclosing this information to the participant is known as “blinding.”

Blinding
N/A
Discuss with the trial center for details

Intervention Type

This is a medicine, device, or other type of treatment that is being studied or is already approved for use. Interventions can also include behavior changes, like diet and exercise.

Intervention Type
Drug

Allocation

This determines how study participants are assigned their treatment.

Allocation
N/A

Trial Purpose

This is the rationale behind why the clinical trial is being performed.

Trial Purpose
Treatment

Trial Arms

These are the groups of participants in a clinical study. Each different “arm” is assigned a different treatment to use. Different types of trial arms in studies can include:

Trial Arms
Experimental

Experimental arm

Experimental arm: The group assigned to use the study treatment

Primary Outcome

Primary Outcome

This is the most important measurement of the study and is used to help researchers evaluate the effect of a treatment.

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

Timeframe: Baseline, Month 24

Secondary Outcome

Secondary Outcome

This measurement is a secondary measurement and is still important in helping researchers evaluate the effect of a treatment.

Change From Baseline in mFARS Subscale Scores (Upright Stability Subscale [USS], Upper Limb [UL], Lower Limb [LL], Bulbar [BUL]) at Month 24

Timeframe: Baseline, Month 24

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

Timeframe: Baseline, Month 24

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

Timeframe: Baseline, Month 24

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

Timeframe: Baseline up to Month 25

Contact Information

No locations found.