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

ConditionFriedreich's Ataxia
Trial statusNot yet recruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age7-21
SponsorPTC Therapeutics

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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.

Trial design

Design model

Single group

Treatments tested in this trial

  • Vatiquinone

    Drug

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

Treatment groups

120 Participants
are divided into 1 treatment group
Group A: VatiquinoneExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

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

Time frame
Baseline, Month 24

Secondary outcomes

1

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
2

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

Time frame
Baseline, Month 24
3

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

Time frame
Baseline, Month 24
4

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

Time frame
Baseline up to Month 25

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

PTC Therapeutics

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: